BioCentury
ARTICLE | Regulation

Talking the walk

Why eteplirsen's smaller DMD data set may be more robust than Kyndrisa's

December 7, 2015 8:00 AM UTC

FDA's comments and questions to its Peripheral and Central Nervous System Drugs Advisory Committee hint that drugs intended to treat Duchenne muscular dystrophy could be approved on a modest efficacy threshold. While it seems doubtful that BioMarin Pharmaceutical Inc.'s Kyndrisa drisapersen has met the hurdle, Sarepta Therapeutics Inc.'s eteplirsen may make a more robust showing with a smaller data set.

FDA essentially signaled that it would be willing to approve a DMD treatment as long as data show a clear benefit - even if a small one - that can be credibly attributed to the drug...