Orphazyme A/S (NASDAQ:ORPH) shares are moving sharply higher Monday 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.
The company's Type A meeting was held to discuss the key topics in the Complete Response Letter (CRL) issued by the U.S. Food and Drug Administration FDA in June 2021. The FDA has recommended the company provides supplemental information and analyses, with the health regulator offering to have further interactions to identify a path to resubmission for arimoclomol in NPC.
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.
An EMA's opinion from the Committee for Medicinal Products for Human Use on arimoclomol in NPC is expected in Q1 2022.
ORPH shares are up 37 cents, or 9.5% at $4.26soon after the opening bell Monday.
