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    [{"orgOrder":0,"company":"Censa Pharmaceuticals","sponsor":"PTC Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Acquisition","leadProduct":"Sepiapterin","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Censa Pharmaceuticals","amount2":0.37,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.37,"dosageForm":"Oral","sponsorNew":"Censa Pharmaceuticals \/ PTC Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Censa Pharmaceuticals \/ PTC Therapeutics"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Sepiapterin","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Sepiapterin","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"PTC Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Sepiapterin","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"PTC Therapeutics \/ Not Applicable"}]

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                            01

                            PTC Therapeutics

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                            Lead Product(s) : Sepiapterin

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            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.

                            Brand Name : PTC923

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            October 01, 2024

                            Lead Product(s) : Sepiapterin

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            02

                            PTC Therapeutics

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                            Lead Product(s) : Sepiapterin

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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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.

                            Brand Name : PTC923

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            March 28, 2024

                            Lead Product(s) : Sepiapterin

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            03

                            PTC Therapeutics

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                            Lead Product(s) : Sepiapterin

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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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.

                            Brand Name : PTC923

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            May 17, 2023

                            Lead Product(s) : Sepiapterin

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            04

                            Censa Pharmaceuticals

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                            Lead Product(s) : Sepiapterin

                            Therapeutic Area : Genetic Disease

                            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.

                            Brand Name : Undisclosed

                            Molecule Type : Small molecule

                            Upfront Cash : $10.0 million

                            May 06, 2020

                            Lead Product(s) : Sepiapterin

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : PTC Therapeutics

                            Deal Size : $366.0 million

                            Deal Type : Acquisition

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