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Recently, the marketing application (relevant acceptance number: CYHS2000381) of the 4-type generic drug "Maglustat Capsule" of Suzhou Amerigen Pharmaceutical Co.
Niemann-Pick disease type C (NPC) is a rare autosomal recessive lysosomal storage disorder in which intracellular lipid transport is impaired due to mutations in the NPC1 and NPC2 genes (95% and 4%, respectively).
The original research miglustat (miglustat, Zavesca) is a glucosylceramide synthase inhibitor developed by Actelon, which reduces the accumulation of such lipids in patients by preventing the biosynthesis of glycosphingolipids
In January 2009, Zavesca was approved in Europe for the treatment of type C Niemann-Pick disease (NPC), becoming the only drug currently approved for the treatment of NPC in the world
In China, the original research Magnastat was approved by the NMPA in November 2016 for the treatment of progressive neurological symptoms in adults and children with NPC, and its trade name is Zeveco
Amerigen Pharmaceutical's megastat capsules were reported for domestic production in June 2020, and were subsequently included in priority review by CDE in September 2020
When it comes to type C Niemann-Pick disease, we have to mention Miplyffa (arimoclomol) from Orphazyme A/S
In addition, Cyclo Therapeutics' Trappsol® Cyclo™ is also being developed for the treatment of NPC
