Sarepta’s Elevidys secures US FDA expansion for Duchenne Muscular Dystrophy

USA – Sarepta Therapeutics, Inc. has announced that the US FDA has expanded the labeled indication for its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys, to include individuals with DMD who are at least 4 years of age and have a confirmed mutation in the DMD gene.

The FDA granted traditional approval for Elevidys in ambulatory DMD patients and accelerated approval for non-ambulatory patients.

However, continued approval for the non-ambulatory population may be contingent upon verification of clinical benefit in a confirmatory trial.

Doug Ingram, president and chief executive officer of Sarepta, emphasized that the expansion of the Elevidys label to treat Duchenne patients aged 4 and above, regardless of ambulatory status, represented many years of dedicated research, development, investment, and creative energy.

He described today as a defining moment for the Duchenne community and a watershed occasion for the promise of gene therapy, emphasizing it as a win for science.

The label expansion comes nearly a year after Elevidys received its initial accelerated approval for DMD patients aged 4 to 5 years.

Sarepta is now conducting the Phase III ENVISION study (NCT05881408) to evaluate Elevidys in non-ambulatory and older ambulatory DMD patients, which will serve as the post-marketing confirmatory trial.

Jerry Mendell, M.D., senior advisor of Medical Affairs at Sarepta, stated that today’s expansion of the Elevidys label represented the culmination of his 50-year pursuit of a treatment for Duchenne patients.

He added that it also marked a nearly 20-year effort, alongside his colleague Dr. Louise Rodino-Klapac, to optimize and develop a gene therapy that could be safely and effectively delivered to muscle.

Elevidys is designed to use a recombinant adeno-associated virus vector serotype 74 (AAV74) to deliver a microdystrophin transgene that encodes a dystrophin protein.

The therapy is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

A GlobalData consensus estimates Elevidys to generate $5.7 billion in sales by 2029. The expanded label comes as a boost for Sarepta, following the recent failure of Pfizer’s competing mini-dystrophin gene therapy for DMD in a Phase III trial. 

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