Miplyffa™ (arimoclomol) – New orphan drug approval
September 20, 2024 - The FDA announced the approval of Zevra Therapeutics’ Miplyffa (arimoclomol), for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older.