Sarepta Therapeutics announced that the U.S. Food and Drug Administration approved dosing in Cohort 8 of its ENDEAVOR study on 25 November 2025. The cohort evaluates a 14‑day peri‑infusion sirolimus regimen followed by 12 weeks of post‑infusion therapy, designed to reduce the risk of acute liver injury in non‑ambulatory patients receiving Elevidys.
Elevidys, the only approved gene therapy for Duchenne muscular dystrophy, had its shipments suspended for non‑ambulatory patients after two deaths from acute liver failure and a FDA boxed warning that limited the indication to ambulatory patients aged four and older. The Cohort 8 approval is a critical step toward re‑establishing commercial dosing for the broader patient population, with the company planning to initiate the cohort by year‑end and complete primary endpoint data collection in the second half of 2026.
Sarepta’s financial performance underscores the commercial importance of this regulatory milestone. In Q4 2024, net product revenues reached $638.2 million, a 75% year‑over‑year increase, with Elevidys contributing $384.2 million. Guidance for 2025 net product revenue has been raised to $2.9 billion–$3.1 billion, reflecting confidence in sustained demand and the potential expansion of Elevidys access.
Management emphasized the significance of the approval. Louise Rodino‑Klapac, Ph.D., president of research & development, said, “Cohort 8 will evaluate integrating sirolimus into our immunosuppression approach, with the goal of mitigating the risk of acute liver injury and restoring access for non‑ambulatory individuals.” Doug Ingram, president and CEO, added, “The approval signals progress in our strategy to become a self‑sustaining, profitable biotech dedicated to improving lives of patients with rare genetic disease.”
The approval is expected to strengthen Sarepta’s commercial prospects and support its ongoing efforts to expand Elevidys reach across all Duchenne patients. While the company has faced headwinds from safety concerns and restructuring, the regulatory decision positions it to address a critical unmet need and potentially unlock a significant portion of its addressable market.
The event is a material regulatory action that could materially alter Sarepta’s revenue trajectory and risk profile, warranting publication for investors and stakeholders monitoring the company’s strategic progress.
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