[{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Divestment","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0.11,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2022","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2023","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2021","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Endocrinology","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Endocrinology","graph2":"Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Endocrinology","amount2New":0,"dosageForm":"Injection, Powder, Lyophilized","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Icon Plc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Icon Plc","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ Icon Plc"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"BMN 331","moa":"C1-INH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Orsini Specialty Pharmacy","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"BMN 351","moa":"DMD exon 51","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Inozyme Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2025","type":"Acquisition","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0.27000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.27000000000000002,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Inozyme Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2025","type":"Acquisition","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0.27000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.27000000000000002,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Nephrology","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"BMN 255","moa":"Glycollate oxidase","graph1":"Nephrology","graph2":"Phase I","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Nephrology","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Spruce Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Acquisition","leadProduct":"Tralesinidase Alfa","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Spruce Biosciences","highestDevelopmentStatusID":"8","companyTruncated":"BioMarin Pharmaceutical \/ Spruce Biosciences"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"DiNAQOR AG","pharmaFlowCategory":"D","therapeuticArea":"Cardiology\/Vascular Diseases","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2020","type":"Licensing Agreement","leadProduct":"DiNA-001","moa":"MYBPC3","graph1":"Cardiology\/Vascular Diseases","graph2":"IND Enabling","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Cardiovascular Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"5","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2025","type":"Inapplicable","leadProduct":"Pegvaliase","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Pegvaliase","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2025","type":"Inapplicable","leadProduct":"Pegvaliase","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"TAIWAN","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"BH4","moa":"Phenylalanine hydroxylase (PAH)","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Tyrosine 3-monooxygenase (TH)","graph1":"Musculoskeletal","graph2":"Phase I","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"6","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BMN 307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BMN 307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"BMN 307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BMN 307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BMN 307","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"BMN 349","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2025","type":"Inapplicable","leadProduct":"BMN 390","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"CAMP4 Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"3","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Entos Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"}]

Find Clinical Drug Pipeline Developments & Deals by BioMarin Pharmaceutical

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

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : Vosoritide is a Peptide drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Hypochondroplasia.

                          Product Name : Undisclosed

                          Product Type : Peptide, Unconjugated

                          Upfront Cash : Inapplicable

                          August 17, 2025

                          Lead Product(s) : Vosoritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Icon Plc

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          02

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : BMN 390 was a preclinical enzyme therapy for phenylketonuria (PKU), designed to break down phenylalanine with reduced immunogenicity by using a novel non-PEG polymer for fewer reactions.

                          Product Name : BMN 390

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          August 05, 2025

                          Lead Product(s) : BMN 390

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Preclinical

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          03

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : Vosoritide is a Peptide drug candidate, which is currently being evaluated in Phase III clinical studies for the treatment of Hypochondroplasia.

                          Product Name : Undisclosed

                          Product Type : Peptide, Unconjugated

                          Upfront Cash : Inapplicable

                          July 18, 2025

                          Lead Product(s) : Vosoritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          04

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : The acquisition will strengthen BioMarin’s enzyme therapies portfolio, adding a late-stage enzyme replacement therapy, INZ-701, for ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency.

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : $270.0 million

                          July 01, 2025

                          Lead Product(s) : INZ-701

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Recipient : Inozyme Pharma

                          Deal Size : $270.0 million

                          Deal Type : Acquisition

                          blank

                          05

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : The acquisition will strengthen BioMarin's enzyme therapies portfolio, adding a late-stage enzyme replacement therapy, INZ-701, which is currently being assessed for the treatment of ENPP1 Deficiency.

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : $270.0 million

                          May 16, 2025

                          Lead Product(s) : INZ-701

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Recipient : Inozyme Pharma

                          Deal Size : $270.0 million

                          Deal Type : Acquisition

                          blank

                          06

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : Through the acquisition, Spruce will focus on the clinical development of AX 250 (tralesinidase alfa) enzyme replacement therapy (TA-ERT) for the treatment of Sanfilippo Syndrome Type B.

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Undisclosed

                          April 15, 2025

                          Lead Product(s) : Tralesinidase Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Spruce Biosciences

                          Deal Size : Undisclosed

                          Deal Type : Acquisition

                          blank

                          07

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : Palynziq (pegvaliase) is a PEG PAL enzyme that converts phenylalanine to ammonia and trans-cinnamic acid. It reduces blood phenylalanine concentrations in patients with PKU.

                          Product Name : Palynziq

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          April 02, 2025

                          Lead Product(s) : Pegvaliase

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          08

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : Pegvaliase is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Phenylketonurias.

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          January 17, 2025

                          Lead Product(s) : Pegvaliase

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          09

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : BMN 349 is a drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of alpha 1-Antitrypsin Deficiency.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          December 17, 2024

                          Lead Product(s) : BMN 349

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          10

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : Vosoritide is a Peptide drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Dwarfism.

                          Product Name : Undisclosed

                          Product Type : Peptide, Unconjugated

                          Upfront Cash : Inapplicable

                          October 31, 2024

                          Lead Product(s) : Vosoritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank