FDA Finds Fault With Third Duchenne Muscular Dystrophy Drug Filing
This article was originally published in The Pink Sheet Daily
PTC Therapeutics receives refuse to file letter from FDA for its DMD drug Translarna; agency says NDA is 'not sufficiently complete' to permit substantive review.
You may also be interested in...
US FDA's Peripheral and Central Nervous System Drugs Advisory Committee to consider application, which PTC Therapeutics filed over protest.
Repeated trial failures in this rare disease space have given birth to new alliances – one backed by Critical Path and the other by the CureDuchenne advocacy group – that aim to model disease progression much more accurately through data sharing and rigorous analysis.
Agency expects to issue 101 guidances in 2017, including those on REMS assessment, refusal to file NDA and BLA submissions, and amendments to ANDAs.