FDA Advisors Officially Turn Down PTC Therapeutics' DMD Drug, Data Inconclusive

Published: Sep 29, 2017

(Reuters) - PTC Therapeutics Inc’s experimental drug to treat Duchenne muscular dystrophy, a devastating degenerative disease that mostly affects young boys, may work but the company will need to do more work to prove it, an advisory panel to the U.S. Food and Drug Administration concluded on Thursday.

The panel’s vote reduces the chance the drug will be approved soon since the FDA generally follows the advice of its advisors. Even so, the vote could have been worse for PTC. The panel could have rejected the drug outright, which in turn could have imperiled European sales of the product.

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