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Sarepta Therapeutics Announces Positive Update on ELEVIDYS (delandistrogene moxeparvovec-rokl) Regulatory Progress for Duchenne Muscular Dystrophy (DMD)

You are here: Home / News / Gene therapy / Sarepta Therapeutics Announces Positive Update on ELEVIDYS (delandistrogene moxeparvovec-rokl) Regulatory Progress for Duchenne Muscular Dystrophy (DMD)
Sarepta Therapeutics Announces Positive Update on ELEVIDYS (delandistrogene moxeparvovec-rokl) Regulatory Progress for Duchenne Muscular Dystrophy (DMD)

June 21, 2024 by John Marrin

Sarepta Therapeutics, Inc. today provided an update on the regulatory progress of ELEVIDYS (delandistrogene moxeparvovec-rokl), its gene therapy for DMD.

FDA Approves Expanded Label for ELEVIDYS

The U.S. Food and Drug Administration (FDA) has approved an expansion of the labelled indication for ELEVIDYS to include patients aged 4 and above who have a confirmed mutation in the DMD gene. This is a significant step forward in making ELEVIDYS available to a broader range of DMD patients.

Representing many years of dedicated research, development, investment and creative energy, the expansion of the ELEVIDYS label to treat Duchenne patients aged 4 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.

Doug Ingram, President and Chief Executive Officer, Sarepta

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. The initial approval of ELEVIDYS was a significant milestone, and the expanded indication means clinicians now have a treatment option for the great majority of boys and young men living with Duchenne. This expansion speaks to the success of the science, the evidence and the improvements in the trajectory of the disease we have seen to date across studies.

Jerry Mendell, M.D., co-inventor of ELEVIDYS and Senior Adviser, Serepta

Traditional Approval for Ambulatory Patients, Accelerated Approval for Non-Ambulatory Patients

The FDA granted traditional approval for ELEVIDYS in ambulatory DMD patients, confirming the treatment’s functional benefits. For non-ambulatory patients, the FDA granted accelerated approval. Sarepta remains committed to providing further data on the long-term benefits of ELEVIDYS in this population through the ongoing ENVISION study (Study SRP-9001-303).

Traditional: Think “gold standard” – There is a higher bar for evidence, requiring strong statistical evidence from large, well-controlled clinical trials demonstrating effectiveness and safety over an extended period (years). Once this label is granted the treatment is considered fully approved by the FDA.

Accelerated: May be based on a “surrogate endpoint,” a measurable factor that is reasonably likely to predict a clinical benefit. This allows for faster approval, potentially years earlier than traditional methods. Once this label is granted, the treatment is conditionally approved and further studies are required to confirm the surrogate endpoints. 

ENVISION Study Continues to Evaluate ELEVIDYS in Non-Ambulatory Patients

Sarepta is actively conducting the ENVISION study, a global, randomised, double-blind, placebo-controlled Phase 3 study (ENVISION, Study SRP-9001-303) to confirm the clinical benefit of ELEVIDYS in non-ambulatory patients. This study is intended to fulfil the post-marketing requirement associated with the accelerated approval for this group.

ELEVIDYS (delandistrogene moxeparvovec-rokl)

ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

Read More

Would you like to know more about Duchenne muscular dystrophy? Increase your knowledge and understanding of Duchenne with our bite-sized science video series.

Section 1 – Facts about Duchenne muscular dystrophy

Section 2 – Signs and Symptoms of Duchenne muscular dystrophy

Section 3 – Diagnosis of Duchenne muscular dystrophy

Section 4 – Crucial Genetic Terminology

Section 5 – Genetics – Blueprint of Duchenne muscular dystrophy

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Category: Gene therapy, News

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