Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company, today provides an update on the regulatory status in the United States of its investigational product arimoclomol for Niemann-Pick disease type C (NPC) following a recently held Type A meeting with the U.S. Food and Drug Administration (FDA).
The Company had a collaborative dialogue with the FDA during the Type A meeting, which was held in order to discuss the key topics in the Complete Response Letter (CRL) issued by the FDA in June 2021. The CRL was issued based on the need for additional confirmatory evidence as well as additional qualitative and quantitative evidence to further substantiate the validity of the 5-domain NPC Clinical Severity Scale (NPCCSS), in particular the swallow domain, in the context of a lack of significant findings when using the FDA’s preferred and recommended statistical approach.
The Type A meeting resulted in the following take-aways:
- The FDA recommended that the Company submit additional data, information, and analyses to address certain topics in the CRL and engage in further interactions with the FDA to identify a pathway to resubmission.
- The FDA concurred with the Company’s proposal to remove the cognition domain from the NPCCSS endpoint, with the result that the primary endpoint is permitted to be recalculated using the 4-domain NPCCSS, subject to the submission of additional requested information which the Company intends to provide. To bolster the confirmatory evidence already submitted, the FDA affirmed that it would require additional in vivo or pharmacodynamic (PD)/pharmacokinetic (PK) data; the Company is considering the optimal path forward to address the FDA’s requests.
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