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Sarepta Therapeutics: Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above

June 21, 2024

Defeat Duchenne Canada is sharing the news that Sarepta Therapeutics announced expanded US FDA approval of ELEVIDYS to Duchenne muscular dystrophy patients ages of 4 years and older.

The 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.

“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.”

“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.”

Doug Ingram, President and Chief Executive Officer of Sarepta

“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.”

Jerry Mendell, M.D., Co-Inventor of ELEVIDYS and Senior Advisor, Medical Affairs with Sarepta

Defeat Duchenne Canada previously funded the work of both Dr. Jerry Mendell and Dr. Louise Rodino-Klapac.

The FDA also released a press release to the community communicating their decision.

“In granting accelerated approval for non-ambulatory individuals aged 4 and older, the FDA considered the totality of the evidence, including clinical data in ambulatory individuals from a study in 4- to 7-year-olds, as well as from a study in 4- to 5-year-olds indicating a correlation of Elevidys micro-dystrophin levels with clinical outcome measures.”

“Based on the evidence and given that the mechanism of action of Elevidys is similar for ambulatory and non-ambulatory populations, the FDA determined that increased levels in micro-dystrophin is reasonably likely to predict clinical benefit in the non-ambulatory population.”

“This conclusion, along with the evidence that Elevidys elevates micro-dystrophin levels, provides substantial evidence of effectiveness to support accelerated approval in non-ambulatory individuals at least 4 years of age with DMD considering the serious nature of the disease and the extent of unmet medical need in this group of individuals. A confirmatory randomized, controlled clinical trial in the non-ambulatory population is currently underway.”

The United States Food and Drug Administration

Sarepta is working with Roche to transform the future for the Duchenne community, enabling those living with the disease to maintain and protect their muscle function. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing. Roche is responsible for regulatory approvals and bringing ELEVIDYS to patients across the rest of the world.

Defeat Duchenne Canada will continue to follow this most recent development and will continue its work with Roche Canada to gain access to treatments for the Canadian Duchenne community. Read both the Sarepta and FDA press releases below.

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