PTC Therapeutics

PTC Therapeutics logo
🇺🇸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.

Positive Pipeline Updates for Friedreich's Ataxia

PTC Therapeutics reported promising long-term data for vatiquinone, showing significant reduction in Friedreich's ataxia progression. The drug hit key endpoints in MOVE-FA studies, indicating a 3.7-point benefit on mFARS at 144 weeks. PTC plans to submit an NDA to the FDA by December 2024, aiming for vatiquinone's approval as a treatment option for FRDA.

EMA agency continues to oppose renewing Translarna's approval

The CHMP maintained its recommendation not to renew Translarna's conditional marketing authorization for treating nonsense mutation Duchenne muscular dystrophy, despite PTC Therapeutics' commitment to keep it available. The European Commission is expected to make a final decision within two months.
hdbuzz.net
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Bringing HD Treatments to Market: The Role of Regulatory Oversight

The article discusses the complexities of drug regulation, focusing on the FDA and EMA, and how drugs move from lab testing to market approval. It highlights the different approval processes in the US and EU, emphasizing the importance of robust clinical trial data for successful approval. The article also touches on the reasons why clinical trials are held in specific countries and the potential for different regulatory decisions between the EMA and FDA.
biopharmadive.com
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PTC rebuffed again by EMA; Novartis licenses China-based biotech's cancer drug

EMA won't renew Translarna's permit; Novartis pays $70M for a cancer drug; Amylyx's AMX0035 shows promise in rare disease; FDA delays Ocaliva approval; Australia rejects Leqembi; Amicus settles with Teva over Galafold.
kilgorenewsherald.com
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PTC Therapeutics Announces FDA Target Regulatory Action Date for Sepiapterin and ...

PTC Therapeutics announced the FDA's target action date of July 29, 2025, for the review of sepiapterin's NDA for treating PKU in pediatric and adult patients. The Phase 3 APHENITY trial results were published in The Lancet, highlighting sepiapterin's potential to address PKU's unmet medical needs.
medcitynews.com
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Scholar Rock Scores $300M for Regulatory Filings & Launch Plans for Neuromuscular Disease Drug

Scholar Rock's experimental drug apitegromab, designed to block myostatin activation, showed significant muscle function improvement in SMA patients in a Phase 3 study, leading to a $300 million stock offering. The company plans FDA and EMA submissions for 2025 commercial launch, and is exploring apitegromab's use in preserving muscle mass during weight loss treatment.
drugs.com
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PTC Therapeutics Announces Positive Results from Long-Term Treatment Studies and Updates on Regulatory Progress for Vatiquinone Friedreich Ataxia Program

PTC Therapeutics reports positive long-term results for vatiquinone in Friedreich ataxia, with significant slowing of disease progression and a strong safety profile. The company plans to submit an NDA for vatiquinone by the end of 2024.
prnewswire.com
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PTC Therapeutics Announces FDA Acceptance for Filing of NDA for Sepiapterin for ...

PTC Therapeutics announced FDA acceptance of sepiapterin's NDA for PKU treatment, with potential to reduce Phe levels and enable diet liberalization. The NDA is supported by phase 3 APHENITY trial results showing significant Phe reduction and Phe control in PKU patients.
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