Zevra Therapeutics, Inc. (ZVRA) obtained FDA approval for arimoclomol capsules for the treatment of Niemann-Pick disease type C (NPC) in September. The drug will be marketed under the brand name Miplyffa. The FDA approved Miplyffa for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients aged two years and above. Following the FDA nod, Miplyffa became the first treatment to be approved for NPC, an ultra-rare and progressive neurodegenerativ ...