Pipeline — registered trials by phase
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
— · Muscular Dystrophy, Duchenne · Completed · est. 2025-03-31
Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort
— · Healthy Volunteers · Completed · est. 2014-12
A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
— · Duchenne Muscular Dystrophy · Completed · est. 2015-10
Effects of Rifampin on the Pharmacokinetics of Ataluren
— · Healthy · Completed · est. 2015-03
PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer
— · Metastatic Breast Cancer · Completed · est. 2012-03
Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
— · Healthy Volunteers · Completed · est. 2015-08
A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics
— · Renal Impairment · Completed · est. 2015-02
A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
— · Hepatic Impairment · Completed · est. 2015-02
A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)
— · Leiomyosarcoma · Completed · est. 2024-02-05
PTC596 in Patients With Advanced Solid Tumors
— · Cancer · Completed · est. 2017-02-06
An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
— · Duchenne Muscular Dystrophy · Completed · est. 2017-08-24
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
— · Friedreich Ataxia · Completed · est. 2024-08-29
Study of Ataluren in Participants With Nonsense Mutation Aniridia
— · Aniridia · Completed · est. 2021-01-22
Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
— · Leigh Syndrome · Completed · est. 2023-10-30
Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
— · Leigh Syndrome · Completed · est. 2015-02-28
Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
— · Friedreich's Ataxia · Completed · est. 2015-10-31
Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
— · Duchenne Muscular Dystrophy, Becker Muscular Dystrophy · Completed · est. 2009-12-31
Study of PTC844 in Participants With Active Rheumatoid Arthritis
— · Rheumatoid Arthritis · Not Yet Recruiting · est. 2027-09-30
Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)
— · Amyotrophic Lateral Sclerosis · Completed · est. 2018-02-23
PTC124 for the Treatment of Cystic Fibrosis
— · Cystic Fibrosis · Completed · est. 2007-07
A Study of CNSA-001 in Women With Diabetic Gastroparesis
— · Gastroparesis · Completed · est. 2019-09-08
Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
— · Duchenne Muscular Dystrophy · Completed · est. 2007-05
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
— · Duchenne Muscular Dystrophy · Completed · est. 2019-06-03
PTC124 for Cystic Fibrosis
— · Cystic Fibrosis · Completed · est. 2006-08
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
— · Duchenne Muscular Dystrophy · Completed · est. 2020-10-23
Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
— · Duchenne Muscular Dystrophy · Completed · est. 2018-02-09
A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
— · AADC Deficiency · Active Not Recruiting · est. 2023-05-22
A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
— · BH4 Deficiency, Hyperphenylalaninemia · Completed · est. 2020-10-02
Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis
— · Cystic Fibrosis · Completed · est. 2006-05-31
A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
— · Cystic Fibrosis · Completed · est. 2008-02-29
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
— · Nonsene Mutation Duchenne Muscular Dystrophy · Completed · est. 2023-08-07
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
— · Friedreich's Ataxia · Not Yet Recruiting · est. 2029-03-15
Extension Study of Ataluren (PTC124) in Cystic Fibrosis
— · Cystic Fibrosis · Completed · est. 2013-12-02
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
— · Friedreich Ataxia · Completed · est. 2023-04-04
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
— · Friedreich Ataxia · Active Not Recruiting · est. 2027-12-31
Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
— · Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic · Completed · est. 2015-08-20
A Study of PTC923 in Participants With Phenylketonuria
— · Phenylketonuria · Completed · est. 2023-04-03
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
— · Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy · Completed · est. 2018-01-19
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
— · Phenylketonuria · Recruiting · est. 2031-02-28
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
— · Phenylketonuria · Completed · est. 2026-07-21
Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
— · Cystic Fibrosis · Completed · est. 2016-11
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
— · Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic · Completed · est. 2022-03-05
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
— · Inherited Mitochondrial Disease · Completed · est. 2025-04-15
Study of Ataluren (PTC124™) in Cystic Fibrosis
— · Cystic Fibrosis · Completed · est. 2011-11-12
No approved products listed under this sponsor in Drugs@FDA (partnered or subsidiary products may be listed elsewhere).
Source: ClinicalTrials.gov (trials where PTC Therapeutics is lead sponsor; 44 active or completed) and Drugs@FDA (0 approved products). Registry data can include older or partnered programs; the company's own pipeline page is the official list.
SEC filings — insider trading forms excluded
- 8-K2026-09-21Current report (material event)
- 8-K2026-08-28Current report (material event)
- 8-K2026-08-13Current report (material event)
- 10-Q2026-07-30Quarterly report
- 8-K2026-07-30Current report (material event)
- 8-K2026-06-18Current report (material event)
- 8-K2026-06-16Current report (material event)
- 8-K2026-06-09Current report (material event)
- 8-K2026-06-05Current report (material event)
- SCHEDULE 13G/A2026-05-15SCHEDULE 13G/A
- SCHEDULE 13G/A2026-05-15SCHEDULE 13G/A
- 10-Q2026-05-07Quarterly report
- 8-K2026-05-07Current report (material event)
- SCHEDULE 13G2026-04-30SCHEDULE 13G
- SCHEDULE 13G2026-04-29SCHEDULE 13G
- 8-K2026-04-28Current report (material event)
- ARS2026-04-20Annual report to holders
- DEFA14A2026-04-20DEFA14A
- DEF 14A2026-04-20Proxy statement
- SCHEDULE 13G/A2026-03-27SCHEDULE 13G/A
- 8-K2026-03-25Current report (material event)
- 10-K2026-02-19Annual report
- 8-K2026-02-19Current report (material event)
- SCHEDULE 13G2026-02-17SCHEDULE 13G
- SCHEDULE 13G2026-02-13SCHEDULE 13G
- 8-K2026-02-12Current report (material event)
- SCHEDULE 13G/A2026-02-10SCHEDULE 13G/A
- SCHEDULE 13G2026-02-09SCHEDULE 13G
- 8-K2026-01-12Current report (material event)
- 8-K2025-12-29Current report (material event)
- SCHEDULE 13G/A2025-12-04SCHEDULE 13G/A
- SCHEDULE 13G/A2025-11-14SCHEDULE 13G/A
- 10-Q2025-11-04Quarterly report
- 8-K2025-11-04Current report (material event)
- SCHEDULE 13G/A2025-10-30SCHEDULE 13G/A
- UPLOAD2025-09-24UPLOAD
- CORRESP2025-09-18CORRESP
- UPLOAD2025-08-21UPLOAD
- 8-K2025-08-19Current report (material event)
- SCHEDULE 13G/A2025-08-14SCHEDULE 13G/A
Source: SEC EDGAR. Forms 3/4/5 and 144 (insider transactions) are intentionally left out.