FDA Approves Miplyffa for the Treatment of Niemann-Pick Disease Type C
Zevra Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved Miplyffa™ (arimoclomol) capsules as an orally delivered treatment for Niemann-Pick disease type C (NPC).
- The first NPC drug approved by the FDA, Miplyffa is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older.
- Those living with NPC experience progressive physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills.
Source: Zevra Therapeutics, Inc.
