The Food and Drug Administration on Monday approved a controversial drug to treat Duchenne muscular dystrophy, a rare disease that confines boys to wheelchairs and condemns them to an early death.
The decision came after months of protracted debate about whether drug maker Sarepta Therapeutics had provided enough evidence to demonstrate that its medication, called eteplirsen, had a meaningful impact on patients. Sarepta said the drug would be priced at about $300,000 a year in the US.
In reaching its decision, the agency essentially overruled its own medical staffers, who earlier this year questioned the effectiveness of the drug, which was tested in a small clinical trial. The wrangling raised still larger questions about standards for approving a drug, especially when it’s intended for patients with a rare and deadly disease and no other treatment options.
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