Medical Spotlight At the 2026 J.P. Morgan Healthcare Conference, Sarepta Therapeutics CEO Douglas Ingram offered an optimistic outlook for the future of its gene therapy, Elevidys, despite significant safety and revenue challenges in 2025. The company’s current focus is on reinforcing clinical efficacy and re-establishing safe treatment protocols for the Duchenne muscular dystrophy (DMD) community.
Key Developments and Facts:
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Safety Challenges: In 2025, Elevidys was linked to two patient deaths due to acute liver failure in non-ambulatory individuals. Consequently, the FDA mandated a Boxed Warning regarding the risk of serious liver injury and restricted the therapy’s indication.
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Revised Indication: Elevidys is currently indicated only for ambulatory DMD patients aged 4 years and older. Distribution for non-ambulatory patients remains suspended pending further safety data.
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Financial Performance: Elevidys’ fourth-quarter 2025 revenue totaled $110.4 million, missing analyst estimates. This was attributed to a severe flu season and the rescheduling of several infusions into 2026. However, overall company revenue remained stable due to strong demand for its legacy PMO products.
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New Safety Strategy: To mitigate risks, Sarepta is implementing an enhanced immunosuppressive regimen using sirolimus. The ongoing ENDEAVOUR study (Cohort 8) is evaluating whether administering sirolimus before and after gene therapy infusion can effectively prevent acute liver injury.
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2026 Roadmap: The company anticipates that data from the sirolimus cohort, expected in late 2026, will serve as a pathway for discussions with the FDA to potentially restore access for non-ambulatory patients.