The Food and Drug Administration approved the first drug to treat a rare fatal disease that kills children, after a public outcry for a treatment.
The agency approved the Exondys 51 injection to treat patients with Duchenne muscular dystrophy, a condition that primarily affects boys and quickly reduces motor function. The approval comes despite the recommendation of FDA experts who had serious questions about the drug’s safety.
A fierce grassroots effort by patient advocacy groups lobbied heavily for the FDA to approve the treatment. The effort got the attention of Congress, with several lawmakers pushing for the approval.
“Parents are crying out for the FDA to hear them: They are engaged and knowledgeable and only want the agency to do what is already within its power,” according to a May letter from Sens. Ron Johnson, R-Wis., and Dan Coats, R-Ind.
The FDA accelerated approval of the drug based on initial clinical data instead of a longer series of clinical trials.
“We eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval,” said Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research.
The disease primarily affects boys, but girls can also contract it. Duchenne occurs in about one out of every 3,600 male infants, the FDA said.
While experts have taken aim at the safety of the drug, patient advocates have charged that they are willing to face higher safety risks to get any treatment for the fatal disorder.
