The European Medicines Agency (EMA) has denied marketing authorization for Elevidys, a gene therapy treatment for Duchenne muscular dystrophy (DMD). The treatment, developed by Sarepta Therapeutics, aims to produce a truncated version of the dystrophin protein to slow down disease progression. However, a recent study failed to show that Elevidys had an effect on movement abilities after 12 months.
The study involved 125 children with DMD who received either delandistrogene moxeparvovec or a placebo. While patients treated with the drug produced a shorter form of dystrophin protein, the levels could not be linked to improved movement abilities. The EMA stated that improvements in movement abilities were observed in both treatment groups, but the difference was too small to be statistically significant.
Tragically, two patients who received Elevidys have died from acute liver failure, prompting the company to temporarily halt clinical studies. The FDA has also requested a “black-box” warning for the risk of acute liver injury and liver failure in patients with DMD who can walk.
Sarepta Therapeutics may re-examine its application for marketing authorization within 15 days after receiving the EMA’s opinion.
Source: https://www.medscape.com/viewarticle/ema-says-no-duchenne-gene-therapyelevidys-2025a1000jqs