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What are the clinical benefits of Exondys 51?

Answers (1)

Official Answer by Drugs.com 30 Nov 2016

Exondys 51 (eteplirsen) was the first drug FDA-approved for Duchenne Muscular Dystrophy (DMD), and was cleared under accelerated approval.

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 percent of the population with DMD.

Exondys 51 was approved under the FDA accelerated approval pathway for serious or life-threatening diseases based on the surrogate endpoint of dystrophin increase in skeletal muscle observed in some patients.

A clinical benefit of Exondys 51, including improved motor function, has not yet been established.

Additional studies are being conducted by the manufacturer, Sarepta Therapeutics, to fully confirm the drug’s clinical benefit.

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