[{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Olipudase alfa","moa":"SMPD1","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Avalglucosidase alfa","moa":"Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Powder for Concentrate for Solution for Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"Marinus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Avalglucosidase alfa","moa":"Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Powder for Concentrate for Solution for Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Avalglucosidase alfa","moa":"Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution For Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Canakinumab","moa":"IL-1 beta","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Satralizumab-mwge","moa":"IL-6R alpha","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Chugai Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Chugai Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Avalglucosidase alfa","moa":"Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution For Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Avalglucosidase alfa","moa":"Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution For Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"GSK","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Mepolizumab","moa":"IL-5","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"GSK \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"GSK \/ Not Applicable"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Agios Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Fosdenopterin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"BridgeBio Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BridgeBio Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Anakinra","moa":"IL-1 receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Swedish Orphan Biovitrum AB \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Abingworth","pharmaFlowCategory":"D","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2021","type":"Private Placement","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0.059999999999999998,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Abingworth","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Abingworth"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Satralizumab-mwge","moa":"IL-6R alpha","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Chugai Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Chugai Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Canakinumab","moa":"IL-1 beta","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Strongbridge Biopharma","sponsor":"Xeris Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Acquisition","leadProduct":"Dichlorphenamide","moa":"CA","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Strongbridge Biopharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Strongbridge Biopharma \/ Xeris Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"Strongbridge Biopharma \/ Xeris Pharmaceuticals"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Liquid Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Enzyvant","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Allogeneic Processed Thymus Tissue-agdc","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Enzyvant","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Enzyvant \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Enzyvant \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Kedrion","sponsor":"Kedrion","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Acquisition","leadProduct":"Plasminogen","moa":"Plasminogen","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Kedrion","amount2":0.02,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Lyophilized Powder For Injection","sponsorNew":"Kedrion \/ Kedrion Biopharma","highestDevelopmentStatusID":"12","companyTruncated":"Kedrion \/ Kedrion Biopharma"},{"orgOrder":0,"company":"Kedrion","sponsor":"Kedrion","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Acquisition","leadProduct":"Plasminogen","moa":"Plasminogen","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Kedrion","amount2":0.02,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Lyophilized Powder for Injection","sponsorNew":"Kedrion \/ Kedrion","highestDevelopmentStatusID":"12","companyTruncated":"Kedrion \/ Kedrion"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"GSK","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Mepolizumab","moa":"DNA synthesis","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"GSK \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"GSK \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"NHS England","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Agreement","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ NHS England","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ NHS England"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Olipudase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Mitapivat","moa":"PKR","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Agios Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Agreement","leadProduct":"Fosdenopterin","moa":"Molybdenum cofactor synthesis","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"12","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Olipudase Alfa","moa":"SMPD1","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Nobelpharma America","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Nobelpharma America","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Nobelpharma America \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Nobelpharma America \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Alpelisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Italian Medicines Agency","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Agreement","leadProduct":"Atidarsagene Autotemcel","moa":"ARSA gene","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ Italian Medicines Agency","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Italian Medicines Agency"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Mitapivat","moa":"PKR","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Agios Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"C5 complement","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Olipudase Alfa","moa":"SMPD1","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Eladocagene Exuparvovec","moa":"AADC gene","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"PTC Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Avalglucosidase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Avalglucosidase Alfa","moa":"Enzyme","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Olipudase Alfa","moa":"SMPD1","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Mitapivat","moa":"PKR","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Agios Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Zenyaku Kogyo","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Rituximab","moa":"CD20","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Zenyaku Kogyo","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Zenyaku Kogyo \/ Chugai Pharmaceutical","highestDevelopmentStatusID":"12","companyTruncated":"Zenyaku Kogyo \/ Chugai Pharmaceutical"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"New Enterprise Associates","pharmaFlowCategory":"D","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2022","type":"Private Placement","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0.059999999999999998,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ New Enterprise Associates","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ New Enterprise Associates"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Birch Bark Extract","moa":"Keratinocytes","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Amryt Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amryt Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Avalglucosidase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Olipudase Alfa","moa":"SMPD1","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Birch Bark Extract","moa":"Transient receptor potential channel","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Amryt Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amryt Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Divestment","leadProduct":"Burosumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Ultragenyx Pharmaceutical","amount2":0.5,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.5,"dosageForm":"Injectable\/Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Kyowa Kirin","highestDevelopmentStatusID":"12","companyTruncated":"Ultragenyx Pharmaceutical \/ Kyowa Kirin"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Eladocagene Exuparvovec","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"PTC Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Nobelpharma America","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Nobelpharma America","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Nobelpharma America \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Nobelpharma