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Treats neurological manifestations of Niemann-Pick disease type C (NPC). NDA 219132, held by Intrabio. Levacetylleucine.
As of Nov 14, 2026, NDA 219132 (Aqneursa), held by Intrabio, was first approved by FDA on Sep 24, 2024, with 1 approved supplement and 0 published complete response letters.
First approved: Sep 24, 2024
Review: Priority
Supplements approved: 1
Complete response letters: 0
What It's For
From the FDA-approved label (Indications and Usage)
AQNEURSA is indicated for the treatment of:
neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.
ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing ≥15 kg. 1.1 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. 1.2 Ataxia-Telangiectasia AQNEURSA is indicated for the treatment of ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing ≥15 kg.
Approvals, supplements, products and documents come from FDA's Drugs@FDA data files, refreshed daily; Drugs@FDA lists approved applications only. Complete response letters are FDA's published letters.Drugs@FDA and FDA complete response letters
