FDA approves Sarepta's controversial muscular dystrophy drug

Cambridge, Mass.-based Sarepta Therapeutics earned tentative approval from the Food and Drug Administration for its Duchenne's muscular dystrophy drug eteplirsen, according to The New York Times.

Duchenne's muscular dystrophy is a rare degenerative condition that causes muscle weakness, loss of movement and eventually death, according to the report. Eteplirsen represents the first FDA-approved treatment for the disease.

The drug was cleared under the FDA's accelerated approval program designed for drugs that show promising results but have not been confirmed. Since the approval was based on Sarepta's study of just 12 boys, the agency is requiring the drugmaker to conduct a larger study examining eteplirsen's ability to improve patient movement and function. If the drug proves ineffective, the FDA could withdraw approval, according to the report.

"Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial," said Janet Woodcock, MD, director of the FDA's drug center.

The FDA deferred its decision on whether to approve the drug in late May after an advisory panel concluded it was not effective at treating the disease. The deferral led to an intense and emotional public campaign from patients, parents and physicians calling for the drug's approval.

Speculations over eteplirsen's imminent approval arose last week after news broke that one of the drug's biggest critics, Ronald Farkas, MD, PhD, left his role at the FDA.

Sarepta's stock increased by more than 75 percent to $21.37 upon news of the approval.


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