Sarepta's ELEVIDYS faces European Medicines Agency rejection.

The European Medicines Agency (EMA) has recommended the refusal of marketing authorization for ELEVIDYS, a Duchenne muscular dystrophy treatment by Sarepta. This follows a series of negative events for the company, including patient deaths, a securities class action, restructuring, and an agreement with the FDA to include a black box warning for acute liver injury and failure in the drug's label. Sarepta's shares have declined significantly due to these developments.

The European Medicines Agency (EMA) has recommended the refusal of marketing authorization for ELEVIDYS, a medication developed by Sarepta Therapeutics (SRPT) for the treatment of Duchenne muscular dystrophy. This decision comes amidst a series of negative events for the company, including patient deaths, a securities class action lawsuit, significant restructuring, and an agreement with the FDA to include a black box warning for acute liver injury and failure in the drug's label.

On July 24, 2025, the EMA reported that data from a key study involving 125 children between the ages of four and seven failed to demonstrate that ELEVIDYS had a significant effect on movement abilities after a year [1]. The EMA concluded that the difference in the change of North Star Ambulatory Assessment (NSAA) scores between the group that received ELEVIDYS and the placebo group was not statistically significant.

This decision follows a series of setbacks for Sarepta, including patient deaths that led to sharp declines in the company's stock price. The company has faced a securities class action lawsuit, Dolgicer v. Sarepta Therapeutics, Inc., filed by investors who allege that Sarepta made misleading statements about the prospects and development of ELEVIDYS [1]. The complaint alleges that Sarepta failed to disclose significant safety risks and inadequate clinical trial protocols.

Sarepta has also undergone significant restructuring, including a 36% workforce reduction and steps to save about $400 million annually. The company has agreed with the FDA to include a black box warning of acute liver injury and acute liver failure in ELEVIDYS' label [2]. These developments have contributed to a significant decline in Sarepta's share price.

National shareholders rights firm Hagens Berman continues to investigate the lawsuit's legal claims and urges Sarepta investors who suffered substantial losses to submit their losses now [1]. The firm also encourages persons with knowledge who may be able to assist in the investigation to contact its attorneys.

References:
[1] https://www.globenewswire.com/news-release/2025/08/13/3132835/32716/en/Sarepta-Therapeutics-SRPT-Declines-Again-On-EMA-Recommendation-to-Refuse-ELEVIDYS-Marketing-Authorization-Securities-Class-Action-Pending-Hagens-Berman.html
[2] https://www.globenewswire.com/news-release/2025/08/15/3134377/32716/en/Sarepta-Therapeutics-SRPT-Declines-Again-On-EMA-Recommendation-to-Refuse-ELEVIDYS-Marketing-Authorization-Securities-Class-Action-Pending-Hagens-Berman.html