European Union committee recommends against Sarepta’s Elevidys

The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended not granting a marketing authorization for Sarepta’s (SRPT) Elevidys for the treatment of Duchenne muscular dystrophy. It stated, “The study failed to show that Elevidys had an effect on movement abilities after 12 months. Improvements in NSAA scores were observed both in patients who received Elevidys and in those who received placebo. The difference in the change in NSAA scores between the two groups was 0.65 on a 34-point scale and was not statistically significant, meaning that it may be due to chance. In addition, although many patients treated with Elevidys were shown to produce a shorter form of the dystrophin protein, the levels of dystrophin could not be linked to an improvement in movement abilities. The company also presented data for a sub-group of patients who seemed to respond better to Elevidys; however, even in this group, effectiveness of treatment was not demonstrated.”