Zevra Therapeutics Receives the US FDA’s Approval of Miplyffa (Arimoclomol Citrate) for Treating Niemann-Pick Disease Type C (NPC) 

Shots:    The US FDA has awarded priority review voucher & approved Miplyffa (47mg to 124mg, oral, TID) + miglustat to treat neurological manifestations in adults & pediatrics (≥2yrs.) with NPC, with its launch planned within 8-12wks.   Approval was based on trial of Miplyffa + miglustat vs PBO in NPC patients (2-19yrs.), showing halted disease progression…