The U.S. Food and Drug Administration (FDA) approved Amondys 45 (casimersen) injection from Sarepta Therapeutics for the treatment of Duchenne muscular dystrophy (DMD) in patients that have a mutation amenable to skipping exon 45. DMD is a genetic disorder that causes muscles to deteriorate and weaken due to dystrophin protein alterations, typically affecting 1 in every 3,500 or 5,000 male children. Doug Ingram, President and Chief Executive Officer of Sarepta, stated, “After years of scientific commitment, investment and development, the approval of Amondys 45, Sarepta’s third approved RNA therapy, offers treatment to the 8% of the DMD community who have a confirmed exon 45 amenable mutation. Along with our other approved RNA therapies, we can now offer treatment options for nearly 30% of Duchenne patients in the U.S. And our commitment to bring therapies to the greatest percentage of the DMD community as soon as possible continues.”
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