Section 12 · Monograph
Levacetylleucine
Reviewed Nov 2026FDA label
AqneursaModified amino acid · NPC therapy
- Forms
- Granules for oral suspension: 1g unit-dose packets
- Dose
- Niemann-Pick type C, weight over 15kg: 15-<25kg 2g/day; 25-<35kg 3g/day; 35kg or more 4g/day, split morning/afternoon/evening per label
- MOA
- Mechanism not established (N-acetyl-L-leucine)
Indications
- Neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing 15kg or moreFDA-labeled
Mechanism of Action
- Modified amino acid (N-acetyl-L-leucine); the mechanism of action in NPC is not established
Full Dosing
Adult / usual
- Weight-based, divided into morning, afternoon and evening doses per the label table: 15 to under 25kg 2g/day; 25 to under 35kg 3g/day; 35kg or more 4g/day
- Granules are mixed with water or soft food and swallowed, or given through a feeding tube per label
- Not used together with arimoclomol (Miplyffa) per payer policies
Contraindications
- None
Drug Interactions
- Avoid coadministration with N-acetyl-DL-leucine products
- Possible effects on transporter substrates — consult the current label
Adverse Effects
- Abdominal pain
- Dysphagia
- Upper respiratory tract infection
- Vomiting
Monitoring Parameters
- Pregnancy status before starting and contraception counseling (embryo-fetal toxicity warning)
- Neurologic function (e.g., SARA scale, functional measures)
- Tolerability and weight
Pregnancy & Lactation
- Embryo-fetal toxicity concern from animal data; verify pregnancy status and use effective contraception
- Unknown whether present in human milk