Sarepta’s CEO Douglas Ingram highlighted a wealth of efficacy data for its lead product Elevidys following a 2025 fraught with safety issues for the single dose gene therapy.
Speaking at the ongoing J.P. Morgan Healthcare Conference in San Franciso, taking place from 12 to 15 January, Ingram contextualised the difficult past year against hope for a more successful 2026.
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“In 2025, because of the issues we were dealing with, we spent all of our time talking about safety, and that was the right thing to do and the necessary thing to do. But one needs both sides of the equation. You really need to understand the efficacy of this therapy, and the efficacy of this therapy was getting lost,” Ingram said.
Elevidys, which was initially approved for both ambulatory and non-ambulatory Duchenne muscular dystrophy (DMD) patients, was linked to two non-ambulatory patient deaths in mid-2025. The US Food and Drug Administration (FDA) placed a commercial hold on Elevidys shipments at the time, which was lifted after a review. However, Sarepta is not dosing non-ambulatory patients with the gene therapy, and a boxed warning regarding liver injury risks was added in November 2025. Elevidys’ label is now only indicated for ambulatory DMD patients.
“They were liver failures associated with older boys who were non-ambulatory. You have to contextualise all that. We’ve dosed over 1,100 patients. You have to look at the numerator and denominator, but nevertheless, a very difficult situation,” Ingram added.
Those issues, combined with a severe flu season and six rescheduled infusions, meant that Elevidys missed sales expectations in Q4. Elevidys’ revenue for the quarter came in at $110.4m, below a William Blair analysts’ estimate of $128.8m.
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By GlobalDataWilliam Blair analyst Sami Corwin said: “Although Elevidys infusions were expected to be down in the fourth quarter, we view the Elevidys revenue miss as unfortunate. In addition, despite the company reiterating the anticipated annual Elevidys sales floor of $500m, the lack of revenue guidance makes it challenging to determine if the company is seeing a true return in Elevidys demand.”
Preliminary total net product revenue for the company in Q4 2025 was $369.6m, above a consensus estimate of $358m. Strong demand for Sarepta’s phosphorodiamidate morpholino oligomer (PMO) products offset the weaker Elevidys sales.
Looking ahead to 2026, Ingram is confident that Elevidys can reach the DMD population yet to be treated. Enhanced monitoring and the use of immunosuppressant sirolimus will be key safety drivers going forward. A cohort in the Phase Ib ENDEAVOUR trial (NCT04626674) is evaluating administering sirolimus before and after treatment with Elevidys, the results of which are expected at the back end of 2026.
“That’s our pathway back to having discussions with the agency about getting [non-ambulatory patients] back on the therapy,” Ingram commented.
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