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[{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma and Origin Biosciences Gets FDA Acceptance Of Its New Drug Application For Fosdenopterin For The Treatment Of MoCD Type A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Bridgebio Pharma and Affiliate Origin Biosciences Announce fda Approval of Nulibry","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"BridgeBio Pharma and Sentynl Therapeutics Announce Asset Purchase Agreement for BridgeBio Pharma\u2019s NULIBRY\u2122 (Fosdenopterin)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Approved"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma and Sentynl Therapeutics Receive Positive CHMP Opinion for NULIBRY (fosdenopterin) for the Treatment of MoCD Type A","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Approved"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutic","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma, Sentynl Therapeutics and Medison Pharma Announce Approval in Israel for NULIBRY\u00ae (fosdenopterin) for the Treatment of MoCD Type A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma and Sentynl Therapeutics Receive Marketing Authorization in the EU for NULIBRY\u00ae (fosdenopterin) for the Treatment of MoCD Type A","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Approved"},{"orgOrder":0,"company":"Sentynl Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sentynl Therapeutics Receives MHRA Authorization of NULIBRY\u00ae (fosdenopterin) for Treatment of MoCD Type A in Great Britain","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2024","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Approved"}]

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            Details:

            NULIBRY (fosdenopterin) for Injection, the first therapy for the treatment of patients in Great Britain with molybdenum cofactor deficiency Type A, an ultra-rare, life-threatening genetic disorder that often progresses rapidly in infants.

            Lead Product(s): Fosdenopterin

            Therapeutic Area: Rare Diseases and Disorders Product Name: Nulibry

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 16, 2024

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

            Lead Product(s): Fosdenopterin

            Therapeutic Area: Rare Diseases and Disorders Product Name: Nulibry

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Sentynl Therapeutics

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 20, 2022

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

            Lead Product(s): Fosdenopterin

            Therapeutic Area: Genetic Disease Product Name: Nulibry

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Sentynl Therapeutic

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 10, 2022

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

            Lead Product(s): Fosdenopterin

            Therapeutic Area: Rare Diseases and Disorders Product Name: Nulibry

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Sentynl Therapeutics

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 25, 2022

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

            Lead Product(s): Fosdenopterin

            Therapeutic Area: Rare Diseases and Disorders Product Name: Nulibry

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Sentynl Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement March 08, 2022

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            Details:

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

            Lead Product(s): Fosdenopterin

            Therapeutic Area: Genetic Disease Product Name: Nulibry

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 28, 2021

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            Details:

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

            Lead Product(s): Fosdenopterin

            Therapeutic Area: Genetic Disease Product Name: ORGN001

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 29, 2020

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