The U.S. Food and Drug Administration approved an updated labeling for Sarepta Therapeutics’ gene therapy Elevidys on November 14, 2025, adding a boxed warning for acute serious liver injury and acute liver failure and limiting the drug to ambulatory patients aged four and older with Duchenne muscular dystrophy (DMD). The change follows a temporary suspension of non‑ambulatory shipments after fatal liver injury cases were reported in that group.
Elevidys had been approved in June 2023 for ambulatory children 4‑5 years old and expanded in June 2024 to include non‑ambulatory patients. The November 2025 update was prompted by reports of fatal acute liver failure in non‑ambulatory pediatric patients and a serious non‑fatal case involving mesenteric vein thrombosis. The FDA now requires a medication guide and a post‑marketing observational study enrolling about 200 patients with 12‑month follow‑up to further assess liver injury risk.
The restriction narrows Elevidys’ addressable market from all DMD patients to only ambulatory patients, a move that is expected to reduce sales volumes, complicate reimbursement negotiations, and slow clinical adoption. The boxed warning signals the highest level of safety concern, which may lead payers to impose stricter coverage criteria and clinicians to exercise greater caution when prescribing the therapy.
Sarepta’s financial performance underscores the commercial impact of the labeling change. In Q3 2024, Elevidys generated $181 million in revenue, rising 39% YoY; the figure jumped to $384 million in Q4 2024 (a 75% YoY increase) and $375 million in Q1 2025 (a 70% YoY rise). Preliminary Q3 2025 results showed a decline to $399 million, a drop from $467 million in the prior year, and an operating loss, reflecting the headwinds from the regulatory shift. In May 2025, Sarepta revised its 2025 guidance down to $2.3 billion–$2.6 billion from the earlier $2.9 billion–$3.1 billion range, citing the Elevidys restriction and broader market uncertainty.
Management has responded with a strategic pivot toward its siRNA platform and a cost‑reduction plan that includes a 36% workforce reduction and $400 million in annual savings. CEO Doug Ingram said the company remains committed to advancing therapies for rare diseases but will focus resources on programs with more established safety profiles. R&D President Louise Rodino‑Klapac emphasized that the updated labeling “provides clear information for families and healthcare professionals” and that the company will continue to monitor safety data closely.
Investors reacted cautiously to the update, weighing the potential decline in Elevidys sales against Sarepta’s broader pipeline and the company’s restructuring efforts. The market’s focus on the regulatory change highlights the importance of safety monitoring for gene therapies and the strategic implications for companies operating in this space.
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