Treatment of Niemann-Pick disease type C (NPC) in patients aged 6 months and older in combination with miglustat.
MIPLYFFA — ZEVRA DENMARK, approved 2024-09-20 (NDA214927).
38 reports across the top reactions. These are spontaneously reported events from the FDA Adverse Event Reporting System — they reflect reporting volume, not incidence or frequency, and a report does not mean the drug caused the event.