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# OrphaZyme Gets ‘Complete Response’ on Arimoclomol
- URL: https://www.fdaweb.com/orphazyme-gets-complete-response-on-arimoclomol/
- Published: 2021-06-18T12:00:00.000Z
- Updated: 2026-09-14T17:04:01.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5149572

FDA has issued Orphazyme a complete response letter on an NDA for arimoclomol, a heat shock protein amplifier intended for treating Niemann-Pick disease Type C (NPC). In the letter, the agency 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 company says.

Additionally, FDA said that additional data are needed to bolster confirmatory evidence beyond the single Phase 2/3 clinical trial to support the NDA’s benefit-risk assessment. “A primary endpoint of the Phase 2/3 clinical trial was progression in disease severity as measured by the 5-domain NPCCSS,” the company says. “This is a disease-specific measure of disease progression consisting of the five clinically most relevant domains to patients with NPC, caregivers and physicians.”

“We are assessing the potential path forward in the U.S. in partnership with the FDA,” the company said in response to the letter.