Sarepta resumes gene therapy shipments after FDA gives green light

In the United States, the FDA has allowed Sarepta Therapeutics to resume shipments of its gene therapy, Elevidys, aimed at treating Duchenne's muscular dystrophy. This decision follows a temporary pause imposed after the deaths of two teenage patients using the therapy, which raised concerns over its safety. Recent evaluations by The FDA determined that a death involving an 8-year-old boy was not linked to the treatment, leading to the recommendation to lift the hold for young patients who can still walk. Nonetheless, the FDA indicated that further safety data would be necessary for addressing the use of the therapy among older, non-ambulatory patients. As the first approved gene therapy in the U.S. for Duchenne's muscular dystrophy, Elevidys received initial accelerated approval in 2023 for a specific patient demographic before its use was expanded to include individuals who can no longer walk. Following the latest regulatory decision, shares of Sarepta Therapeutics saw a significant increase, reflecting investor optimism about the favorable change in Elevidys' sales prospects. The company faced scrutiny over the safety of its gene therapy, particularly after the incidents of acute liver injury in several young patients, which triggered an FDA investigation and a temporary halt. The FDA expressed ongoing collaboration with Sarepta regarding the use of Elevidys to ensure patient safety, particularly for older patients who are not ambulatory. Analysts suggested that the FDA's decision could restore confidence in the therapy and lead to a strong uptick in stock value due to anticipated demand from the patient community. The developments have highlighted both the potential of gene therapies in treating severe conditions and the critical need for stringent safety evaluations in the clinical setting. As the market reacts to this news, the lifting of the shipment pause is expected to maintain a focus on Elevidys and its impact on the future of Duchenne's muscular dystrophy treatments. Regulatory bodies, medical professionals, and families affected by this condition are particularly invested in the therapy's safety and efficacy as it becomes available to more patients.