Sarepta Therapeutics Faces Backlash Over Gene Therapy Safety Issues and Regulatory Rejection

Sarepta Therapeutics faces a securities class action lawsuit and European regulatory rejection of its gene therapy, ELEVIDYS, due to safety and efficacy concerns. The lawsuit alleges that Sarepta misled investors about ELEVIDYS' safety and approval prospects, potentially damaging investor trust. The company's stock price has declined significantly due to safety issues and halted trials. Sarepta must navigate these challenges to maintain its financial stability and market position.

Sarepta Therapeutics (SRPT) is facing significant challenges following the European Medicines Agency’s (EMA) rejection of its gene therapy, ELEVIDYS, and a securities class action lawsuit. The EMA’s decision, announced on July 24, 2025, concluded that ELEVIDYS failed to demonstrate clear benefits for patients with Duchenne muscular dystrophy, a rare and fatal muscle-wasting disorder [1]. This setback comes amidst a securities class action lawsuit alleging that Sarepta misled investors about the drug's safety and approval prospects.

The EMA’s recommendation to refuse marketing authorization for ELEVIDYS was based on data from a pivotal trial involving 125 children aged 4 to 7. The trial showed no significant difference in movement improvement between those who received ELEVIDYS and those who were given a placebo [1]. Sarepta’s stock price has declined significantly due to these safety issues and halted trials. The company’s stock price experienced sharp declines after disclosures of patient deaths and regulatory scrutiny [2].

The securities class action lawsuit, Dolgicer v. Sarepta Therapeutics, Inc., alleges that Sarepta misled investors by portraying ELEVIDYS as a safe and effective treatment with a clear path to broader regulatory approval. The complaint claims that Sarepta withheld crucial information about significant safety risks and that the company knew these risks would likely lead to regulatory scrutiny and trial halts [2]. Key disclosures, including patient deaths and regulatory investigations, have triggered sharp drops in Sarepta’s stock price.

Hagens Berman, a national shareholders rights firm, is leading the investigation into the lawsuit’s legal claims and urges Sarepta investors who suffered substantial losses to submit their losses now. The firm also encourages persons with knowledge who may assist in the investigation to contact its attorneys [1]. The deadline for Sarepta investors with losses who wish to be considered by the court to serve as Lead Plaintiff is August 25, 2025 [1].

Sarepta must navigate these challenges to maintain its financial stability and market position. The company is currently restructuring and implementing cost-cutting measures, including a 36% workforce reduction and a black box warning for acute liver injury and acute liver failure in ELEVIDYS’s label [2].

References:
[1] https://www.globenewswire.com/news-release/2025/08/18/3135187/32716/en/Sarepta-Therapeutics-SRPT-August-25-2025-Lead-Plaintiff-Deadline-Approaching-SRPT-Investors-with-Substantial-Losses-Encouraged-to-Contact-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