FDA Rejects arimoclomol for treatment of Niemann-Pick disease type C. (NPC)
FDA rejected this heat shock protein amplifier for treating this rare genetic condition on the grounds that further qualitative and quantitative data are needed to support the validity and interpretation of the study’s 5-domain NPC Clinical Severity Scale.
Source:
Biospace Inc.
SPS commentary:
The manufacture expects to receive an opinion on it Marketing Authorization Application in EU in late 2021.