Levacetylleucine ("N"-acetyl--leucine), 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 -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 (FDA) 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 (FDA) 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.
Names.
Levacetylleucine is the international nonproprietary name.
Research.
Levacetylleucine is being studied for the treatment of GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases), ataxia-telangiectasia, Lewy body dementia, amyotrophic lateral sclerosis, restless legs syndrome, multiple sclerosis, and migraine.