Sarepta resumes gene therapy shipments after controversial FDA review

In the United States, Sarepta Therapeutics reported an increase in its stock shares after announcing the resumption of shipments for its gene therapy, Elevidys, targeting young patients with Duchenne's muscular dystrophy. This decision follows a brief suspension requested by the Food and Drug Administration (FDA) after incidents involving the deaths of two older teenagers and a separate death of an 8-year-old boy who had been using different Sarepta therapies. The FDA's recommendation lifted the hold specifically for younger patients who are still able to walk, as the agency determined that the boy's death was unrelated to the gene therapy. The FDA previously requested a pause in the distribution of Elevidys after identifying potential safety concerns associated with the treatment. The fatalities reported were linked to acute liver injury, a known side effect of the drug. As regulatory bodies oversee the safety of gene therapies, they have emphasized the importance of monitoring clinical outcomes and proposed that Sarepta provide new study data to address safety concerns among older, non-ambulatory patients who may still be at risk due to the paused use of the therapy. Following the news of the FDA's decision, shares of Sarepta rose significantly, suggesting a recovery in the company's outlook. Jefferies analyst Andrew Tsai noted that this development may indicate an improved sales forecast for Elevidys and potentially exhibit stock upside potential. The FDA continues to collaborate with Sarepta to ensure the safety of its therapies, especially concerning older patients who have not been cleared for ongoing treatment as the investigation into the deaths continues. Sarepta has faced challenges in navigating the regulatory landscape, particularly as the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy. The accelerated approval it received in 2023 was initially limited in scope but was later expanded to include older patients. With the current lifting of the hold and the FDA's ongoing engagement regarding safety, Sarepta is looking to stabilize its operations and address the concerns raised by stakeholders while continuing its mission to treat a debilitating condition affecting young boys and men.