Posted by Michael Wonder on 18 Jun 2021
Orphazyme provides regulatory update from FDA on arimoclomol for Niemann-Pick disease type C
18 June 2021 - Orphazyme today announced it has received a complete response letter from the U.S. FDA following its review of the new drug application for arimoclomol, a heat shock protein amplifier intended for the treatment of Niemann-Pick disease type C.
The FDA issued the complete response letter based on needing additional qualitative and quantitative evidence to further substantiate the validity and interpretation of the 5-domain clinical severity scale and, in particular, the swallow domain.
