Pharma Focus Asia

CytRx Notes Orphazyme’s Regulatory Update from the FDA on Arimoclomol for Niemann-Pick Disease Type C

Saturday, June 19, 2021

CytRx Corporation, a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today noted that Orphazyme A/S announced it has received a Complete Response Letter (“CRL”) from the U.S. Food and Drug Administration (“FDA”) following its review of the new drug application for arimoclomol, a heat shock protein amplifier intended for the treatment of Niemann-Pick disease type C (“NPC”).

Orphazyme’s announcement disclosed that the FDA issued the CRL based on needing 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. Further, the FDA noted in the CRL that additional data are needed to bolster confirmatory evidence beyond the single phase 2/3 clinical trial to support the benefit-risk assessment of the NDA.

A primary endpoint of the phase 2/3 clinical trial was progression in disease severity as measured by the 5-domain NPCCSS. This is a disease-specific measure of disease progression consisting of the five clinically most relevant domains to patients with NPC, caregivers and physicians.

Orphazyme CEO Christophe Bourdon stated: “We are disheartened by the outcome of the FDA’s review, given the urgent need for a new therapeutic option for NPC, but we remain committed to working with the regulators, with the goal of delivering arimoclomol to families managing this challenging disease. We will focus our efforts on pursuing the European regulatory approval, with CHMP opinion expected in Q4 2021 and potential Marketing Authorization in Q1 2022. We are assessing the potential path forward in the U.S. 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 NPC.”

Orphazyme disclosed that as stated in its Annual Report 2020, initial outlook for the year was subject to various risks and uncertainties, including but not limited to the timing of regulatory decisions, the success of Orphazyme’s commercial efforts and development activities. The outcome of the FDA decision has significant influence on Orphazyme’s outlook for full-year 2021. Orphazyme’s cash position at year-end 2021 is now expected to be approximately $8 million (previously $56 million).

Orphazyme noted it will provide an update and further information in the coming weeks.

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