Innovative therapy to improve brain's ability of "removing rubbish" has been recognized by FDA as a breakthrough therapy

Today, alphazyme announced that the U.S FDA has awarded its research drug arimoclomol breakthrough therapy to treat Niemann Pick disease type C (NPC) The company plans to submit a new drug application (NDA) to the FDA in the first half of 2020 NPC is a rare and fatal genetic disease, and also one of lysosomal storage diseases It is because NPC gene mutation leads to a large amount of lipid accumulation in tissues and organs, which can not be digested by lysosomes, and then leads to cell stress response and toxicity Currently, there is no approved treatment for the disease in the United States and only one approved drug in Europe Alphazyme's proprietary heat shock protein (HSP) platform is designed to build a natural system that allows proteins to work properly It can refold the misfolded protein into the correct conformation and restore its own function, or it can not recover the normal function of the protein by using lysosome, so that it will no longer form toxic accumulation It can also protect cells by inhibiting the permeability of lysosomal membrane, stabilize lysosomes, and make cells function normally to remove waste Hsp70 is a kind of heat shock protein which has been proved to prevent protein accumulation, and it is also an essential auxiliary factor in the process of lipid metabolism The HSP platform restores the original conformation and function of the abnormal protein (photo source: orphozyme official website) Arimoclomol is an oral small molecule heat shock stress inducer which can penetrate the blood-brain barrier developed by orphozyme company It plays a role in stress cells by stimulating the heat shock response of cells, increasing the production of HSP70, and then reducing the accumulation of lipids Previously, the FDA has granted arimoclomol orphan drug qualification, rare pediatric disease identification, and fast track qualification Currently, arimoclomol is being developed as a potential therapy for four rare diseases, including ALS, sIBM, NPC, and Gaucher's disease ▲ arimoclomol can increase the production of HSP70 and reduce the accumulation of lipid (photo source: Official Website of orphozyme) "The FDA's approval of arimoclomol as a breakthrough treatment demonstrates the potential of arimoclomol in the treatment of NPC patients We hope to bring this product to NPC patients as soon as possible, "said Ms Kim Stratton, chief executive officer of orphozyme." we look forward to working closely with FDA to further develop the potential of arimoclomol in different indications We are expected to submit the regulatory application of arimoclomol in the first half of 2020 " reference material: [1] Orphazyme receives Breakthrough Therapy Designation for arimoclomol in Niemann-Pick Disease Type C (NPC)，Retrieved November 19, 2019, from http:// A kind of