Sarepta resumes gene therapy shipments after FDA clears recent deaths

In the United States, Sarepta Therapeutics announced the resumption of shipments for its gene therapy targeting Duchenne's muscular dystrophy after a review by federal regulators. This pivotal decision occurred following a requested pause from the Food and Drug Administration, which resulted from the tragic deaths of two teenage patients who were undergoing treatment. The FDA assessed these incidents and determined that the recent death of an 8-year-old boy was unrelated to the gene therapy. The approval to lift the hold primarily applies to young patients who are still capable of walking, a demographic deeply affected by the disease known for causing muscle-wasting in boys and young men, leading to early mortality. Elevidys is the name of the gene therapy that has garnered attention as it stands as the first gene therapy approved in the United States for Duchenne's muscular dystrophy. Analysts viewed the FDA's recent decision as a significant positive for Sarepta, indicating a substantially improved sales outlook in the short term. The stock surged over 16% after the announcement, rising to $13.86 in after-hours trading. Jefferies analyst Andrew Tsai noted the relief among investors and the potential for considerable stock upside following the announcement. However, Sarepta's therapy remains under scrutiny. The FDA expressed that the company would need to provide new safety data to reassure regulators about therapy’s use in older, non-ambulatory patients, who are still subject to a voluntary hold following the previous incidents. The FDA stated that it will continue to collaborate with Sarepta regarding this subgroup of patients, indicating that while some aspects of the therapy can proceed, others remain cautiously monitored by regulators.