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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.

VativoRx Bottle update

Aqneursa Approved for Niemann-Pick Disease Type C

On Sept. 24, 2024, the U.S. Food and Drug Administration (FDA) approved IntraBio’s Aqneursa (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15kg. This is the second drug approved for NPC, but the first stand-alone therapy as Miplyffa (arimoclomol – Zevra Therapeutics) must be used in combination with miglustat. The recommended oral dose of Aqneursa depends on the patient’s body weight. For those weighing 15kg (about 33 pounds) to less than 25kg (55 pounds), the recommended dose is 1g in the morning and evening. For patients weighing 25kg to less than 35kg (77 pounds), the recommended dose is 1g three times daily. Patients weighing 35kg or more should be treated with 2g in the morning and 1g for the afternoon and evening doses. Females with reproductive potential must verify they are not pregnant prior to initiating treatment. Aqneursa is expected to be available within the coming days. Pricing information is not known at this time. Full prescribing information can be found here.

At a Glance

  • Brand Drug: Aqneursa(levacetylleucine)
  • Manufacturer: IntraBio
  • Date Approved: Sept. 24, 2024
  • Indication: neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15kg
  • Dosage Forms Available: 1g unit-dose packet for oral suspension
  • Launch Date: Upon approval
  • 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.
  • Aqneursa is the second drug approved by the FDA to treat NPC and the first stand-alone treatment. Miplyffa was approved last Friday to treat NPC in combination with miglustat.
  • Approval was based on a placebo-controlled clinical trial showing Aqneursa significantly improved neurological signs and symptoms and functioning, quality of life and cognition in 12 weeks. Aqneursa showed a statistically significant 1.37-point reduction in the Scale for the Assessment and Rating of Ataxia (SARA) when compared to placebo.
  • The most common adverse events in treated patients were abdominal pain, difficulty swallowing, upper respiratory tract infections, and vomiting. The labeling for Aqneursa carries a warning that it may cause embryo-fetal harm if used during pregnancy.
  • Aqneursa was granted Rare Pediatric Disease designation, Orphan Drug designation, Priority Review and Fast Track designation by the FDA for the treatment of NPC.