America \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"12","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"","productType":"Small molecule","year":"2022","type":"Divestment","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0.11,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.11,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Novo Nordisk","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Novo Nordisk"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutic","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutic","highestDevelopmentStatusID":"12","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutic"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Atidarsagene Autotemcel","moa":"ARSA gene","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Olipudase Alfa-rpcp","moa":"Acid sphingomyelinase","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Nobelpharma America","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Nobelpharma America","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Nobelpharma America \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Nobelpharma America \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"12","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Mitapivat","moa":"PKR","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Agios Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Atidarsagene Autotemcel","moa":"ARSA gene","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Sagard Healthcare Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Financing","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Sagard Healthcare Partners","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Sagard Healthcare Partners"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Oleogel-S10","moa":"Transient receptor potential channel","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Amryt Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amryt Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"C5 complement","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"SVB Securities","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"","productType":"Small molecule","year":"2022","type":"Public Offering","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0.059999999999999998,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ SVB Securities","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ SVB Securities"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Eculizumab","moa":"Completent C5","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Tenacia Biotechnology","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"","productType":"Small molecule","year":"2022","type":"Collaboration","leadProduct":"Ganaxolone","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0.27000000000000002,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.27000000000000002,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Tenacia Biotechnology","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Tenacia Biotechnology"},{"orgOrder":0,"company":"Altavant Sciences","sponsor":"Enzyvant Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Merger","leadProduct":"Allogeneic Processed Thymus Tissue-agdc","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Altavant Sciences","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Altavant Sciences \/ Enzyvant Therapeutics","highestDevelopmentStatusID":"12","companyTruncated":"Altavant Sciences \/ Enzyvant Therapeutics"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Volanesorsen","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"PTC Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Iptacopan Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Iptacopan Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"ANI Pharmaceuticals Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Levocarnitine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"ANI Pharmaceuticals Inc","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"ANI Pharmaceuticals Inc \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"ANI Pharmaceuticals Inc \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Chiesi Farmaceutici","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Acquisition","leadProduct":"Oleogel-S10","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Amryt Pharma","amount2":1.48,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":1.48,"dosageForm":"Gel","sponsorNew":"Amryt Pharma \/ Chiesi Farmaceutici","highestDevelopmentStatusID":"12","companyTruncated":"Amryt Pharma \/ Chiesi Farmaceutici"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Amgen Inc","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"IRELAND","productType":"Large molecule","year":"2022","type":"Acquisition","leadProduct":"Teprotumumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":27.800000000000001,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":27.800000000000001,"dosageForm":"Injectable\/Injection","sponsorNew":"Horizon Therapeutics \/ Amgen Inc","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Amgen Inc"},{"orgOrder":0,"company":"Octapharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Human Pooled Plasma","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Octapharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Octapharma \/ Not Applicable"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Crovalimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"12","companyTruncated":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Avalglucosidase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"New Therapies Council","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Agreement","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ New Therapies Council","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ New Therapies Council"},{"orgOrder":0,"company":"Nobelpharma America","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Sirolimus","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Nobelpharma America","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Nobelpharma America \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Nobelpharma America \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Incyte Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Pemigatinib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Incyte Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Incyte Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Incyte Corporation \/ Not Applicable"},{"orgOrder":0,"company":"CSL Vifor","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Avacopan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"CSL Vifor","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"CSL Vifor \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"CSL Vifor \/ Not Applicable"},{"orgOrder":0,"company":"Zydus Lifesciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Levothyroxine Sodium","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Zydus Lifesciences","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Zydus Lifesciences \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Zydus Lifesciences \/ Not Applicable"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"PANTHERx Rare Pharmacy \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"PANTHERx Rare Pharmacy \/ Not Applicable"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Chiesi Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Acquisition","leadProduct":"Oleogel-S10","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Amryt Pharma","amount2":1.48,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":1.48,"dosageForm":"Gel","sponsorNew":"Amryt Pharma \/ Chiesi Group","highestDevelopmentStatusID":"12","companyTruncated":"Amryt Pharma \/ Chiesi Group"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Iptacopan Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Piper Sandler","pharmaFlowCategory":"D","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2023","type":"Private Placement","leadProduct":"Mavorixafor","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0.070000000000000007,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.070000000000000007,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Piper Sandler","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Piper Sandler"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Apadamtase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Takeda Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Ganaxolone","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Divestment","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0.02,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Tablet","sponsorNew":"Pharming \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Danicopan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Crovalimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"12","companyTruncated":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Greenwich Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Cannabidiol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Greenwich