[{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"BridgeBio Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"BridgeBio Pharma","sponsor":"Sentynl Therapeutic","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Sentynl Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : Nulibry (fosdenopterin) is the first therapy for patients in Great Britain with molybdenum cofactor deficiency Type A, an ultra-rare genetic disorder that often progresses rapidly in infants.

                          Brand Name : Nulibry

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          April 16, 2024

                          Lead Product(s) : Fosdenopterin

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : Nulibry (fosdenopterin) is a first-in-class cPMP substrate replacement therapy that was approved by the U.S. Food and Drug Administration (FDA) in 2021 to reduce the risk of mortality in patients with MoCD Type A.

                          Brand Name : Nulibry

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          September 20, 2022

                          Lead Product(s) : Fosdenopterin

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Sentynl Therapeutics

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : NULIBRY (fosdenopterin) is first and only approved therapy in U.S. and in Israel to treat patients with MoCD Type A, an ultra-rare, life-threatening genetic disorder that often progresses rapidly in infants with a median overall survival age of about fou...

                          Brand Name : Nulibry

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          August 10, 2022

                          Lead Product(s) : Fosdenopterin

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Sentynl Therapeutic

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : CHMP recommendation for approval of NULIBRY (fosdenopterin) in European Union (EU) for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A is based on the efficacy and safety data collected to date compared to data from a natural ...

                          Brand Name : Nulibry

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          July 25, 2022

                          Lead Product(s) : Fosdenopterin

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Sentynl Therapeutics

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : Under the Agreement, Sentynl will acquire global rights to NULIBRY and will be responsible for the ongoing development and commercialization of NULIBRY in the United States and developing, manufacturing and commercializing Fosdenopterin globally.

                          Brand Name : Nulibry

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          March 08, 2022

                          Lead Product(s) : Fosdenopterin

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Sentynl Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          06

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : The efficacy of NULIBRY for the treatment of patients with MoCD Type A was established based on data from three clinical trials compared to data from a natural history study in which it showed a meaningful increase in overall survival compared to a natur...

                          Brand Name : Nulibry

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          February 28, 2021

                          Lead Product(s) : Fosdenopterin

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : FDA has accepted its New Drug Application (NDA) for fosdenopterin, a cyclic pyranopterin monophosphate (cPMP) substrate replacement therapy, for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A.

                          Brand Name : ORGN001

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          September 29, 2020

                          Lead Product(s) : Fosdenopterin

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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