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Levacetylleucine
Medication From Wikipedia, the free encyclopedia
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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][2] Levacetylleucine is a modified version of the amino acid leucine.[1] It is the L-form of acetylleucine. It is taken by mouth.[1]
The most common side effects include abdominal pain, difficulty swallowing, upper respiratory tract infections, and vomiting.[1][2]
Levacetylleucine was approved for medical use in the United States in September 2024.[1][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.[2] The FDA considers it to be a first-in-class medication.[4]
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Medical uses
Levacetylleucine is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C in people weighing at least 15 kilograms (33 lb).[1][2]
Adverse effects
The most common side effects include abdominal pain, difficulty swallowing, upper respiratory tract infections, and vomiting.[2]
Levacetylleucine may cause embryo-fetal harm if used during pregnancy.[1][2]
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.[2] The duration was twelve weeks for each treatment period.[2] The study enrolled 60 participants.[2] 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.[2] Participants could receive miglustat, an enzyme inhibitor, as background treatment in the study.[2] The trial was conducted at eleven sites in seven countries in Europe and Australia.[5]
The US Food and Drug Administration (FDA) granted the application for levacetylleucine priority review, fast track, orphan drug, and rare pediatric disease designations.[2] The FDA granted approval of Aqneursa to IntraBio Inc.[2]
Society and culture
Legal status
Levacetylleucine was approved for medical use in the United States in September 2024.[1][2][6]
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).[7] The applicant for this medicinal product is IntraBio Ireland Ltd.[7][8]
Names
Levacetylleucine is the international nonproprietary name.[9]
Levacetylleucine is sold under the brand name Aqneursa.[1]
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Research
Levacetylleucine is being studied for the treatment of GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases),[10] ataxia-telangiectasia,[11] Lewy body dementia,[12] amyotrophic lateral sclerosis, restless legs syndrome, multiple sclerosis, and migraine.[13]
References
Further reading
External links
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