Biosciences","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Greenwich Biosciences \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Greenwich Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"Jazz Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Cannabidiol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Jazz Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Jazz Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Jazz Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Guggenheim Securities, LLC","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Private Placement","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0.19,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.19,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Guggenheim Securities, LLC","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Guggenheim Securities, LLC"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Hercules Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Financing","leadProduct":"Mavorixafor","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Hercules Capital","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Hercules Capital"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Pozelimab-bbfg","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regeneron Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Regeneron Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Agreement","leadProduct":"Pozelimab-bbfg","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orsini Specialty Pharmacy \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"Orsini Specialty Pharmacy \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"Daewoong Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Botulinum toxin type A","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Daewoong Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Daewoong Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Daewoong Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Satralizumab-mwge","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chugai Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Chugai Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Acquisition","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0.39000000000000001,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.47999999999999998,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Kyowa Kirin","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Kyowa Kirin"},{"orgOrder":0,"company":"American Regent","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Levocarnitine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"American Regent","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"American Regent \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"American Regent \/ Not Applicable"},{"orgOrder":0,"company":"Pint Pharma","sponsor":"OrphanDC","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2023","type":"Not Applicable","leadProduct":"Pegcetacoplan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pint Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pint Pharma \/ OrphanDC","highestDevelopmentStatusID":"12","companyTruncated":"Pint Pharma \/ OrphanDC"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Nedosiran","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Pre-Filled Syringe","sponsorNew":"Novo Nordisk \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novo Nordisk \/ Not Applicable"},{"orgOrder":0,"company":"EA Pharma","sponsor":"Eisai","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Risedronate Sodium","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"EA Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"EA Pharma \/ Eisai","highestDevelopmentStatusID":"12","companyTruncated":"EA Pharma \/ Eisai"},{"orgOrder":0,"company":"OMERS Life Sciences","sponsor":"Xencor","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Large molecule","year":"2023","type":"Divestment","leadProduct":"Ravulizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"OMERS Life Sciences","amount2":0.22,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.22,"dosageForm":"Infusion","sponsorNew":"OMERS Life Sciences \/ Xencor","highestDevelopmentStatusID":"12","companyTruncated":"OMERS Life Sciences \/ Xencor"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"ADAMTS13 Recombinant-krhn","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Takeda Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Acquisition","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0.47999999999999998,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.47999999999999998,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Kyowa Kirin","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Kyowa Kirin"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Beneluxa Initiative","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Agreement","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Beneluxa Initiative","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Beneluxa Initiative"},{"orgOrder":0,"company":"Jazz Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Cannabidiol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Jazz Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Jazz Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Jazz Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Avalglucosidase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Danicopan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Iptacopan Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"12","companyTruncated":"Alexion Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Apadamtase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Takeda Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Crovalimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"12","companyTruncated":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Danicopan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Alembic Pharmaceuticals Limited","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Bimatoprost","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Alembic Pharmaceuticals Limited","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution\/Drops","sponsorNew":"Alembic Pharmaceuticals Limited \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Alembic Pharmaceuticals Limited \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Danicopan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Crovalimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"12","companyTruncated":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Harrow","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Acquisition","leadProduct":"Triamcinolone Acetonide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0.13,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.13,"dosageForm":"Suspension","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Harrow","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Harrow"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Iptacopan Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Danicopan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Avenacy","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Desmopressin Acetate","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Avenacy","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avenacy \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Avenacy \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Sentynl Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Fosdenopterin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sentynl Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sentynl Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sentynl Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Danicopan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Divestment","leadProduct":"Mavorixafor","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0.11,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.11,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2024","type":"Not Applicable","leadProduct":"Pegcetacoplan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Swedish Orphan Biovitrum AB \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Swedish Orphan Biovitrum AB \/ Not Applicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Crovalimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"F. Hoffmann-La Roche \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"F. Hoffmann-La Roche \/ Not Applicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Apadamtase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Takeda Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Takeda Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Eculizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Amgen Inc \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amgen Inc \/ Not Applicable"}]
Find Clinical Drug Development Pipelines & Deals | PipelineProspector
Filters
×
FILTER:
Company Name
Year
DEALS // DEV.
Country
Sponsor
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Reset All
Sanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.
