FDA approves first treatment for Niemann-Pick disease type C

Arimoclomol (Miplyffa), an oral medication given in combination with the enzyme inhibitor miglustat, is indicated for the treatment of Niemann-Pick disease, type C (NPC) in adults and children ≥2 years of age. Approval was based on data from a phase 2/3 trial involving 50 patients ages 2 to 19 years with a molecularly confirmed diagnosis of NPC. Compared with placebo, arimoclomol resulted in slower disease progression as measured by the rescored 4-domain NPC Clinical Severity Scale (R4DNPCCSS) score.