PTC Therapeutics, Inc. (NASDAQ: PTCT) has recently received a negative opinion from the Committee for Medicinal Products for Human Use (CHMP) for the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmdMD) in ambulatory patients aged 2 years and older. This negative opinion was issued following the European Commission's request for a review. Despite the negative opinion, the marketing authorization for Translarna remains in effect pending the outcome of the re-examination procedure and subsequent EC ratification.
Matthew B. Klein, M.D., the Chief Executive Officer of PTC Therapeutics, expressed disappointment at the CHMP's decision, noting that the scientific advisory group convened as part of the recent review procedure concluded that the evidence of significant efficacy provided by the Stride real-world evidence "should not be ignored." PTC Therapeutics plans to request re-examination of the CHMP opinion to maintain the availability of Translarna on the market for as long as possible for boys and young men with nmdMD.
Translarna™ (ataluren) is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. The treatment is licensed in multiple countries for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged 2 years and older.
Duchenne muscular dystrophy (Duchenne) is a rare and fatal genetic disorder primarily affecting males, resulting in progressive muscle weakness from early childhood and leading to premature death in the mid-20s due to heart and respiratory failure. It is caused by the lack of functional dystrophin protein, critical for the structural stability of muscles.
As of June 28, 2024, PTC Therapeutics expects Translarna to remain on the market through the end of 2024, even if the negative opinion is maintained and ratified.
PTC Therapeutics, Inc. is a global biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines for patients with rare disorders. The company's mission is to provide access to best-in-class treatments for patients who have little to no treatment options.
For more information about PTC Therapeutics, visit www.ptcbio.com or follow the company on social media platforms like Facebook, Instagram, LinkedIn, and Twitter at @ptcbio. The market has reacted to these announcements by moving the company's shares -13.0% to a price of $30.17. For the full picture, make sure to review PTC Therapeutics's 8-K report.
