Version July 2025
Niemann-Pick disease type C:
Note: Dose using actual body weight.
Children and Adolescents:
15 to <25 kg: Oral: 1,000 mg twice daily.
25 to <35 kg: Oral: 1,000 mg three times daily.
≥35 kg: Oral: 2,000 mg in the morning, 1,000 mg in the afternoon, and 1,000 mg in the evening.
There are no dosage adjustments provided in the manufacturer's labeling (has not been studied).
There are no dosage adjustments provided in the manufacturer's labeling (has not been studied).
(For additional information see "Levacetylleucine: Drug information")
Niemann-Pick disease type C: Oral: 2 g once every morning and 1 g once every afternoon and evening for a total of 4 g daily.
There are no dosage adjustments provided in the manufacturer’s labeling (has not been studied).
There are no dosage adjustments provided in the manufacturer’s labeling (has not been studied).
The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified. Adverse reactions reported in children, adolescents, and adults.
>10%: Respiratory: Upper respiratory tract infection
1% to 10%:
Dermatologic: Exacerbation of acne rosacea
Gastrointestinal: Abdominal pain, dysphagia, vomiting
Hematologic & oncologic: Thrombocytopenia
There are no contraindications listed in the manufacturer’s labeling.
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Packet, Oral:
Aqneursa: 1 g (28 ea) [strawberry flavor]
No
Pack (Aqneursa Oral)
1 g (per each): $576.43
Disclaimer: A representative AWP (Average Wholesale Price) price or price range is provided as reference price only. A range is provided when more than one manufacturer's AWP price is available and uses the low and high price reported by the manufacturers to determine the range. The pricing data should be used for benchmarking purposes only, and as such should not be used alone to set or adjudicate any prices for reimbursement or purchasing functions or considered to be an exact price for a single product and/or manufacturer. Medi-Span expressly disclaims all warranties of any kind or nature, whether express or implied, and assumes no liability with respect to accuracy of price or price range data published in its solutions. In no event shall Medi-Span be liable for special, indirect, incidental, or consequential damages arising from use of price or price range data. Pricing data is updated monthly.
Oral:
Packet for oral suspension: Administer with or without food. Add contents of one packet to 40 mL of water, orange juice, or almond milk and stir to form a suspension; do not use hot liquids for preparation. Administer the mixture within 30 minutes. If more than one packet is required to complete a dose (eg, 2,000 mg dose), repeat this process.
Administration via feeding tube:
Gastric tube (eg, G-tube) (≥18 French): Add contents of one packet to 40 mL of purified water (do not use hot water) and stir to form a suspension. Draw up mixture into enteral dosing syringe and immediately administer via feeding tube. After administration, flush feeding tube with 20 mL of purified water. Continue to flush the feeding tube until no residual suspension remains in the enteral dosing syringe or feeding tube. If more than one packet is required to complete the dose, repeat this process for each packet.
Oral: Administer with or without food. Add contents of 1 packet to 40 mL of water, orange juice, or almond milk and stir to form a suspension; do not use hot liquids. Administer the mixture immediately (within 30 minutes). If >1 packet is required to complete a dose (eg, 2 g dose), repeat this process.
Administration via feeding tube:
Gastric (eg, G-tube) tube (≥18 French): Add contents of 1 packet to 40 mL of purified water and stir to form a suspension; do not use hot water. Draw up mixture into enteral dosing syringe and immediately administer via feeding tube. After administration, flush feeding tube with an additional 20 mL of water. Continue to flush the feeding tube until no residual suspension remains in the enteral dosing syringe or feeding tube. If >1 packet is required to complete the dose, repeat this process.
This medication is not on the NIOSH (2024) list; however, it may meet the criteria for a hazardous drug. Levacetylleucine may cause teratogenicity.
Use appropriate precautions for receiving, handling, storage, preparation, dispensing, transporting, administration, and disposal. Follow NIOSH and USP 800 recommendations and institution-specific policies/procedures for appropriate containment strategy (Ref).
Note: Facilities may perform risk assessment of some hazardous drugs to determine if appropriate for alternative handling and containment strategies (Ref). Refer to institution-specific handling policies/procedures.
Store at 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C to 30°C (59°F to 86°F).
Treatment of neurological manifestations of Niemann-Pick disease type C (NPC) (FDA approved in pediatric patients weighing ≥15 kg and adults).
Substrate of OAT1/3;
Note: Interacting drugs may not be individually listed below if they are part of a group interaction (eg, individual drugs within “CYP3A4 Inducers [Strong]” are NOT listed). For a complete list of drug interactions by individual drug name and detailed management recommendations, use the drug interactions program by clicking on the “Launch drug interactions program” link above.
Note: Interacting drugs may not be individually listed below if they are part of a group interaction (eg, individual drugs within “CYP3A4 Inducers [Strong]” are NOT listed). For a complete list of drug interactions by individual drug name and detailed management recommendations, use the drug interactions program
P-glycoprotein/ABCB1 Substrates (Narrow Therapeutic Index/Sensitive with Inhibitors): Levacetylleucine may increase serum concentration of P-glycoprotein/ABCB1 Substrates (Narrow Therapeutic Index/Sensitive with Inhibitors). Risk C: Monitor
Verify pregnancy status prior to initiating treatment in patients who could become pregnant. Patients who could become pregnant should use effective contraception during therapy and for 7 days after the last dose of levacetylleucine.
Based on data from animal reproduction studies, in utero exposure to levacetylleucine may cause fetal harm.
Pregnancy test (in patients who can become pregnant) prior to initiation of therapy.
The distinct molecular target for levacetylleucine in the treatment of Niemann-Pick disease type C is unknown.
Distribution: Vd: 253 L.
Metabolism: Metabolized into acetate and L-leucine by ubiquitously expressed enzymes, which are used endogenously in catabolic and metabolic pathways.
Half-life elimination: ~1 hour.
Time to peak: 1 hour (range: 0.5 to 2.5 hours).
Excretion: Clearance: 139 L/hour.
