BioMarin Fails to Win Approval for Drug to Treat Duchenne

BioMarin Pharmaceutical Inc. failed to win U.S. approval Jan. 14 for a drug to treat Duchenne muscular dystrophy, a deadly genetic disease that usually affects young boys.

The Food and Drug Administration rejected the once-a-week injection known as Kyndrisa, according to a Jan. 14 statement from the company. A panel of outside advisers voted in November that the medication, known chemically as drisapersen, didn’t conclusively show improved walking ability in patients with the muscle-wasting disease.

“FDA has concluded that the standard of substantial evidence of effectiveness has not been met,” BioMarin said in the statement announcing the rejection. “BioMarin is ...