FDA Approves Miplyffa for the Treatment of Niemann-Pick Disease Type C

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Sept. 20, 2024 – Zevra Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved MiplyffaTM (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.

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