Orphazyme Stock Gains After FDA Update On Arimoclomol In Rare Neurodegenerative Disorder

Orphazyme A/S ORPH shares are moving higher after providing a regulatory update for arimoclomol in Niemann-Pick disease type C (NPC).
The Company had a collaborative dialogue with the FDA during the Type A meeting to discuss the Complete Response Letter.
Related: FDA Turned Down Arimoclomol Application Asking For Additional Evidence.
The FDA recommended that Orphazyme submit additional data, information, and analyses to address the CRL and engage in further interactions with the FDA to identify a pathway to resubmission.
The agency also agreed with the Company's proposal to remove the cognition domain from the NPCCSS endpoint. The primary endpoint is permitted to be recalculated using the 4-domain NPCCSS, subject to the submission of the additional requested information.
The FDA affirmed requiring additional pharmacodynamic (PD) / pharmacokinetics (PK) data to bolster the confirmatory evidence already submitted.
Related Link: Investors React Positively To Data From Orphazyme's Neurodegenerative Disease Candidate.
An EMA's opinion from the Committee for Medicinal Products for Human Use (CHMP)on arimoclomol in NPC is expected in Q1 2022.
Related content: Benzinga's Full FDA Calendar.
Price Action: ORPH shares are up 13.6% at $4.42 during the premarket session on the last check Monday.