Sarepta Therapeutics has received the Food and Drug Administration's blessing for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. Exons are pieces of DNA that provide information for making proteins in a person's genome.
"Developing drugs designed for patients with specific mutations is a critical part of personalized medicine," said Eric Bastings, deputy director of the Office of Neuroscience in the FDA's Center for Drug Evaluation and Research. "Today's approval of Amondys 45 provides a targeted treatment option for Duchenne muscular dystrophy patients with this confirmed mutation."
DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. It is the most common type of muscular dystrophy. DMD is caused by mutations in the DMD gene that results in an absence of dystrophin, a protein found in muscle fiber. The first symptoms are usually seen between three and five years of age and worsen over time. DMD occurs in approximately one out of every 3,600 male infants worldwide; in rare cases, it can affect females.