FDA approves first gene therapy for Duchenne muscular dystrophy

Approval stamp

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US.

The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD.

It is approved in ambulatory paediatric patients aged four through five years with DMD with a confirmed mutation in the DMD gene based on expression of Elevidys micro-dystrophin observed in patients treated with Elevidys. It is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

The accelerated approval follows a vote of support from the FDA Cellular, Tissue and Gene Therapies Advisory Committee and the granting of priority review status.

Elevidys addresses the root genetic cause of Duchenne – mutations in the dystrophin gene that result in the lack of dystrophin protein – by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as Elevidys micro-dystrophin.

The FDA’s accelerated approval is based on an increase in Elevidys micro-dystrophin protein expression in skeletal muscle.

“Duchenne is a relentlessly progressive, degenerative disease, robbing children of muscle function,” said Jerry Mendell, Paediatric Neurologist and principal investigator in the Center for Gene Therapy at Nationwide Children’s Hospital. “The increases in Elevidys dystrophin expression and the functional results that we see can make a difference in the lives of our patients.”

Phase III EMBARK trial

Consistent with the accelerated approval pathway, the company has committed to the completion of a confirmatory trial. EMBARK, the global, randomised, double-blind, placebo-controlled Phase III trial for Elevidys, will serve as the post-marketing confirmatory trial and is fully enrolled with top-line results expected in late 2023.

“The approval of Elevidys is a watershed moment for the treatment of Duchenne,” said Doug Ingram, President and Chief Executive Officer of Sarepta. “If EMBARK confirms the benefits seen in our prior trials, Sarepta will move rapidly to submit a BLA supplement to expand the approved label as broadly as good science permits.”

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