Sarepta’s Duchenne Muscular Dystrophy Therapy Wins FDA Approval

The FDA has approved Sarepta Therapeutics Amondys 45, an antisense oligonucleotide, for the treatment of Duchenne muscular dystrophy (DMD) in certain patients. Consistent with the accelerated approval pathway, the continued approval of Amondys 45 may be contingent on confirmation of a clinical benefit in confirmatory trials. The Essence trial – a placebo-controlled confirmatory trial to support the Amondys 45 approval – is ongoing and expected to conclude in 2024.

“This is an important day for Sarepta and, far more importantly, for the patients that we serve. 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,” said Doug Ingram, president and chief executive officer, Sarepta. “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.”

Sarepta has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. (Source: Sarepta Website, 25 February, 2021)