NEW YORK, Feb. 25, 2021 /PRNewswire/ — The Muscular Dystrophy Association (MDA) today celebrated the decision by the US Food and Drug Administration (FDA) to grant accelerated marketing approval to casimersen (Amondys 45) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 45. It is the fourth exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. Amondys 45 will be made available in the United States and marketed by Sarepta Therapeutics.
In the last five years, three targeted exon-skipping drugs have been approved by the FDA to treat DMD: Exondys 51 and Vyondys 53 from Sarepta Therapeutics and Viltepso from NS Pharma. Exondys 51 was the first targeted therapy approved to treat DMD in a subset of patients with a genetic mutation amenable to skipping exon 51, while Vyondys 53 and Viltepso were approved in December 2019 and August