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 (ClinicalTrials.gov 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.
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.