Translarna approval for DMD in EU not recommended for renewal | Therapy developer PTC to seek new review by EMA committee | Muscular Dystrophy News

The EMA's CHMP recommended against renewing Translarna's conditional approval for Duchenne muscular dystrophy (DMD) treatment, citing insufficient evidence of efficacy in a key patient group. PTC Therapeutics plans to request a re-examination, emphasizing Translarna's benefits. Translarna remains available in the EU pending review, with a final decision expected by January 2024.