Miplyffa

Arimoclomol is used alongside another medication to help manage neurological symptoms in children and adults with Niemann-Pick disease type C, a rare inherited condition where fatty substances build up in the body and damage the nervous system. The drug belongs to a class that activates heat shock proteins, though the exact mechanism of how it works is not fully understood.[1] Miplyffa is currently being investigated across 4 indications[3], with its most advanced program in Phase 2.[1]

Miplyffa is one of 24 small molecule programs targeting Gaucher disease type 1. It is currently in Phase 2.