PTC Therapeutics
- Country
- 🇺🇸United States
- Ownership
- Public
- Established
- 1998-01-01
- Employees
- 1K
- Market Cap
- $2.7B
- Website
- http://www.ptcbio.com
- Introduction
PTC Therapeutics, Inc. is a biopharmaceutical company, which focuses on the discovery, development, and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. The company was founded by Allan Steven Jacobson and Stuart Walter Peltz on March 31, 1998 and is headquartered in Warren, NJ.
Clinical Trials
225
Trial Phases
3 Phases
Drug Approvals
1
Drug Approvals
Clinical Trials
Distribution across different clinical trial phases (184 trials with phase data)• Click on a phase to view related trials
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
- First Posted Date
- 2024-03-08
- Last Posted Date
- 2025-06-05
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 56
- Registration Number
- NCT06302348
- Locations
- 🇺🇸
Indiana University, Indianapolis, Indiana, United States
An Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
- First Posted Date
- 2024-02-12
- Last Posted Date
- 2025-03-11
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 250
- Registration Number
- NCT06254482
- Locations
- 🇦🇺
Monash Health, Clayton, Australia
🇦🇺Westmead Hospital, Westmead, Australia
🇦🇹Medical University Innsbruck, Innsbruck, Austria
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
- First Posted Date
- 2022-08-25
- Last Posted Date
- 2025-06-06
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 130
- Registration Number
- NCT05515536
- Locations
- 🇺🇸
UCLA, Los Angeles, California, United States
🇺🇸University of Florida, Gainesville, Florida, United States
🇺🇸University of South Florida, Tampa, Florida, United States
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
- First Posted Date
- 2022-08-03
- Last Posted Date
- 2024-10-08
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 5
- Registration Number
- NCT05485987
- Locations
- 🇺🇸
The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
- First Posted Date
- 2022-05-03
- Last Posted Date
- 2025-02-20
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 252
- Registration Number
- NCT05358717
- Locations
- 🇩🇪
Charite University Medicine Berlin, Berlin, Germany
🇺🇸University of California, San Diego, La Jolla, California, United States
🇺🇸Vanderbilt University Medical Center, Nashville, Tennessee, United States
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News
uniQure Appoints Kylie O'Keefe as Chief Customer and Strategy Officer to Lead AMT-130 Commercialization for Huntington's Disease
uniQure has appointed Kylie O'Keefe as Chief Customer and Strategy Officer to lead the global commercialization strategy for AMT-130, the company's investigational gene therapy for Huntington's disease.
Huntington's Disease Pipeline Shows Robust Growth with 20+ Companies Developing Novel Therapies
DelveInsight's 2025 pipeline analysis reveals over 20 active companies developing more than 20 pipeline drugs for Huntington's disease, including gene-targeting and disease-modifying therapies.
PTC518 Shows Promise in Huntington's Disease: Phase 2 Trial Meets Primary Endpoint
PTC Therapeutics' investigational drug PTC518 demonstrated statistically significant reductions in Huntingtin protein levels in Phase 2 PIVOT-HD study, with 23-39% decreases observed across different dosages over 12 months.
PTC Therapeutics' Sephience Receives Positive CHMP Opinion for Phenylketonuria Treatment Across All Age Groups
The European Medicines Agency's CHMP has issued a positive opinion for Sephience (sepiapterin) to treat phenylketonuria in patients of all ages and disease severities, addressing a significant unmet medical need.
FDA Sets Q3 2025 Approval Dates for Three Breakthrough Therapies Targeting Rare Diseases
The FDA has established PDUFA target action dates for three promising therapies in Q3 2025: sepiapterin for phenylketonuria (July 29), apitegromab for spinal muscular atrophy (September 22), and paltusotine for acromegaly (September 25).
Phenylketonuria Pipeline Shows Robust Growth with 25+ Therapies in Development
DelveInsight's latest report reveals a robust phenylketonuria (PKU) pipeline with over 20 companies developing 25+ therapeutic candidates, signaling significant advancement in treatment options for this rare metabolic disorder.
European Commission Withdraws Translarna's Marketing Authorization for Duchenne Muscular Dystrophy
The European Commission has adopted the CHMP's negative opinion, withdrawing conditional marketing authorization for PTC Therapeutics' Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy.
FDA Priority Review Vouchers Face Transparency Challenges Amid Rising Prices
Priority Review Vouchers (PRVs) have surged in value to $150-158 million in recent months, up from the typical $100 million, following Congress's failure to reauthorize the rare pediatric disease program.
First Brain-Directed Gene Therapy Upstaza Receives EU Recommendation for Rare AADC Deficiency
PTC Therapeutics' Upstaza has secured CHMP recommendation as the first disease-modifying treatment for AADC deficiency, marking a milestone as the first gene therapy administered directly to the brain.
DMD Market Set to More Than Double to $5.2 Billion by 2033, Driven by Novel Therapies
The Duchenne muscular dystrophy market across seven major markets is projected to grow from $2.3 billion in 2023 to $5.2 billion by 2033, fueled by recent approvals of Elevidys and Agamree.