Sarepta Therapeutics has received clearance from the FDA to resume shipping its gene therapy, Elevidys, after a temporary halt sparked by safety concerns. This reversal marks a significant development for both the company and patients with Duchenne muscular dystrophy (DMD), a rare and severe muscle-wasting disease primarily affecting young boys.

The pause was initially prompted by a series of adverse events, including the deaths of three patients who experienced acute liver failure after receiving either Elevidys or closely related gene therapies. In response to these incidents, the FDA requested Sarepta suspend shipments and halted ongoing clinical trials to thoroughly assess the underlying risks. The scrutiny focused on ensuring the safety profile of Elevidys, particularly as the gene therapy had recently received expanded approval for ambulatory DMD patients.

Following detailed discussions and data reviews, the FDA decided to allow Sarepta to start shipping Elevidys again — but only for certain patients. The therapy is currently approved for ambulatory DMD patients, and the agency is maintaining restrictions and additional oversight for non-ambulatory individuals. Sarepta is expected to conduct further studies to monitor liver function more closely and confirm the long-term safety of the treatment. The FDA emphasized its commitment to protecting patient safety and signaled that further regulatory action could be taken if new risks emerge.

The news of Elevidys returning to the market has been welcomed by many patient advocates and drove Sarepta’s stock price sharply higher. Despite these positive developments, the situation remains fluid as both the FDA and Sarepta continue to investigate and monitor adverse events associated with gene therapy for muscular dystrophy. Investors, patients, and healthcare providers will be watching closely as Sarepta navigates these heightened safety and regulatory demands.