Introduction to the instructions and marketing status of levacetylleucine

1. Name:levacetylleucine, Aqneursa, L-Acetylleucine, levoacetylleucine,

2. Indications:

Levacetylleucine is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC; rare disease) in adults and children weighing ≥15kg.

3. Usage and dosage:

1. Before use: For women with reproductive potential, confirm that the patient is not pregnant.

2. Recommended dosage: The recommended dosage of levoacetylleucine is based on the patient's actual weight (kg) and can be taken orally up to three times a day. Levacetylleucine can be taken with or without food. For patients weighing 35kg and above, take 2g of leacetylleucine orally in the morning, 1g in the afternoon, and 1g in the evening; for patients weighing 25kg-35kg, take 1g orally in the morning, 1g in the afternoon, and 1g in the evening; for patients weighing 15kg-25kg, take 1g orally in the morning and 1g in the evening.

3. Medication Administration: To prepare a dose of levoacetylleucine granules for suspension, sprinkle the entire packet of granules into a cup containing 40 ml of cold or room temperature water, orange juice, or almond milk; stir with a spoon and drink the entire mixture within 30 minutes. Do not use any other liquid to disperse the particles. Do not use hot liquids.

4. Adverse reactions:

In clinical studies of levoacetylleucine, common adverse reactions include upper respiratory tract infection, abdominal pain, dysphagia, vomiting, etc.

5. Supply and storage:

Levoacetylleucine oral suspension is provided as white to off-white granules. Each sachet contains1.7 grams of white to offwhite granules, equivalent to 1 gram of levoacetylleucine. May be stored at room temperature between 20°C and 25°C (68°F and 77°F); excursions allowed between 15°C and 30°C (59°F and 86°F).

6. Special groups:

1. Women: For women of reproductive potential, please confirm that the patient is not pregnant before starting treatment with this drug. Advise females of reproductive potential to use effective contraception during treatment and, if treatment needs to be discontinued, for 7 days after the last dose.

7. Mechanism of action:

After oral administration, levoacetylleucine is absorbed and distributed to tissues throughout the body via ubiquitously expressed monocarboxylic acid transporters. It enters enzyme-controlled pathways that correct metabolic dysfunction and improve energy production from adenosine triphosphate (ATP). Normalization of energy metabolism improves mitochondrial and lysosomal dysfunction and leads to reduced storage of unesterified cholesterol and sphingolipids. Levacetylleucine has also been shown to normalize neuronal membrane potential in guinea pig models, thereby improving cell signaling processes and restoring and protecting neuronal circuits.

Levoacetylleucine therapyThe unique molecular targets of NPC are unknown.

8. Listing situation:

Levoacetylleucine was approved in the United States in September 2024 by the U.S. Food and Drug Administration (FDA) for medical use and sold under the trade name Aqneursa. It is not currently available for sale in other countries.

Reference materials:https://www.drugs.com/aqneursa.html