[{"orgOrder":0,"company":"Natco Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Natco Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Natco Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Natco Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Chugai Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Chugai Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet, Film Coated","sponsorNew":"Chugai Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Chugai Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ PTC Therapeutics"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Phase II","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ PTC Therapeutics"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ PTC Therapeutics"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ PTC Therapeutics"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ PTC Therapeutics"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ Inapplicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"F. Hoffmann-La Roche \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"F. Hoffmann-La Roche \/ PTC Therapeutics"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Collaboration","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Royalty Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Acquisition","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Royalty Pharma","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Royalty Pharma"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Licensing Agreement","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Spinal Muscular Atrophy Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Partnership","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Spinal Muscular Atrophy Foundation","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Spinal Muscular Atrophy Foundation"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Royalty Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Agreement","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Royalty Pharma","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Royalty Pharma"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Genentech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"Genentech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"Genentech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Inapplicable"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"Genentech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Inapplicable"},{"orgOrder":0,"company":"Genentech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"Genentech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Genentech \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Genentech \/ Inapplicable"}]

Find Clinical Drug Pipeline Developments & Deals for Risdiplam

Menu
Xls
Filters Filter
×
FILTER:
filter
Company Name
    filter

    Year

      filter

      DEALS // DEV.

        filter

        Country

          filter
          Sponsor
            filter

            Therapeutic Area

              filter

              Study Phase

                filter

                Deal Type

                  filter

                  Product Type

                    filter

                    Dosage Form

                      filter

                      Lead Product

                        filter

                        Target

                          Loading...

                          Therapeutic Area by Lead Product

                          Study Phase by Lead Product

                          Company by Lead Product

                          Top Deals by Deal Size (USD bn)

                          01

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Evrysdi (risdiplam) is a survival of motor neuron 2 (SMN2) splicing modifier indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

                          Product Name : Evrysdi

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 29, 2025

                          Lead Product(s) : Risdiplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          02

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier, now approved in tablet form for the treatment of patients with spinal muscular atrophy.

                          Product Name : Evrysdi

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          February 12, 2025

                          Lead Product(s) : Risdiplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          03

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Risdiplam is a survival of motor neuron 2 (SMN2) splicing modifier, which is being evaluated for the treatment of spinal muscular atrophy.

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          November 21, 2024

                          Lead Product(s) : Risdiplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          04

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Evrysdi (risdiplam) is a survival of motor neuron 2 (SMN2) splicing modifier, which is being evaluated for the treatment of infants with pre-symptomatic spinal muscular atrophy.

                          Product Name : Evrysdi

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          October 14, 2024

                          Lead Product(s) : Risdiplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Chugai Pharmaceutical

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          05

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Evrysdi (risdiplam) is a survival of motor neuron 2 (SMN2) splicing modifier, which is being evaluated for the treatment of infants with pre-symptomatic spinal muscular atrophy.

                          Product Name : Evrysdi

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          February 15, 2024

                          Lead Product(s) : Risdiplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          06

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Under the agreement, Royalty Pharma acquires additional royalties on Evrysdi (risdiplam), a survival motor neuron 2 splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron protein deficiency.

                          Product Name : Evrysdi

                          Product Type : Other Small Molecule

                          Upfront Cash : $1,000.0 million

                          October 19, 2023

                          Lead Product(s) : Risdiplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Royalty Pharma

                          Deal Size : $1,500.0 million

                          Deal Type : Agreement

                          blank

                          07

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency, which is used for babies under two months old with spinal mu...

                          Product Name : Evrysdi

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          August 29, 2023

                          Lead Product(s) : Risdiplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          08

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Evrysdi (risdiplam) is the only non-invasive spinal muscular atrophy therapy approved to treat people of all ages in the European Union now including babies from birth, Approval was based on interim data from ongoing RAINBOWFISH trial.

                          Product Name : Evrysdi

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          August 29, 2023

                          Lead Product(s) : Risdiplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          09

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency.

                          Product Name : Evrysdi

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          July 21, 2023

                          Lead Product(s) : Risdiplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          10

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency.

                          Product Name : Evrysdi

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          June 30, 2023

                          Lead Product(s) : Risdiplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank