Actelion needs to provide more info to FDA for Zavesca in Niemann-Pick disease
This article was originally published in Scrip
Executive Summary
The US FDA has issued Actelion with a complete response letter for its drug Zavesca (miglustat), for the treatment of progressive neurological manifestations in adults and children with Niemann-Pick type C (NP-C) disease – a rare, fatal neurodegenerative genetic disorder.