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Pharmacy Bulletin

Pharmacy Bulletin

We share important prescription drug information to help you stay informed about updates concerning particular prescription medicines.

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Miplyffa Approved for Niemann-Pick Disease Type C

On Sept. 20, 2024, Zevra Therapeutics received approval from the U.S. Food and Drug Administration (FDA) for Miplyffa (arimoclomol) for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and children two years of age and older. It is the first drug approved by the FDA to treat NPC. The recommended oral dose of Miplyffa depends on the patient’s body weight. For those weighing 8kg (about 18 pounds) to 15kg (33 pounds), the recommended dose is 47mg three times daily. For patients weighing more than 15kg to 30kg (66 pounds), the recommended dose is 62mg three times daily. Patients weighing more than 30kg to 55kg (121 pounds) should be treated with 93mg three times daily and those weighing more than 55kg should take 124mg three times daily. Miplyffa is expected to be available within eight to 12 weeks. Pricing information is not known at this time. Full prescribing information can be found here.

At a Glance

  • Brand Drug: Miplyffa (arimoclomol)
  • Manufacturer: Zevra Therapeutics
  • Date Approved: Sept. 20, 2024
  • Indication: for use in combination with miglustat for the treatment of neurological manifestations of NPC in adults and children two years of age and older
  • Dosage Forms Available: 47mg, 62mg, 93mg, and 124mg capsules
  • Launch Date: eight to 12 weeks
  • Estimated Annual Cost: Not yet available
  • NPC is a progressive and neurodegenerative lysosomal storage disorder that affects approximately 900 patients in the U.S.; however, only about 300 of these patients have been diagnosed. This genetic disease is characterized by the body’s inability to transport cholesterol and other lipids within cells, which causes the build-up of these substances in a variety of cell types, including neurons. NPC can cause physical limitations as well as neurological impairments impacting speech, cognition, swallowing, ambulation, and fine motor skills.
  • Miplyffa is the first drug approved by the FDA to treat NPC; however, Zavesca® (miglustat – Actelion, generics), an oral drug approved for Gaucher disease, is frequently used off-label.
  • Approval was based on a clinical study that showed that patients treated with Miplyffa in combination with miglustat had a 0.2-point decrease in clinical severity from baseline as measured by the rescored 4-domain NPC Clinical Severity Scale (R4DNPCCSS) compared to 1.9 points of progression for patients treated with miglustat alone.
  • The most common adverse events in treated patients were upper respiratory tract infection, diarrhea, and decreased weight.
  • IntraBio’s acetylleucine is another oral drug that works differently than Miplyffa for the treatment of NPC. Approval is expected by Sept. 24, 2024.
  • Miplyffa was granted Breakthrough Therapy designation, Rare Pediatric Disease designation, Orphan Drug designation and Fast Track designation by the FDA for the treatment of NPC.