PTC Therapeutics, Inc. (NASDAQ: PTCT) recently received a regulatory update regarding Translarna™ (ataluren) in Europe. The European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to not renew the authorization of Translarna™ for the treatment of nonsense mutation Duchenne muscular dystrophy. This decision effectively removes the drug’s conditional marketing authorization in the European Economic Area.
Matthew B. Klein, M.D., the Chief Executive Officer of PTC Therapeutics, expressed disappointment at the EC’s decision but noted that individual countries within the European Union can potentially leverage specific EU directives to allow continued use of Translarna.
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. It 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 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 to the structural stability of muscles.
As for PTC Therapeutics, Inc., it is a global biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines for rare disorders. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to maximize value for patients and other stakeholders.
While the European Commission's decision is disappointing, PTC Therapeutics, Inc. remains committed to working on a country-by-country basis to provide continued access to Translarna where possible. The market has reacted to these announcements by moving the company's shares -1.9% to a price of $53.93. Check out the company's full 8-K submission here.
