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FDA Medication Tracker

← All approvals · Rare & genetic · 2024

Miplyffa (arimoclomol)

The FDA approved Miplyffa (arimoclomol) on September 20, 2024 to treat Niemann-Pick disease type C.
Brand name
Miplyffa
Generic name
arimoclomol
FDA approval date
September 20, 2024
Approved use (FDA wording)
To treat Niemann-Pick disease type C
Company
Zevra Denmark
Application
NDA214927
Dosage form / route
CAPSULE · ORAL
Boxed warning on label
Not listed
Category on this site
Rare & genetic

Indications and usage, from the FDA label

MIPLYFFA is indicated 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. MIPLYFFA is indicated 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. ( 1 )

Official sources

Facts only. This site lists what the FDA has approved and what the FDA says each drug is approved for, quoted or closely paraphrased from FDA and openFDA records. It gives no medical advice, no dosing, and no comparisons, and it says nothing about whether a drug is right for anyone. Talk to a licensed clinician about any medication. Records can lag or contain errors; the FDA's own pages are authoritative.