EMA Rejects Marketing Authorization for Sarepta’s DMD AAV Gene Therapy

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended against granting marketing authorization for Sarepta Therapeutics’ AAV gene therapy, delandistrogene moxeparvovec (Elevidys), for Duchenne muscular dystrophy (DMD). The decision was made at its July 2025 meeting.

DMD is a genetic disorder where patients lack normal dystrophin, leading to progressive muscle weakness. Elevidys is a one-time, adeno-associated virus-based gene therapy designed to introduce genetic material to produce a truncated version of dystrophin, aiming to slow disease progression in ambulatory DMD patients aged 4 and older with a confirmed DMD gene mutation.

The EMA based its negative recommendation on a study that failed to demonstrate a statistically significant effect on movement abilities after 12 months. In the study involving 125 children aged 4 to 7, the difference in improvement on the North Star Ambulatory Assessment (NSAA) score between those receiving Elevidys and placebo was only 0.65, which was not statistically significant. The EMA also noted that while many patients produced a shorter form of dystrophin, these levels could not be linked to improved movement abilities.

Furthermore, the decision comes amidst safety concerns, including two recent deaths from acute liver failure in patients treated with Elevidys. A 16-year-old boy died in March after receiving treatment in December, and a 15-year-old patient also died in June from acute liver failure. Following the first death, Sarepta temporarily halted clinical studies of Elevidys in the EU at the EMA’s request in April. Earlier this month, the FDA in the US asked Sarepta to add a “black-box” warning regarding the risk of acute liver injury and liver failure for ambulatory DMD patients.

Sarepta Therapeutics has the option to request a re-examination of the EMA’s opinion within 15 days.