Approval for the first gene therapy for Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc. has announced the US Food and Drug Administration (FDA) is accelerating the approval of ELEVIDYS (delandistrogene moxeparvovec-rokl), an Adeno-associated virus (AAV)-based gene therapy for the outpatient treatment of children aged 4 to 5 years with Duchenne muscular dystrophy (DMD) with mutations confirmed in the DMD gene.

This indication was approved with accelerated approval based on the expression of the ELEVIDYS micro-dystrophin observed in patients treated with ELEVIDYS. Further approval for this indication may depend on verification and description of clinical benefit in confirmatory trials. ELEVIDYS is contraindicated in patients with exon 8 and/or exon 9 deletions in the DMD gene.

ELEVIDYS addresses the genetic root cause of Duchenne – a mutation in the dystrophin gene resulting in a deficiency of the dystrophin protein – by delivering a gene that codes for a short form of dystrophin to muscle cells known as the ELEVIDYS micro-dystrophin.

This accelerated approval is based on increased expression of the ELEVIDYS micro-dystrophin protein in skeletal muscle. ELEVIDYS is supported by biological and empirical evidence, in addition to efficacy data from two clinical studies: SRP-9001-102 and SRP-9001-103 as well as safety data from SRP-9001-101, SRP-9001-102 and SRP-9001-103.

Acute serious liver injury, immune-mediated myositis and myocarditis have occurred in patients treated with ELEVIDYS. The most common adverse reactions in clinical studies were vomiting, nausea, increased liver function tests, pyrexia and thrombocytopenia.

Consistent with the expedited approval pathway, the company has committed to complete the confirmation trial. EMBARK, the global, randomized, double-blind, placebo-controlled phase 3 trial for ELEVIDYS, will serve as a post-marketing confirmation trial and is fully registered with best results expected in late 2023.

Sarepta Therapeutics makes a difference

“Duchenne is a relentlessly progressive degenerative disease, robbing children of muscle function,” said Jerry Mendell, pediatric neurologist and principal investigator in the Center for Gene Therapy at Children’s Hospital Nationwide.

“The increased expression of ELEVIDYS dystrophin and the functional outcomes we are seeing can make a difference in the lives of our patients.”

“The ELEVIDYS approval is a watershed moment for Duchenne’s care. ELEVIDYS is the first and only gene therapy approved for Duchenne, and this approval brings us closer to our goal of advancing a treatment that has the potential to change the trajectory of this degenerative disease,” said Doug Ingram, president and chief executive, Sarepta Therapy.

“As we prepare to launch ELEVIDYS, we must acknowledge and celebrate decades of dedication and hard work from our community of patients, families, doctors and Sarepta colleagues that led to today’s approval. Our confirmation trial, Embark, should be read out in the fourth quarter of this year. If Embark confirms the benefits seen in our previous trial, Sarepta will move quickly to ship BLA supplements to expand the approved label as widely as good science allows.

“Today’s decision marks an important moment in gene therapy for patients living with Duchenne,” said Pat Furlong, founding president and chief executive officer, Parent Project Muscular Dystrophy.

“This is the lifelong work of so many people in the Duchenne community. Our work continues until all patients in our community have access to therapy.”

Last year, Sarepta Therapeutics, announced it had submitted a Biological Licensing Application for expedited approval for SRP-9001 (delandistrogene moxeparvovec) to treat ambulance individuals with Duchenne muscular dystrophy.

SRP-9001 is an investigative gene therapy for Duchenne developed in partnership with Roche.

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