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

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

In the CRL, the FDA requested “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.” The 5-domain version of the NPCCSS includes ambulation, fine motor skills, swallow, cognition, and speech. In a study funded by the Company, the 5-domain NPCCSS was found to be a valid trial endpoint based on patient, caregiver, and clinician assessments.

The Agency also requested more data, beyond the single trial, to provide additional support for approval.

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Orphazyme CEO Christophe Bourdon stated: “We are assessing the potential path forward in the US in partnership with the FDA. In the short-term, we will need to reduce our costs substantially and freeze all company efforts not related to clinical and regulatory activities to support approval for [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 Disease Type C. [press release]. Los Angeles, CA: CytRx Corporation; June 18, 2021.

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