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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":"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"},{"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":"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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                            01

                            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 gets Positive CHMP Opinion for Sephience in Treating PKU in Children and Adults

                            Details : Sephience (sepiapterin) is an oral formulation of sepiapterin, has a dual mechanism of action to increase activity of the phenylalanine hydroxylase enzyme. It is being evaluated for Phenylketonuria.

                            Product Name : Sephience

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            April 25, 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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                            02

                            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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                            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 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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                            04

                            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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                            05

                            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 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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                            06

                            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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