Sarepta Therapeutics, Inc. Provides Update on UK Dosing in ENVISION Study of Eletidys for the Treatment of Duchenne Muscular Dystrophy

Published: 2025-05-21 11:38:31 ET SRPT

Published on 05/21/2025 at 11:38

Sarepta Therapeutics, Inc. shared the following update related to ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy. The company has received feedback from the Medicines & Healthcare products Regulatory Agency (MHRA) in the United Kingdom (U.K.) that dosing may continue uninterrupted in ENVISION, study SRP-9001-303. ENVISION is a global, randomized, double-blind, placebo-controlled Phase 3 study of ELEVIDYS in non-ambulatory and older ambulatory individuals with Duchenne.

Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unre remarkable (normal clinical exam, GGT, and Total bilirubin levels return to near baseline levels).

Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended.

Immune-mediated Myositis: In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71.

Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagIA, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient's clinical presentation and medical history if these symptoms occur. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials.

If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. Adverse Reactions: The most common adverse reactions (incidence 5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia.

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