The European Medicines Agency (EMA) has recommended against approving Sarepta’s gene therapy, Elevidys, for Duchenne muscular dystrophy (DMD) after a key clinical trial failed to show meaningful benefits. The decision comes after the EMBARK trial did not meet its main goal of improving movement abilities in patients over 12 months. Sarepta accepted the decision, with Dr. Louise Rodino-Klapac stating that while they are disappointed, they remain committed to finding treatments for DMD, a severe disease that progresses quickly and often shortens life expectancy. Roche, which holds the rights to Elevidys outside the U.S., plans to work with the EMA to explore future options. Roche’s Dr. Levi Garraway emphasized the urgent need for treatments for DMD, which significantly reduces life expectancy. While the trial did not meet its primary goal, some secondary measures showed improvements in function, though the EMA noted that increased dystrophin protein levels did not translate to better movement. Sarepta recently paused U.S. shipments of Elevidys following FDA concerns after three patient deaths linked to its gene therapies. Analysts say these setbacks highlight the challenges in developing safe and effective gene therapies, stressing the need for transparency and patient safety. The gene therapy market is projected to grow significantly in the coming years. This news is part of broader coverage on cell and gene therapies, supported by Cytiva, with content produced independently to maintain journalistic integrity.