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    [{"orgOrder":0,"company":"Censa Pharmaceuticals","sponsor":"PTC Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Endocrinology","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Acquisition","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Endocrinology","graph2":"Phase II","graph3":"Censa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Endocrinology","amount2New":0,"dosageForm":"Oral","sponsorNew":"Censa Pharmaceuticals \/ PTC Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Censa Pharmaceuticals \/ PTC Therapeutics"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Powder","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Powder","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Powder","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Inapplicable"}]

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    PTC Therapeutics

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    Lead Product(s) : Sepiapterin

    Therapeutic Area : Genetic Disease

    Study Phase : Phase III

    Sponsor : Inapplicable

    Deal Size : Inapplicable

    Deal Type : Inapplicable

    DEALS_DEV arrow-down

    PTC Therapeutics Presents New Sepiapterin Data from Ongoing Studies

    Details : PTC923 (sepiapterin) is an oral formulation, that has a dual moa to increase the activity of the PAH enzyme. It is being evaluated for the treatment of pediatric and adult phenylketonuria patients.

    Product Name : PTC923

    Product Type : Other Small Molecule

    Upfront Cash : Inapplicable

    March 20, 2025

    Lead Product(s) : Sepiapterin

    Therapeutic Area : Genetic Disease

    Highest Development Status : Phase III

    Sponsor : Inapplicable

    Deal Size : Inapplicable

    Deal Type : Inapplicable

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    PTC Therapeutics

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    Lead Product(s) : Sepiapterin

    Therapeutic Area : Genetic Disease

    Study Phase : Phase III

    Sponsor : Inapplicable

    Deal Size : Inapplicable

    Deal Type : Inapplicable

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    PTC Announces Submission of Sepiapterin MAA for Treatment of PKU to EMA

    Details : PTC923 (sepiapterin) is a precursor to intracellular tetrahydrobiopterin that acts as nitric oxide synthase modulator. It is being evaluated for the treatment of Phenylketonuria.

    Product Name : PTC923

    Product Type : Other Small Molecule

    Upfront Cash : Inapplicable

    March 28, 2024

    Lead Product(s) : Sepiapterin

    Therapeutic Area : Genetic Disease

    Highest Development Status : Phase III

    Sponsor : Inapplicable

    Deal Size : Inapplicable

    Deal Type : Inapplicable

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    03

    PTC Therapeutics

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    Lead Product(s) : Sepiapterin

    Therapeutic Area : Genetic Disease

    Study Phase : Phase III

    Sponsor : Inapplicable

    Deal Size : Inapplicable

    Deal Type : Inapplicable

    DEALS_DEV arrow-down

    PTC’s Sepiapterin NDA Accepted by FDA for Phenylketonuria

    Details : PTC923 (sepiapterin) is an oral formulation, that has a dual moa to increase the activity of the PAH enzyme. It is being evaluated for the treatment of pediatric and adult phenylketonuria patients.

    Product Name : PTC923

    Product Type : Other Small Molecule

    Upfront Cash : Inapplicable

    January 10, 2024

    Lead Product(s) : Sepiapterin

    Therapeutic Area : Genetic Disease

    Highest Development Status : Phase III

    Sponsor : Inapplicable

    Deal Size : Inapplicable

    Deal Type : Inapplicable

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    PTC Therapeutics

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    Lead Product(s) : Sepiapterin

    Therapeutic Area : Genetic Disease

    Study Phase : Phase III

    Sponsor : Inapplicable

    Deal Size : Inapplicable

    Deal Type : Inapplicable

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    PTC Therapeutics Announces APHENITY Trial Achieved Primary Endpoint with Sepiapterin in PKU Patients

    Details : PTC923 (sepiapterin) is a precursor to intracellular tetrahydrobiopterin, which is a critical enzymatic cofactor involved in the metabolism and synthesis of numerous metabolic products and has the potential to treat the broad range of PKU patients.

    Product Name : PTC923

    Product Type : Other Small Molecule

    Upfront Cash : Inapplicable

    May 17, 2023

    Lead Product(s) : Sepiapterin

    Therapeutic Area : Genetic Disease

    Highest Development Status : Phase III

    Sponsor : Inapplicable

    Deal Size : Inapplicable

    Deal Type : Inapplicable

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    Censa Pharmaceuticals

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    Lead Product(s) : Sepiapterin

    Therapeutic Area : Endocrinology

    Study Phase : Phase II

    Sponsor : PTC Therapeutics

    Deal Size : $366.0 million

    Deal Type : Acquisition

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    PTC Therapeutics to Acquire Censa Pharmaceuticals

    Details : Censa Pharmaceuticals acquisition diversifies and strengthens PTC 's portfolio of rare disorders by adding the lead orphan metabolic disease drug, CNSA-001.

    Product Name : Undisclosed

    Product Type : Other Small Molecule

    Upfront Cash : $10.0 million

    June 05, 2020

    Lead Product(s) : Sepiapterin

    Therapeutic Area : Endocrinology

    Highest Development Status : Phase II

    Sponsor : PTC Therapeutics

    Deal Size : $366.0 million

    Deal Type : Acquisition

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