Sarepta Gains FDA Nod for ELEVIDYS in Duchenne Muscular Dystrophy for Ages 4+

Sarepta Therapeutics, Inc., a leader in precision genetic medicine for rare diseases, announced that the U.S. Food and Drug Administration (FDA) has expanded the labeled indication for ELEVIDYS (delandistrogene moxeparvovec-rokl) to treat Duchenne muscular dystrophy (DMD) in patients aged 4 and older with a confirmed DMD gene mutation. The FDA granted traditional approval for ambulatory patients, confirming the functional benefits, while non-ambulatory patients received accelerated approval. Continued approval for non-ambulatory patients may depend on verification of clinical benefits in a confirmatory trial. ELEVIDYS is contraindicated in patients with deletions in exon 8 and/or exon 9 of the DMD gene.

Doug Ingram, Sarepta's president and CEO, described the expanded indication as a significant milestone for the Duchenne community and gene therapy's promise. He credited the tireless efforts of Drs. Jerry Mendell and Louise Rodino-Klapac and thanked the FDA for expediting the therapy's delivery. He also acknowledged the contributions of clinical investigators and Duchenne families involved in the studies leading to this approval.

Jerry Mendell, M.D., co-inventor of ELEVIDYS and senior advisor in Medical Affairs at Sarepta, stated that the expansion represented the culmination of his 50-year effort to find a treatment for Duchenne patients. He emphasized that the expanded indication now provides a treatment option for most boys and young men with Duchenne, reflecting the success seen in studies.

Following the accelerated approval pathway, Sarepta has committed to a randomized, controlled trial to verify and confirm ELEVIDYS's clinical benefits in non-ambulatory DMD patients. ENVISION (Study SRP-9001-303), a global Phase 3 study, is underway and aims to fulfill this postmarketing requirement.

Sarepta is collaborating with Roche to transform the future for the Duchenne community. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., while Roche handles regulatory approvals and patient access outside the U.S.

ELEVIDYS is a single-dose, adeno-associated virus (AAV)-based gene therapy designed to address the genetic cause of DMD by delivering a transgene that produces ELEVIDYS micro-dystrophin in skeletal muscle. It is indicated for DMD patients aged 4 and older, both ambulatory and non-ambulatory, with confirmed DMD gene mutations. For non-ambulatory patients, the FDA's accelerated approval hinges on the expression of micro-dystrophin in skeletal muscle and may require confirmation of clinical benefits in a trial.

ELEVIDYS is contraindicated for patients with deletions in exon 8 and/or exon 9 of the DMD gene. Warnings include the risk of infusion-related reactions, acute serious liver injury, immune-mediated myositis, and myocarditis. Patients with preexisting liver conditions or deletion mutations involving exon 8 and/or exon 9 may be at higher risk for severe reactions. The therapy is not recommended for patients with elevated anti-AAVrh74 antibody titers.

Adverse reactions reported in clinical studies include vomiting, nausea, liver injury, fever, and thrombocytopenia. Sarepta advises monitoring patients for infusion-related reactions and liver function and recommends systemic corticosteroid treatment before and after infusion.

Sarepta is committed to advancing genetic medicine for rare diseases, holding leading positions in Duchenne and limb-girdle muscular dystrophies. The company has over 40 programs in development, driven by its Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing.

Sarepta will host an investor conference call on June 21, 2024, at 8:30 a.m. ET to discuss the latest updates. The event will be webcast live and archived for one year on Sarepta’s website. Interested participants can register online to receive dial-in details.

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