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PTC Therapeutics (PTCT) Drug Pipeline

Drug pipeline
Drug NameConditionStageStatusStudy NameTrial Start DateArticle
Ptc844Rheumatoid ArthritisPhase IINot Yet RecruitingStudy of PTC844 in Participants With Active Rheumatoid ArthritisAug 18, 2026―
VatiquinoneFriedreich's AtaxiaPhase IIINot Yet RecruitingLong-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)Jun 26, 2026
Sepiapterin, Ptc923, SephiencePhenylketonuriaPhase IIIRecruitingA Study of Sepiapterin in Participants With Phenylketonuria (PKU)Mar 04, 2024―
Ptc518Huntington DiseasePhase IIEnrolling By InvitationAn Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)Feb 02, 2024―
VatiquinoneFriedreich AtaxiaPhase IIIActive Not RecruitingA Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich AtaxiaAug 23, 2022―
VatiquinoneFriedreich AtaxiaPhase IICompletedA Study of Vatiquinone for the Treatment of Participants With Friedreich AtaxiaAug 01, 2022―
Ptc518Huntington DiseasePhase IIActive Not RecruitingA Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)Apr 27, 2022―
Ptc857Amyotrophic Lateral SclerosisPhase IITerminatedStudy to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALSApr 21, 2022―
Dacarbazine, UnesbulinLeiomyosarcomaphase2AndPhase3TerminatedA Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)Feb 25, 2022―
Ptc923PhenylketonuriaPhase IIIActive Not RecruitingA Long-Term Safety Study of PTC923 in Participants With PhenylketonuriaDec 08, 2021―

FAQ

What are the FDA phases for drug approval?
The FDA approval process for new drugs is divided into several phases: a. Preclinical testing b. Phase 1 – Safety and dosage c. Phase 2 – Effectiveness and side effects d. Phase 3 – Confirmation of effectiveness, monitoring of adverse reactions, and comparison with existing treatments e. Phase 4 – Post-marketing studies to track long-term safety and effectiveness
What is a clinical trial?
A clinical trial is a carefully designed study in which researchers test a drug, treatment, or medical device in people to evaluate its safety, effectiveness, and potential side effects. These trials are essential for determining whether a new treatment should be approved for widespread use.
What drugs does PTC Therapeutics (PTCT) have in its pipeline
PTC Therapeutics PTCT is currently developing the following drugs: Ptc844, Vatiquinone, Sepiapterin, Ptc923, Sephience, Ptc518, Ptc857, Dacarbazine, Unesbulin, Ptc923, Eladocagene Exuparvovec, Ptc299, Soc, Ataluren, Deflazacort, Ptc299, Cnsa-001, Epi-589, Ptc596, Rifampin, Ataluren, Deflazacort And Rifampin, Deflazacort And Clarithromycin, Epi-743. These drug candidates are in various stages of clinical development as the company works toward FDA approval.