Levacetylleucine
Levacetylleucine, sold under the brand name Aqneursa, is a medication used for the treatment of neurological manifestations of Niemann-Pick disease type C. Levacetylleucine is a modified version of the amino acid leucine. It is the L-form of acetylleucine. It is taken by mouth.
The most common side effects include abdominal pain, difficulty swallowing, upper respiratory tract infections, and vomiting.
Levacetylleucine was approved for medical use in the United States in September 2024. Levacetylleucine is the second medication approved by the US Food and Drug Administration for the treatment of Niemann-Pick disease type C. The FDA considers it to be a first-in-class medication.
Medical uses
Levacetylleucine is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C in people weighing at least.Adverse effects
The most common side effects include abdominal pain, difficulty swallowing, upper respiratory tract infections, and vomiting.Levacetylleucine may cause embryo-fetal harm if used during pregnancy.
History
The safety and efficacy of levacetylleucine for the treatment of Niemann-Pick disease type C were evaluated in a randomized, double-blind, placebo-controlled, two-period, 24-week crossover study. The duration was twelve weeks for each treatment period. The study enrolled 60 participants. To be eligible for the study participants had to be four years of age or older with a confirmed diagnosis of Niemann-Pick disease type C and at least mild disease-related neurological symptoms. Participants could receive miglustat, an enzyme inhibitor, as background treatment in the study. The trial was conducted at eleven sites in seven countries in Europe and Australia.The US Food and Drug Administration granted the application for levacetylleucine priority review, fast track, orphan drug, and rare pediatric disease designations. The FDA granted approval of Aqneursa to IntraBio Inc.
Society and culture
Legal status
Levacetylleucine was approved for medical use in the United States in September 2024.In July 2025, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Aqneursa, intended for the treatment of neurological manifestations of Niemann-Pick Type C disease in adults and children from six years of age weighing at least. The applicant for this medicinal product is IntraBio Ireland Ltd.
Names
Levacetylleucine is the international nonproprietary name.Levacetylleucine is sold under the brand name Aqneursa.