FDA expands gene therapy use in Duchenne muscular dystrophy

FDA headquarters

The US Food and Drug Administration (FDA) has expanded its approval 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 four 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 four 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.

ENVISION, a Phase III study of Elevidys in non-ambulatory and older ambulatory individuals with Duchenne, is underway to confirm the clinical benefit of Elevidys in patients with Duchenne muscular dystrophy who are non-ambulatory.

Elevidys is part of a collaboration agreement between Sarepta and Roche. Sarepta is responsible for regulatory approval and commercialisation in the US, while Roche is responsible for the rest of the world.

“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 optimise and develop a gene therapy that could be safely and effectively delivered to muscle,” said Jerry Mendell, co-inventor of ELEVIDYS and Senior Advisor, Medical Affairs, Sarepta.

Diana Spencer, Senior Digital Content Editor, DDW

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