Sarepta Therapeutics Wins FDA Approval for Duchenne Muscular Dystrophy Injection
Sarepta Therapeutics — which already covers about 30 percent of patients with Duchenne Muscular Dystrophy (DMD) with its two approved muscular dystrophy drugs — got FDA’s approval for a third, Amondys 45 (casimersen) injection.
Amondys 45 is geared toward the 8 percent of Duchenne patients whose MS is connected to a specific genetic mutation. This is the first FDA-approved targeted treatment for patients with this type of mutation.
The agency approved Amondys 45 based on an increase in dystrophin (a protein that helps keep muscle cells intact) production in skeletal muscle observed in patients treated with the therapy.