Translarna by PTC Therapeutics: Awaiting Another EU Regulatory Review for Muscular Dystrophy Treatment

1. PTC Therapeutics' drug Translarna (ataluren) is designed to treat Duchenne muscular dystrophy (DMD), a rare genetic disorder characterized by progressive muscle degeneration and weakness.
2. Translarna has been granted conditional marketing authorization by the European Medicines Agency (EMA) for the treatment of DMD in ambulatory patients aged five years and older.
3. The drug is designed to enable the formation of a functioning protein in patients with genetic mutations known as "nonsense mutations," which account for around 10-15% of DMD cases.
4. PTC Therapeutics is awaiting another review of Translarna by the EMA, following a previous review in 2017 that resulted in the conditional marketing authorization being maintained.
5. The upcoming review will assess the efficacy, safety, and overall benefit-risk profile of Translarna, based on additional data and evidence gathered since the previous review.
6. If the EMA's Committee for Medicinal Products for Human Use (CHMP) provides a positive opinion, Translarna's conditional marketing authorization will be confirmed, allowing continued access to the drug for eligible DMD patients in the EU.
7. Translarna is currently available in over 30 countries, and PTC Therapeutics is working to expand its availability to more patients with DMD worldwide.