Miplyffa FDA Approval History
Last updated by Judith Stewart, BPharm on Sep 23, 2024.
FDA Approved: Yes (First approved September 20, 2024)
Brand name: Miplyffa
Generic name: arimoclomol
Dosage form: Capsules
Company: Zevra Therapeutics, Inc.
Treatment for: Niemann-Pick Disease
Miplyffa (arimoclomol) is a heat shock protein amplifier used in combination with miglustat for the treatment of Niemann-Pick disease Type C.
- 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.
- Niemann-Pick disease type C (NPC) is a rare, neurodegenerative lysosomal storage disorder caused by mutations in the NPC1 or NPC2 genes, which are responsible for making the NPC1 and NPC2 lysosomal proteins. It is characterized by an inability of the body to transport cholesterol and other lipids within cells, leading to the abnormal accumulation of these substances within various tissues of the body, including brain tissue. This results in impaired speech, cognition, swallowing, ambulation, and fine motor skills.
- Miplyffa works in the treatment of NPC by amplifying the production of heat shock proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes.
- Miplyffa capsules are administered orally, three times a day, with or without food.
Development timeline for Miplyffa
Further information
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