Complete Response Letter for arimoclomol, a heat shock protein amplifier intended for the treatment of Niemann-Pick disease type C.- Orphazyme A/S
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.
Learning Zones
The Learning Zones are an educational resource for healthcare professionals that provide medical information on the epidemiology, pathophysiology and burden of disease, as well as diagnostic techniques and treatment regimens.
2 mins