FDA grants Orphazyme Inc. to study drug arimoclomol breakthrough therapy

The U.SFDA(http://granted its drug(http://arimoclomol breakthrough therapy for the treatment of Type C Niemann-Disease Type Type C,announced http://
todayOrphazyme plans to submitnew drug(http://application (NDA) to the FDA in the first half of 2020Arimoclomol is an oral small molecule heat shock stress-resusor developed by Orphazyme that penetrates the blood-brain barrierIt plays a role in stress cells by stimulating the cell's own heat shock response, increasing the production of HSP70, which in turn reduces the build-up of lipidsPreviously, the FDA has granted arimoclomol orphan drug eligibility, rare pediatric disease identification, and fast-track eligibilityCurrently, arimoclomol is being developed as a potential therapy for treatment stoicism for four rare diseases, including amyotrophic lateral sclerosis (ALS), the distribution of obsessive-body myocarditis (sIBM), NPC, and Gosche disease 　　Orphazyme's proprietary heat shock protein (HSP) platform aims to create a natural system that allows proteins to work properly It can refold the misfolded protein into the correct conformation and restore its function, or use the lysosome to recover the protein that will not return to normal function, so that it no longer forms a toxic accumulation 　　It can also protect cells by inhibiting the permeability of the lysosome membrane, stabilizing the lysosome, and enabling the function of the cell to remove waste HSP70 is a heat shock protein that has been shown to prevent protein build-up and is an essential cofactor in the process of metabolism of lipids