Sarepta shares decline after EMA recommends refusal of ELEVIDYS marketing authorization.

European Medicines Agency (EMA) recommends refusal of marketing authorization for Sarepta's ELEVIDYS, a treatment for Duchenne muscular dystrophy. This follows a series of negative events for Sarepta, including patient deaths, a securities class action, a significant workforce reduction, and a black box warning for acute liver injury and failure. Hagens Berman is investigating the legal claim.

On July 24, 2025, the European Medicines Agency (EMA) recommended the refusal of the marketing authorization for ELEVIDYS, Sarepta Therapeutics' (NASDAQ: SRPT) gene therapy medication intended for the treatment of Duchenne muscular dystrophy. This decision follows a series of negative events for Sarepta, including patient deaths, a securities class action, a significant workforce reduction, and a black box warning for acute liver injury and failure.

The EMA's recommendation is based on a key study involving 125 children between the ages of four and seven, which failed to demonstrate that ELEVIDYS had a significant effect on movement abilities after a year. The study showed improvements in North Star Ambulatory Assessment (NSAA) scores in both the ELEVIDYS group and the placebo group, but the difference was not statistically significant [1].

Sarepta has faced numerous challenges in recent months. On March 18, 2025, the company issued a safety update revealing that a non-ambulatory patient in an ELEVIDYS trial had died. This was followed by the company disclosing that EU authorities had requested a review of the patient death and that Sarepta had halted recruitment and dosing in some ELEVIDYS studies. A second death was announced on June 15, 2025, from acute liver failure in another non-ambulatory patient. Sarepta suspended shipments of ELEVIDYS for this patient group and paused dosing in one of its clinical studies. The FDA also issued a safety communication confirming its investigation into the risk of acute liver failure associated with ELEVIDYS treatment [2].

These events have led to a securities class action lawsuit, Dolgicer v. Sarepta Therapeutics, Inc., filed on behalf of investors who allege that Sarepta made misleading statements about the prospects and development of ELEVIDYS. The complaint alleges that Sarepta failed to disclose significant risks and inadequate clinical trial protocols, which resulted in a sharp drop in Sarepta's stock price [2].

Hagens Berman, a national shareholders rights firm, is investigating the legal claims and urges Sarepta investors who suffered substantial losses to submit their losses now. The firm also encourages persons with knowledge who may be able to assist in the investigation to contact its attorneys. The class period for the lawsuit is from June 22, 2023, to June 24, 2025, and the lead plaintiff deadline is August 25, 2025 [1].

Sarepta announced a major restructuring on July 16, 2025, involving a 36% workforce reduction (500 employees) and other steps to annually save about $400 million. The company also agreed with the FDA to include a black box warning of acute liver injury and acute liver failure in Sarepta's ELEVIDYS label [2].

The EMA's recommendation to refuse ELEVIDYS' marketing authorization and the ongoing lawsuit have led to a significant decline in Sarepta's stock price. Investors and financial professionals should closely monitor the developments in this case, as it has the potential to impact Sarepta's future prospects.

References:

[1] https://www.globenewswire.com/news-release/2025/08/06/3128798/32716/en/Sarepta-Therapeutics-SRPT-Declines-Again-On-EMA-Recommendation-to-Refuse-ELEVIDYS-Marketing-Authorization-Securities-Class-Action-Pending-Hagens-Berman.html
[2] https://www.tradingview.com/news/reuters.com,2025-08-05:newsml_NFC6QHMfG:0-sarepta-therapeutics-srpt-announces-restructuring-and-elevidys-black-box-warning-ema-recommends-refusal-of-marketing-authorization-securities-class-action-pending-hagens-berman/