Aqneursa FDA Approval History
Last updated by Judith Stewart, BPharm on Sep 26, 2024.
FDA Approved: Yes (First approved September 24, 2024)
Brand name: Aqneursa
Generic name: levacetylleucine
Dosage form: Granules for Oral Suspension
Previous Name: IB1001
Company: IntraBio Inc.
Treatment for: Niemann-Pick Disease
Aqneursa (levacetylleucine) is a modified amino acid used for the treatment Niemann-Pick disease type C.
- Aqneursa is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.
- Niemann-Pick disease type C (NPC) is a rare autosomal recessive neurodegenerative lysosomal disease characterized by multiple symptoms such as progressive cerebellar ataxia and cognitive decline.
- Aqneursa contains the modified amino acid levacetylleucine, which has been associated with positive symptomatic and neuroprotective disease-modifying effects in NPC.
- FDA approval for Aqneursa was based on the results of a Pivotal Phase 3 trial (IB1001-301) that met all primary and key secondary endpoints, and showed that Aqneursa improved neurological signs and symptoms, functioning, and quality of life versus placebo.
Development timeline for Aqneursa
Further information
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