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.
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