The first heat shock inducer Miplyffa was rejected by the U.S. FDA

The Danish biopharmaceutical company Orphazyme A/S recently announced that the U.

The FDA issued the CRL based on the need for more qualitative and quantitative evidence to further confirm the validity and interpretation of the 5-domain NPC Clinical Severity Scale (NPCCSS), especially the swallowing domain

Molecular structure of arimoclomol (picture source: Wikipedia)

Arimoclomol is a lead compound of Orphazyme.

After oral administration, arimoclomol can be quickly dispersed throughout the body, and can pass through the blood-brain barrier and enter the brain

Currently, arimoclomol is being developed as a potential therapy for the treatment of 4 rare diseases, including: 2 lysosomal storage diseases (C type Niemann-Pick disease [NPC] and Gaucher disease [GD]) And 2 neuromuscular diseases (sporadic inclusion body myositis [sIBM], amyotrophic lateral sclerosis [ALS])

Niemann-Pick disease (NPC) type C is an inherited, progressive, debilitating, and often fatal neurological disease

NPC is a devastating disease, and there is an urgent need for disease modification therapy (DMT)

Data from the pivotal phase 2/3 randomized placebo-controlled trial (CT-ORZY-NPC-002) showed that arimoclomol has a positive effect in stabilizing the neurological progression of NPC, especially in the subgroup of patients ≥4 years of age and receiving miglustat (Meglustat) Stalag) as a subgroup of patients in routine clinical care

Arimoclomol has been shown to have a clinically significant effect on the disease progression of NPC, which is further supported by the biomarker effect, which shows an impact on the biological basis of the disease and has good safety and tolerability

Original source: Orphazyme provides regulatory update from FDA on arimoclomol for Niemann-Pick disease type C