Sarepta Therapeutics, Inc. (NASDAQ: SRPT) had a significant increase in the value of its shares on Monday, rising 48.77% in after-hours trading to $20.62 after closing at $13.86 in the regular session, when it had gained 16.18%.
Following the FDA’s decision to let Sarepta to lift its voluntary halt on shipments of ELEVIDYS (delandistrogene moxeparvovec), their gene therapy for ambulatory individuals with Duchenne muscular dystrophy, the rally was held. The only FDA-approved gene treatment for this uncommon, progressive, and potentially fatal illness is still ELEVIDYS.
FDA Review and Resumption of Supply
Sarepta had voluntarily paused shipments of ELEVIDYS last week at the FDA’s suggestion to allow a thorough safety review. The agency completed its assessment rapidly and recommended the resumption of supply for ambulatory patients.
Shipments to treatment centers are set to recommence immediately. Discussions regarding risk mitigation for non-ambulatory patients are ongoing, with shipments for this group remaining paused pending further agreement on safety protocols.
Safety Evaluation and Brazilian Case Review
A case involving the death of an 8-year-old kid in Brazil was investigated as part of the FDA’s safety evaluation. The FDA affirmed the Brazilian health authorities’ conclusion that ELEVIDYS was not likely to be connected to the occurrence. The regulator allowed Sarepta to restart shipments for the approved patient population after concluding that the therapy was not linked to the death.
Arrowhead Pharmaceuticals’s Significant Accomplishment
Arrowhead Pharmaceuticals said in a related release that Sarepta has made a milestone payment of $100 million. In its Phase 1/2 clinical study of ARO-DM1, an experimental RNA interference (RNAi) treatment for type 1 myotonic dystrophy (DM1), the most prevalent adult-onset muscular dystrophy, Arrowhead met the first of two predetermined enrollment objectives, which led to the payment.
Authorization to escalate dosing has also been granted. Arrowhead anticipates reaching the second enrollment target by late 2025, which would result in an additional $200 million milestone payment from Sarepta.