FDA rejects muscular dystrophy drug, says it doesn't work
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October 26, 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 chi...
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