Levacetylleucine
Medication
From Wikipedia, the free encyclopedia
Levacetylleucine (N-acetyl-L-leucine), sold under the brand name Aqneursa, is a medication used for the treatment of neurological manifestations of Niemann-Pick disease type C.[1][4] Levacetylleucine is a modified version of the amino acid leucine.[1] It is the L-form of acetylleucine. It is taken by mouth.[1]
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| Trade names | Aqneursa |
| Other names | IB1001 |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a624063 |
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| Routes of administration | By mouth |
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| ECHA InfoCard | 100.013.370 |
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| Formula | C8H15NO3 |
| Molar mass | 173.212 g·mol−1 |
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The most common side effects include abdominal pain, difficulty swallowing, upper respiratory tract infections, and vomiting.[1][4]
Levacetylleucine was approved for medical use in the United States in September 2024,[1][4][5] and in the European Union in January 2026.[2][3] Levacetylleucine is the second medication approved by the US Food and Drug Administration (FDA) for the treatment of Niemann-Pick disease type C.[4] The FDA considers it to be a first-in-class medication.[6]
Medical uses
Adverse effects
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.[4] The duration was twelve weeks for each treatment period.[4] The study enrolled 60 participants.[4] 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.[4] Participants could receive miglustat, an enzyme inhibitor, as background treatment in the study.[4] The trial was conducted at eleven sites in seven countries in Europe and Australia.[7]
The US Food and Drug Administration (FDA) granted the application for levacetylleucine priority review, fast track, orphan drug, and rare pediatric disease designations.[4] The FDA granted approval of Aqneursa to IntraBio Inc.[4]
Society and culture
Legal status
Levacetylleucine was approved for medical use in the United States in September 2024.[1][4][8]
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 (NPC) disease in adults and children from six years of age weighing at least 20 kilograms (44 lb).[2] The applicant for this medicinal product is IntraBio Ireland Ltd.[2][9] The CHMP concluded that the active substance, levacetylleucine, could not be considered a new active substance; this conclusion was confirmed in November 2025 after re-examination of the initial opinion.[2] Levacetylleucine was authorized for medical use in the European Union in January 2026.[2][3]
Names
Levacetylleucine is the international nonproprietary name.[10]
Levacetylleucine is sold under the brand name Aqneursa.[1]
Research
Levacetylleucine is being studied for the treatment of GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases),[11] ataxia-telangiectasia,[12] Lewy body dementia,[13] amyotrophic lateral sclerosis, restless legs syndrome, multiple sclerosis, and migraine.[14]
