FDA panel finds Kyndrisa efficacy inconclusive

Panelists at a meeting of FDA's Peripheral and Central Nervous System Drugs Advisory Committee to discuss Kyndrisa drisapersen from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) generally agreed that the therapy's efficacy to treat Duchenne muscular dystrophy (DMD) has not been established, although some panelists thought some boys benefited from the treatment.

The biotech argued that while Kyndrisa missed the primary endpoint of a Phase III study in which it numerically improved six-minute walking distance (6MWD) vs. placebo by 10 meters (p=0.42), pooled results from a smaller patient group across three studies showed a consistent direction and magnitude of treatment effect. Kyndrisa significantly improved 6MWD vs. placebo in one dose group of one Phase II study and trended towards improvement in another Phase II study. ...