**Sarepta Therapeutics Defies FDA Request Amid Safety Concerns**
Sarepta Therapeutics Inc. has declined to halt shipments of its Elevidys treatment, despite the Food and Drug Administration (FDA) linking three deaths to the company’s gene therapies. The FDA reported that two teenage boys, who were being treated for Duchenne muscular dystrophy and were unable to walk due to the disease, died from acute liver failure after receiving Elevidys. Additionally, a 51-year-old patient in an early-stage trial for a gene therapy aimed at limb-girdle muscular dystrophy also succumbed to acute liver failure last month.
In a statement, the FDA revealed that its leaders met with Sarepta and requested a voluntary suspension of all Elevidys shipments, which represent the company’s most significant product. However, Sarepta chose to continue distribution, asserting that their comprehensive analysis of the data indicated no new safety concerns for patients who can still walk.
Following this news, Sarepta’s stock plummeted by 36%, reaching its lowest point since 2016. The company has seen a staggering loss of approximately $8.5 billion in market value since March 18, when the first patient death was reported. Elevidys is crucial for Sarepta, accounting for over half of its net product revenue in the second quarter of this year. In June, the company had already suspended shipments for patients who are unable to walk, and the FDA has now advised that the gene therapy should only be administered to boys who can still walk, who represent about 85% of Elevidys recipients.
The recent developments have raised significant concerns regarding the future of Elevidys. Analyst Brian Skorney from Baird noted that the FDA’s request for Sarepta to cease shipments amplifies the risk of the drug being completely withdrawn from the market. The latest patient death occurred during a trial of a gene therapy that employs a similar viral delivery method to Elevidys, raising potential safety implications for current patients.
In an interview, FDA Commissioner Marty Makary stated that the agency is reviewing whether Elevidys should remain available for sale, as it was already investigating the previous two patient deaths. Makary’s comments came in response to a question from a news outlet, although he did not provide further details. The FDA is facing scrutiny for its approval of Sarepta’s gene therapy, which was granted despite early trials not definitively demonstrating its efficacy in slowing the disease. This situation presents a significant challenge for Vinay Prasad, the new head of the FDA’s gene therapy division, who has previously criticized the expedited approval process for Sarepta.
**FAQ**
**What is Elevidys and why is it significant?**
Elevidys is a gene therapy developed by Sarepta Therapeutics for treating Duchenne muscular dystrophy. It is significant as it accounts for a large portion of the company’s revenue and represents a critical treatment option for patients with this debilitating condition.
