HealthDay News — The US Food and Drug Administration has approved the first ever drug, Exondys 51 (eteplirsen), to be used to help treat patients with Duchenne muscular dystrophy (DMD).

Exondys 51 is approved for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, affecting some 13% of patients with the disorder.

Accelerated approval was granted to the new drug based on clinical studies that found an increase in the dystrophin protein among some patients treated with Exondys 51.

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While no clinical benefit was found, approval was granted based on the disorder’s potential risks, its life-threatening nature, and the lack of any other available therapy, the FDA said. The drug’s most common side effects include balance disorder and vomiting.

The drug’s maker, Sarepta Therapeutics, is required to conduct additional studies to assess whether the drug improves motor function or provides any other clinical benefit, the agency said. The FDA added that in the case of negative results from the ongoing studies, it reserves the right to “initiate proceedings to withdraw approval of the drug.”


FDA Grants Accelerated Approval to First Drug for Duchenne Muscular Dystrophy [press release]. Silver Spring, MD: Food and Drug Administration; September 19, 2016.

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