Sarepta Therapeutics Faces Losses After Refusing FDA's Halt Request

Sarepta Therapeutics shares fell 4.8% premarket on Monday, extending losses for a second session after a 36% plunge on Friday. The company refused to halt all shipments of its Elevidys gene therapy, following a request from the FDA. The FDA had raised concerns about the therapy's efficacy and safety, leading to Sarepta's shares falling significantly. The company's refusal to halt shipments has led to further losses.

Sarepta Therapeutics Inc. (SRPT) experienced a significant drop in its stock price on Monday, extending losses from a 36% plunge on Friday. The company refused to halt all shipments of its Elevidys gene therapy, following a request from the Food and Drug Administration (FDA) [1]. The FDA had raised concerns about the therapy's efficacy and safety, leading to Sarepta's shares falling significantly. The company's refusal to halt shipments has led to further losses.

The FDA requested that Sarepta voluntarily stop all shipments of Elevidys, a gene therapy for Duchenne muscular dystrophy, due to safety issues related to the treatment [1]. The company has reported three patient deaths linked to its gene therapies, including Elevidys and another experimental therapy [1]. Sarepta has maintained that the deaths are isolated incidents and that the therapy remains safe for ambulant patients [2].

Sarepta's stock has been on a downward trajectory since the FDA's request. The company's shares have fallen more than 87% this year, with further losses following its refusal to halt shipments [1]. The top concern for investors is whether the FDA will pull the drug from the market, which could be catastrophic for Sarepta [1].

The company has stated that it is committed to working with the FDA to ensure patient safety and informed access to care. Sarepta has paused shipments of Elevidys for non-ambulant patients while it evaluates a safer way to administer the treatment [2]. The company also reported the death of a 51-year-old non-ambulant patient in a Phase 1 clinical trial for a different gene therapy [2].

The FDA has been investigating two patient deaths tied to Elevidys, which accounts for more than half of Sarepta's total net product revenue. The company has also reported a third death tied to a separate experimental gene therapy [1]. The safety risks of Elevidys are especially important given the uncertain benefit, said BMO analyst Kostas Biliouris [1].

Sarepta's executives have tried to reassure investors that the therapy should bring in at least $500 million a year if it can only treat patients who can still walk, where deaths haven't been reported [1]. However, the company's stock has continued to decline, reflecting investor concerns about the FDA's decision-making process.

References:
[1] https://www.cnbc.com/2025/07/18/sarepta-stock-falls-on-fda-gene-therapy-elevidys-comment.html
[2] https://www.statnews.com/2025/07/18/sarepta-therapeutics-duchenne-elevydis-fda-shipments-request/