Sarepta Therapeutics saw a significant uptick in its stock value after the U.S. Food and Drug Administration (FDA) announced revised prescribing information for Elevidys, their gene therapy designed for Duchenne muscular dystrophy (DMD). This update introduces the FDA's most stringent safety alert, a Boxed Warning, and narrows the approved patient population for the treatment. These regulatory adjustments stem from tragic reports of fatal liver injuries observed in young, non-ambulatory patients who had received the therapy.
The updated FDA guidelines specify that Elevidys is now exclusively indicated for use in ambulatory patients aged four years and older who have a confirmed Duchenne muscular dystrophy genetic mutation. This restriction was implemented directly in response to instances of acute liver failure, some of which were fatal, occurring in patients unable to walk. Prior to the FDA's formal announcement, Sarepta had already proactively suspended the administration of Elevidys to non-ambulatory individuals.
Reports indicated that in the fatal cases, children developed severely elevated liver enzymes and required hospitalization within two months of receiving the treatment. Beyond these tragic outcomes, another severe, non-fatal liver injury case involved complex complications, including mesenteric vein thrombosis, damage to the bowel, tissue necrosis, and portal hypertension. These adverse events underscored the critical need for a reevaluation of the therapy's safety profile and appropriate usage.
Following a comprehensive review of all available safety data, the FDA enacted several key changes to the Elevidys label. These included the addition of a prominent Boxed Warning highlighting the potential for severe and life-threatening liver damage. Furthermore, eligibility for the treatment was strictly limited to ambulatory patients aged four and older, thereby excluding those who are non-ambulatory. The updated label also provides new recommendations for when the therapy should or should not be used, alongside revisions in the safety, dosage, side effect, and patient counseling sections. To ensure patient and caregiver awareness, a new Medication Guide has also been released.
Despite a near 50% decline over the previous six months, Sarepta's stock experienced a considerable rally following the FDA's announcement. On Friday, shares closed 5.56% higher, settling at $18.81, reflecting a positive market response to the clarified regulatory pathway and the company's proactive measures in light of the safety concerns.