Press release
Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above
– FDA grants traditional approval to ELEVIDYS for ambulatory Duchenne patients – FDA grants accelerated approval to ELEVIDYS for non-ambulatory Duchenne
About this update from Sarepta Therapeutics, Inc.
[{"type":"text","content":"\n– FDA grants traditional approval to ELEVIDYS for ambulatory Duchenne patients\n\n\n– FDA grants accelerated approval to ELEVIDYS for non-ambulatory Duchenne patients\n\n\n– Sarepta will host an investor conference call on June 21, 2024, at 8:30 a.m. ET\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nSarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced U.S. Food and Drug Administration (FDA) approval of an expansion to the labeled indication for ELEVIDYS (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least 4 years of age. Confirming the functional benefits, the FDA granted traditional approval for ambulatory patients. The FDA granted accelerated approval for non-ambulatory patients. Continued approval for non-ambulatory Duchenne patients may be contingent upon verification of clinical benefit in a confirmatory trial. ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.\n\n\n“Representing many years of dedicated research, development, investment and creative energy, the expansion of the ELEVIDYS label to treat Duchenne patients aged 4 and above, regardless of ambulatory status, is a defining moment for the Duchenne community. Today also stands as a watershed occasion for the promise of gene therapy and a win for science,” said Doug Ingram, president and chief executive officer, Sarepta. “At this pivotal moment, I want to give warm thanks to Drs. Jerry Mendell and Louise Rodino-Klapac for their dogged, 20-year pursuit of a gene therapy to treat this ruthless and life-robbing disease, to the FDA for following the scientific evidence to speed delivery of a therapy for a life-threatening rare disease to waiting patients, and to the many clinical investigators and courageous Duchenne families who have participated in the multiple studies that led to this important day.”\n\n\n“Today’s expansion of the ELEVIDYS label represents the culmination of my 50-year pursuit of a treatment for Duchenne patients and, along with my colleague Dr. Louise Rodino-Klapac, a nearly 20-year effort to optimize and develop a gene therapy that could be safely and effectively delivered to muscle,” said Jerry Mendell, M.D., co-inventor of ELEVIDYS and ...