US Pharm. 2016;41(10):10.
Silver Spring, MD—The FDA has granted accelerated approval to eteplirsen injection (Exondys 51, Sarepta Therapeutics), the first drug approved to treat patients with Duchenne muscular dystrophy (DMD), the most common type of muscular dystrophy. DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13% of the DMD population. Under the accelerated approval provisions, the FDA is requiring the manufacturer to conduct a clinical trial to confirm the drug’s clinical benefit in improving motor function.
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