Pharma Focus Asia

Sarepta Therapeutics Announces FDA Expansion of ELEVIDYS for Duchenne Muscular Dystrophy Patients Aged 4 and Up

Friday, June 21, 2024

Sarepta Therapeutics, Inc. (NASDAQ), a leading company in precision genetic medicine for rare diseases, has announced that the U.S. Food and Drug Administration (FDA) has expanded the indication for ELEVIDYS (delandistrogene moxeparvovec-rokl) to include patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene and are at least 4 years old. The FDA has granted traditional approval for ambulatory patients and accelerated approval for non-ambulatory patients. Continued approval for non-ambulatory patients is contingent on verification of clinical benefit through a confirmatory trial. ELEVIDYS should not be used in patients with deletions in exon 8 and/or exon 9 of the DMD gene.

“This label expansion for ELEVIDYS to treat Duchenne patients aged 4 and above, regardless of their ambulatory status, is the result of years of research, development, and investment,” said Doug Ingram, president and CEO of Sarepta. “This is a pivotal moment for the Duchenne community and for the advancement of gene therapy. I extend my gratitude to Drs. Jerry Mendell and Louise Rodino-Klapac for their two-decade-long effort to develop this therapy, to the FDA for expediting its approval, and to the clinical investigators and families who participated in the trials.”

Dr. Jerry Mendell, co-inventor of ELEVIDYS and senior advisor for Medical Affairs at Sarepta, added, “The expanded approval of ELEVIDYS is the culmination of a 50-year journey to find a treatment for Duchenne patients and a nearly 20-year effort to develop an effective gene therapy. This expansion allows clinicians to offer treatment to a wider range of patients and highlights the success of our scientific efforts.”

As part of the accelerated approval pathway, Sarepta is conducting the global Phase 3 ENVISION study (Study SRP-9001-303), a randomized, double-blind, placebo-controlled trial to confirm the clinical benefit of ELEVIDYS in non-ambulatory Duchenne patients.

Under a 2019 collaboration agreement, Sarepta handles regulatory approval and commercialization of ELEVIDYS in the U.S., while Roche manages regulatory approvals and distribution outside the U.S. More information about ELEVIDYS can be found at or by calling 1-888-727-3782.

ELEVIDYS is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion. It targets the genetic cause of Duchenne muscular dystrophy (DMD) by delivering a transgene that codes for ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is indicated for patients aged 4 and older with a confirmed DMD gene mutation, including both ambulatory and non-ambulatory patients. Continued approval for non-ambulatory patients requires verification of clinical benefit.

ELEVIDYS should not be administered to patients with deletions in exon 8 and/or exon 9 of the DMD gene.

Reactions such as hypersensitivity and anaphylaxis have occurred during or after ELEVIDYS administration. Monitor patients during and for at least 3 hours post-infusion. If reactions occur, slow or stop the infusion and provide appropriate treatment. Restart at a lower rate once symptoms resolve. Ensure treatment is available for immediate use. Discontinue for anaphylaxis.

Elevations in liver enzymes or total bilirubin may occur within 8 weeks. Patients with preexisting liver conditions are at higher risk. Postpone administration in cases of acute liver disease until resolved. Monitor liver function weekly for 3 months post-infusion and continue if needed. Use systemic corticosteroids before and after infusion.

Observed approximately 1 month post-infusion in patients with deletions in exon 8 and/or exon 9. Symptoms include severe muscle weakness, dysphagia, dyspnea, and hypophonia. Limited data is available for mutations in exons 1-17 and/or 59-71. Monitor for symptoms and consider additional immunomodulatory treatment if needed.

Monitor troponin-I levels before and weekly for the first month post-infusion, continuing if clinically indicated. Advise patients to seek immediate medical attention for cardiac symptoms.

Preexisting antibodies may affect transgene expression. Perform baseline testing for anti-AAVrh74 antibodies. Administration is not recommended for titers ≥1:400.

Common adverse reactions (≥5%) include vomiting, nausea, liver injury, pyrexia, and thrombocytopenia.



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