Levacetylleucine (Monograph)

Brand name: Aqneursa
Drug class: Other Miscellaneous Therapeutic Agents

Medically reviewed by Drugs.com on Nov 10, 2024. Written by ASHP.

Introduction

Levacetylleucine is a modified amino acid.

Uses for Levacetylleucine

Levacetylleucine has the following uses:

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

Levacetylleucine Dosage and Administration

General

Levacetylleucine is available in the following dosage form(s) and strength(s):

For oral suspension: 1 g levacetylleucine in a unit-dose packet.

Dosage

It is essential that the manufacturer's labeling be consulted for more detailed information on dosage and administration of this drug. Dosage summary:

Adults and Pediatric Patients ≥15 kg

Dosage and Administration

• For females of reproductive potential, verify that the patient is not pregnant prior to initiating treatment.

• Administer levacetylleucine orally up to 3 times daily, with or without food.

• Obtain the required number of packets for the prescribed dose (one or two packets). Open and empty the entire contents of one packet into a container with 40 mL of water, orange juice, or almond milk; do not use hot liquid. Stir to form a suspension. For doses requiring 2 packets, repeat steps.

• Recommended dosage is based on the patient's actual body weight (kg). See Table 1.

Cautions for Levacetylleucine

Contraindications

None.

Warnings/Precautions

Embryo-fetal Toxicity

Based on findings from animal reproduction studies, levacetylleucine may cause embryo-fetal harm when administered during pregnancy. Administration of levacetylleucine to pregnant rats and rabbits during the period of organogenesis caused an increase in embryo-fetal death (post implantation loss/resorption) and skeletal malformations at a dose that was approximately 1.4-fold and 6-fold, respectively, the maximum recommended human dose (MRHD) of 4 g/day of levacetylleucine (based on body surface area).

The decision to continue or discontinue levacetylleucine treatment during pregnancy should consider the female’s need for levacetylleucine, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal disease.

For females of reproductive potential, verify that the patient is not pregnant prior to initiating treatment with levacetylleucine. Advise females of reproductive potential to use effective contraception during treatment with levacetylleucine and for 7 days after the last dose if the drug is discontinued.

Specific Populations

Pregnancy

Based on findings from animal reproduction studies, levacetylleucine may cause embryo-fetal harm when administered during pregnancy. In animal reproduction studies, an increase in embryo-fetal death (post implantation loss/resorption), decrease in fetal body weight, and increase in external and skeletal malformations were observed in rats and rabbits when levacetylleucine was administered in these pregnant animals during the period of organogenesis. These effects were observed in rats and rabbits at the doses that were approximately 1.4-fold and 6-fold, respectively, the maximum recommended human dose (MRHD) in patients taking 4 g of levacetylleucine per day.

There are no available data on levacetylleucine use in pregnant females to evaluate a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes. Advise a pregnant female of the potential risk to the fetus. The decision to continue or discontinue levacetylleucine treatment during pregnancy should consider the female’s need for levacetylleucine, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal disease.

The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, miscarriage, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively.

Lactation

There are no data on the presence of levacetylleucine or its metabolites in either human or animal milk, the effects on the breastfed infant or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for levacetylleucine and any potential adverse effects on the breastfed infant from levacetylleucine or from the underlying maternal condition.

Females and Males of Reproductive Potential

Levacetylleucine may cause embryo-fetal harm when administered to a pregnant female.

For a female of reproductive potential, verify that the patient is not pregnant prior to initiating treatment with levacetylleucine.

Advise a female of reproductive potential to use effective contraception during treatment and for 7 days after the last dose if levacetylleucine is discontinued.

Pediatric Use

The safety and effectiveness of levacetylleucine for the treatment of Niemann-Pick disease have been established in 23 pediatric patients weighing ≥15 kg in the principal efficacy study. Use of levacetylleucine for this indication is supported by evidence from one adequate and well-controlled study in adults and pediatric patients weighing ≥15 kg with additional pharmacokinetic data from 40 adults and 17 pediatric patients who participated in two open-label studies. The safety and effectiveness of levacetylleucine have not been established in pediatric patients weighing <15 kg.

Geriatric Use

Niemann-Pick disease is largely a disease of pediatric and young adult patients. Clinical studies of levacetylleucine did not include sufficient numbers of patients 65 years of age and older to determine whether they respond differently from younger adult patients.

Common Adverse Effects

Most common adverse reactions (incidence ≥5% and greater than placebo) are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.

Drug Interactions

Specific Drugs

It is essential that the manufacturer's labeling be consulted for more detailed information on interactions with this drug, including possible dosage adjustments. Interaction highlights:

• N-acetyl-DL-leucine or N-acetyl-D-leucine: Avoid concomitant use with levacetylleucine.

• P-glycoprotein (P-gp) Transporter Substrates: Monitor for adverse reactions if used with levacetylleucine.

Actions

Mechanism of Action

The distinct molecular target for levacetylleucine in the treatment of Niemann-Pick disease is unknown.

Advice to Patients

• Advise the patient and/or caregiver to read the FDA-approved patient labeling (Instructions for Use).

• Levacetylleucine may cause embryo-fetal harm. Advise a pregnant female of the potential risk to the fetus. Advise a female of reproductive potential and caregiver to inform their healthcare provider of a known or suspected pregnancy.

Additional Information

AHFSfirstRelease^™. For additional information until a more detailed monograph is developed and published, the manufacturer's labeling should be consulted. It is essential that the manufacturer's labeling be consulted for more detailed information on usual uses, dosage and administration, cautions, precautions, contraindications, potential drug interactions, laboratory test interferences, and acute toxicity.

Preparations

Excipients in commercially available drug preparations may have clinically important effects in some individuals; consult specific product labeling for details.

Please refer to the ASHP Drug Shortages Resource Center for information on shortages of one or more of these preparations.

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