Orphazyme slumps as FDA issues CRL on lead candidate

Having spiked 95% the day before the anticipated positive US regulatory decision on its rare disease treatment arimoclomol, Danish biopharma company Orphazyme (CPH: ORPHA) fell sharply on Friday (June 18), closing down 44% at 56.00 Danish kroner on the news of a delay for its lead pipeline candidate.

Rather than granting approval of Orphazyme’s new drug application for arimoclomol, a heat shock protein amplifier intended for the treatment of Niemann-Pick disease type C (NPC), the Food and Drug Administration issued a Complete Response Letter (CRL).

The FDA issued the CRL based on needing additional qualitative and quantitative evidence to further substantiate the validity and interpretation of the 5-domain NPC Clinical Severity Scale (NPCCSS) and, in particular, the swallow domain. Further, the FDA noted in the CRL that additional data are needed to bolster confirmatory evidence beyond the single phase 2/3 clinical trial to support the benefit-risk assessment of the NDA.