Sunstone Life Sciences Fund II portfolio company Orphazyme has received a “Study May Proceed” letter from the FDA on Orphazyme’s protocol for arimoclomol as a new treatment for Niemann-Pick disease type C (NP-C), the AIDNPC intervention study. This makes the US Investigational New Drug Application (IND) effective, complementing the corresponding European Clinical Trial Application (CTA) submitted for the same protocol.

Orphazyme runs an international, multi-centre, double-blind intervention study enrolling approximately 46 NP-C patients to be treated with three daily oral doses of arimoclomol or placebo. The objective of the study is to determine the efficacy and safety of arimoclomol in the treatment of NP-C.