FDA Rejects BioMarin's Much-Anticipated DMD Drug

Published: Jan 15, 2016

BioMarin Pharmaceutical Inc said on Thursday the U.S. Food and Drug Administration had rejected the company's drug to treat a rare muscle-wasting disorder.

The drug, Kyndrisa, was expected to be the first treatment in the United States for Duchenne muscular dystrophy (DMD) - a disorder that affects one in 3,600 newborn boys and causes rapid muscle degeneration.

The rejection comes after a panel of independent advisers to the FDA indicated that efficacy data on the drug was not persuasive enough for an approval.

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