By Fineline Cube 
Swiss giant Roche AG (SWX: ROG, OTCMKTS: RHHBY) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a negative opinion, recommending against conditional marketing authorization for Elevidys (delandistrogene moxeparvovec) for the treatment of ambulatory Duchenne muscular dystrophy (DMD) patients aged three to seven years.
CHMP Opinion Basis
TheMP CH’s decision is based on data from the largest and most comprehensive DMD gene therapy clinical program to date, including results from the pivotal Phase III EMBARK study. Although Elevidys showed sustained disease stabilization or slowed progression in ambulatory patients and demonstrated a manageable safety profile, the primary endpoint was not met after one year of treatment.
Clinical Trial Results
Despite the primary endpoint not being met, Elevidys demonstrated clinically meaningful and statistically significant improvements across important secondary endpoints of functional outcome measures compared to placebo. Over 900 individuals with DMD, including 760 ambulatory patients, have been treated with Elevidys in both clinical and real-world settings.
Elevidys Overview
Elevidys (delandistrogene moxeparvovec, also known as SRP-9001) is the first approved disease-modifying gene therapy for Duchenne. It targets the root cause of the disease by promoting the production of a shortened dystrophin protein in skeletal, respiratory, and cardiac muscles. Administered as a one-time intravenous treatment, Elevidys is contraindicated in individuals with deletions in exons 8 and/or 9 of the DMD gene.-Fineline Info & Tech