Sarepta Therapeutics Faces Challenges After Second Death Linked to Gene Therapy
Washington, D.C. – Shares of Sarepta Therapeutics took a significant hit on Monday following the announcement of a second death tied to its gene therapy for muscular dystrophy. The biotech firm revealed over the weekend that a teenage boy passed away due to acute liver injury, a known side effect associated with its treatment, Elevidys.
Sarepta has paused the therapy’s distribution for older patients who can no longer walk. This one-time treatment has been approved for children with a specific genetic variant of Duchenne’s muscular dystrophy, a severe condition marked by muscle weakness and early mortality in males.
While Elevidys was the first gene therapy of its kind approved in the U.S., it has faced intense scrutiny since receiving accelerated approval in 2023. The latest death adds to concerns surrounding the therapy’s safety, prompting Sarepta to halt its clinical trial for older patients. The company plans to consult with an expert panel to develop more stringent safety protocols, which might involve increased use of immune-suppressing medications.
The U.S. Food and Drug Administration (FDA) is engaged with Sarepta regarding any modifications needed in the therapy’s usage. This situation has raised alarms among investors, leading to a staggering 42% drop in Sarepta’s stock, closing at approximately $21 per share.
Analysts are speculating that the FDA might impose further restrictions on Elevidys or could potentially halt its use altogether. This comes as Dr. Vinay Prasad, a key leader at the FDA, has expressed skepticism about the therapy since its initial approval. As stated by Leerink Partners analyst Joseph Schwartz, the recent fatalities could lead the FDA to reassess the therapy’s market availability for non-ambulatory patients.
The therapy employs a disabled virus to deliver a replacement gene aimed at producing dystrophin in patient cells, and it comes at a hefty price tag of $3.2 million for a one-time treatment.
In light of these developments, Sarepta faces critical choices ahead, balancing the needs of patients and the financial implications for their business. The health community watches closely as this story unfolds, echoing debates about the responsibilities of biotech companies in ensuring patient safety.
