Spotlight On Sarepta Post-BioMarin Duchenne Rejection

It was highly likely the FDA was going to turn down BioMarin Pharmaceutical Inc. on its Duchenne muscular dystrophy (DMD) drug Kyndrisa (drisapersen), given the beating it took this past November at the meeting of the agency's Peripheral and Central Nervous System (PCNS) Drugs Advisory Committee and the fact regulators had earlier called the firm's data "inconsistent," and in some cases, "contradictory."