Sarepta Sees ELEVIDYS Demand Stabilizing as FSHD, DM1 Readouts Near

ELEVIDYS demand is stabilizing after 2025 safety-related disruption, aided by expanded commercial and medical support, though second-half 2026 demand is expected to be modestly below the first half. Sarepta expects multiple-ascending-dose readouts for its FSHD and DM1 programs in the second half of 2026, with early data showing target engagement and no dose-limiting toxicities. The company is enrolling non-ambulatory patients in a study testing whether sirolimus can reduce ELEVIDYS-related acute liver injury, with enrollment expected to finish in 2026 and data anticipated in early 2027.

Sarepta Therapeutics NASDAQ: SRPT outlined plans to stabilize demand for its ELEVIDYS gene therapy while advancing RNA-based programs for facioscapulohumeral muscular dystrophy, or FSHD, and myotonic dystrophy type 1, or DM1, during a Morgan Stanley Global Healthcare Conference fireside chat. Chief Executive Officer Michael Severino, who said he had been with the company for nearly two months, described Sarepta as a company with marketed products that generate revenue and support a pipeline intended to expand its therapeutic footprint. He said the company’s near-term strategy centers on supporting its commercial products and progressing its siRNA pipeline through upcoming clinical readouts.

Severino said ELEVIDYS experienced disruption in 2025 following disclosed safety events, shifting attention among physicians and families toward product risks. He said Sarepta is working to return the discussion to a balanced assessment of benefit and risk, citing data from registrational trials, longer-term studies and three-year data released earlier this year. “ELEVIDYS clearly demonstrates a strong benefit for patients,” Severino said, while noting that the disease it treats is progressive, debilitating and life-shortening.

The company has expanded its commercial field force and medical support for ELEVIDYS, according to Severino. He said Sarepta has increased the number of physician discussions and has seen demand stabilize, with early signs of positive momentum. However, Severino said second-half demand is expected to be modestly below first-half levels. First-half performance benefited from patients already in the treatment queue from 2025, he said. In addition, the average journey from enrollment form to treatment is roughly six months, meaning improvements in new enrollment activity are more likely to affect revenue in 2027 than in the latter half of 2026. Severino characterized the outlook for the currently indicated population as a gradual build rather than a sharp inflection in demand. He also said the company is not providing long-term guidance.

Sarepta is conducting Cohort 8 of its ENDEAVOR study in non-ambulatory patients to evaluate whether sirolimus can reduce acute liver injury associated with ELEVIDYS. The company’s stated hypothesis is that sirolimus could reduce the incidence of acute liver injury by 50% in that population. Severino said the study is enrolling and is expected to complete enrollment in 2026, with 12-week data anticipated in the first quarter of 2027. Sarepta plans to discuss those data with the Food and Drug Administration during the first quarter of next year as it considers a potential return to the non-ambulatory population.

The company expects separate read...