More data on arimoclomol’s support in Niemann-Pick type C disease. required

The Food and Drug Administration (FDA) has issued Orphazyme a Complete Response Letter (CRL) to the New Drug Application (NDA) for arimoclomol, an investigational heat shock protein booster for the treatment of Niemann-Pick type C disease.

The NDA filing included data from a randomized, double-blind, placebo-controlled phase 2/3 study ( Identifier: NCT02612129) evaluating the efficacy and safety of arimoclomol in patients with Niemann-Pick type C disease.

In the CRL, the FDA called for “additional qualitative and quantitative evidence to further substantiate the validity and interpretation of the 5-Domain NPC Clinical Severity Scale (NPCCSS), and particularly the swallow domain.” The 5-domain version of the NPCCSS includes walking, fine motor skills, swallowing, cognition, and language. In a company-funded study, the 5-domain NPCCSS was found to be a valid study endpoint based on reviews from patients, caregivers, and clinicians.

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In addition to the individual study, the agency requested additional data to provide additional support for the approval.

Christophe Bourdon, CEO of Orphazyme stated, “We are evaluating the potential path in the US in collaboration with the FDA. In the short term, we need to significantly reduce our costs and freeze all non-clinical and regulatory efforts in order to obtain approval for the Company [Niemann-Pick disease type C]. “

The company is expected to provide an update in the coming weeks.


CytRx notes Orphazyme’s regulatory update from the FDA on arimoclomol for Niemann-Pick type C disease. [press release]. Los Angeles, CA: CytRx Corporation; 06/18/2021.

This article originally appeared on MPR


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