Levacetylleucine Application Scope

Treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.

Levacetylleucine Characteristics

Specification: 1 gram of levacetylleucine per unit-dose packet
Ingredients:
- Active Ingredient: Levacetylleucine
- Inactive Ingredients: Hypromellose, isomalt, strawberry flavor
Properties: Modified amino acid; mechanism of action is not fully understood but is thought to involve normalization of energy metabolism.
Packaging Specification: Supplied in cartons containing 28 unit-dose packets.
Storage: Store at room temperature between 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C to 30°C (59°F to 86°F).
Expiry Date: Refer to the packaging for the specific expiration date.
Executive Standard: Approved under NDA 219132 by the FDA on September 24, 2024.
Approval Number: NDA 219132
Date of Revision: January 29, 2025
Manufacturer: IntraBio Inc.

Dosage and Administration:
- 15 to <25 kg: 1 gram in the morning and 1 gram in the evening.
- 25 to <35 kg: 1 gram in the morning, 1 gram in the afternoon, and 1 gram in the evening.
- ≥35 kg: 2 grams in the morning, 1 gram in the afternoon, and 1 gram in the evening.
Each dose should be prepared by mixing the contents of one packet with 40 mL of water, orange juice, or almond milk, and consumed within 30 minutes.
Adverse Reactions:
- Common side effects include abdominal pain, vomiting, dysphagia (difficulty swallowing), and upper respiratory tract infections.
Contraindications: None identified.
Precautions:
- May cause fetal harm; verify that females of reproductive potential are not pregnant before initiating treatment.
- Advise effective contraception during treatment and for 7 days after the last dose.

Drug Interactions:
- Avoid concomitant use with N-acetyl-DL-leucine or N-acetyl-D-leucine, as they may affect the effectiveness of Aqneursa.
- Monitor for adverse reactions if used with P-glycoprotein (P-gp) transporter substrates such as apixaban, colchicine, cyclosporine, dabigatran, digoxin, edoxaban, rivaroxaban, and tacrolimus.

Note:

If there is a new packaging for the drug, the new packaging shall prevail. The above information is sourced from DengYue Medicine.
It is only for internal discussion among medical staff and does not serve as a basis for medication. For specific medication guidelines, please consult the attending physician.