News
Complete Response Letter for arimoclomol, a heat shock protein amplifier intended for the treatment of Niemann-Pick disease type C.- Orphazyme A/S
Orphazyme A/S announced it has received a Complete Response Letter from the 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).
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 III clinical trial to support the benefit-risk assessment of the NDA. A primary endpoint of the phase II/III 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.
Condition: Niemann Pick Disease/ASMD
Type: drug