FDA rejects muscular dystrophy drug, says it doesn't work
By Linda A. Johnson
Oct. 25, 2017
TRENTON, N.J. (AP) — The U.S. Food and Drug Administration has rejected an experimental drug for a common type of muscular dystrophy.
The FDA's decision Wednesday was expected because the agency had said the drug didn't work in two key patient tests. Last month, FDA advisers voted 10-1 against recommending approval.
The drug from PTC Therapeutics was intended for Duchenne muscular dystrophy patients with a certain genetic mutation. The muscle-destroying disorder affects 1 in 3,500 to 5,000 boys in the U.S.
The South Plainfield, New Jersey company said it will appeal. Stuart W. Peltz, PTC's chief executive, wrote in an email that the company believes the FDA didn't consider evidence that it helped some patients or the pleas from many doctors and patients wanting the drug approved.
The FDA had twice refused to review the company's applications, citing inadequate evidence to approve the drug. The company used a rare maneuver to force a review.
Last year, the FDA approved the first drug for Duchenne's, for a different group of patients, despite inconclusive evidence it worked. But the agency required drugmaker Sarepta Therapeutics to do more studies on its effectiveness.
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