Press release
Sarepta Therapeutics Acknowledges CHMP Negative Opinion for ELEVIDYS in the European Union
Partner Roche will continue its dialogue with the European Medicines Agency to explore a potential path forward to make ELEVIDYS available to individuals
About this update from Sarepta Therapeutics, Inc.
[{"type":"text","content":"\n\nPartner Roche will continue its dialogue with the European Medicines Agency to explore a potential path forward to make ELEVIDYS available to individuals living with Duchenne muscular dystrophy in the EU\n\n\n\nELEVIDYS is the first and only disease-modifying gene therapy for Duchenne\n\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nSarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, acknowledges that the Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion on the conditional marketing authorization (CMA) for ELEVIDYS (delandistrogene moxeparvovec) in ambulatory individuals ages three to seven years for the treatment of Duchenne muscular dystrophy (DMD).\n\n\n“While we are disappointed by the CHMP’s negative opinion, we understand the urgent need for continued dialogue and collaboration to bring transformative therapies to people with Duchenne who live with a relentless disease that steals their mobility, independence and ultimately life – often by early adulthood,\" said Louise Rodino-Klapac, Ph.D., president of research & development and technical operations, Sarepta.\n\n\n“Following the initial FDA approval of ELEVIDYS on June 22, 2023, the therapy has subsequently received regulatory approval in several other countries. In the U.S., we are actively working with the FDA to address recent safety questions. We remain committed to working with regulators to address outstanding questions on safety so that people living with Duchenne have access to this important therapy.”\n\n\nELEVIDYS is the first and only approved gene therapy targeting the underlying cause of disease that has consistently demonstrated stabilization or slowing of DMD disease progression, with durable effects on functional and biological outcomes and muscle health.\n\n\nWhile the primary endpoint was not met in EMBARK after one year, ELEVIDYS showed clinically meaningful and statistically significant improvements across important secondary endpoints of functional outcome measures when compared to placebo. Longer term efficacy data were also submitted to EMA, including two-year results from the EMBARK study and three-year pooled efficacy analysis from three other ELEVIDYS studies that showed clinically meaningful improvements across key measures of motor function. One-year data from part...