WHY is this medicine prescribed?
Levacetylleucine is used to treat neurologic symptoms in adults and children weighing at least 33 pounds (15 kg) who have Niemann-Pick disease type C (NPC; an inherited disease in which a certain fatty substance is not broken down and used normally in the body and instead builds up in some organs and can cause nerve and brain problems). Levacetylleucine is in a class of medications called modified amino acids. It is not known how levacetylleucine works to treat the neurologic symptoms in patients with Niemann-Pick disease type C.
HOW should this medicine be used?
Levacetylleucine comes as packets of granules for suspension (to be mixed with liquid) to take by mouth or be given through a feeding tube. It is usually taken with or without food two or three times a day. Take levacetylleucine at around the same times every day. Follow the directions on your prescription label carefully, and ask your doctor or pharmacist to explain any part you do not understand. Take levacetylleucine exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.
To prepare a dose of levacetylleucine granules for suspension, sprinkle the entire packet of granules in a cup that contains 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. Ask your pharmacist for a cup if one is not included with your medication. Do not use any other liquid to disperse the granules. Do not use hot liquids. If your doctor has told you that need 2 packets of levacetylleucine granules for your dose, repeat the steps above to prepare a second packet of granules.
If you have a nasogastric (NG) tube, your doctor or pharmacist will explain how to prepare levacetylleucine to give through an NG tube.
Levacetylleucine controls Niemann-Pick disease but does not cure it. Continue to take levacetylleucine even if you feel better. Do not stop taking levacetylleucine without talking to your doctor.
Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.
Are there OTHER USES for this medication?
This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.
What SPECIAL PRECAUTIONS should I follow?
Before taking levacetylleucine,
What SPECIAL DIETARY instructions should I follow?
Unless your doctor tells you otherwise, continue your normal diet.
What should I do IF I FORGET to take a dose?
Skip the missed dose and continue your regular dosing schedule. Do not take a double dose to make up for a missed one.
What SIDE EFFECTS can this medicine cause?
Levacetylleucine may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:
Levacetylleucine may cause other side effects. Call your doctor if you have any unusual problems while taking this medication.
If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online (http://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).
What should I do in case of OVERDOSE?
In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at http://www.poisonhelp.org/help. If the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services at 911.
What should I know about STORAGE and DISPOSAL of this medication?
Keep this medication in the container it came in, tightly closed, and out of reach of children. Store it at room temperature and away from excess heat and moisture (not in the bathroom).
Dispose of unneeded medications in a way so that pets, children, and other people cannot take them. Do not flush this medication down the toilet. Use a medicine take-back program. Talk to your pharmacist about take-back programs in your community. Visit the FDA's Safe Disposal of Medicines website http://goo.gl/c4Rm4p for more information.
What OTHER INFORMATION should I know?
Keep all appointments with your doctor.
Do not let anyone else take your medication. Ask your pharmacist any questions you have about refilling your prescription.
Keep a written list of all of the prescription and nonprescription (over-the-counter) medicines, vitamins, minerals, and dietary supplements you are taking. Bring this list with you each time you visit a doctor or if you are admitted to the hospital. You should carry the list with you in case of emergencies.
This report on medications is for your information only, and is not considered individual patient advice. Because of the changing nature of drug information, please consult your physician or pharmacist about specific clinical use.
The American Society of Health-System Pharmacists, Inc. represents that the information provided hereunder was formulated with a reasonable standard of care, and in conformity with professional standards in the field. The American Society of Health-System Pharmacists, Inc. makes no representations or warranties, express or implied, including, but not limited to, any implied warranty of merchantability and/or fitness for a particular purpose, with respect to such information and specifically disclaims all such warranties. Users are advised that decisions regarding drug therapy are complex medical decisions requiring the independent, informed decision of an appropriate health care professional, and the information is provided for informational purposes only. The entire monograph for a drug should be reviewed for a thorough understanding of the drug's actions, uses and side effects. The American Society of Health-System Pharmacists, Inc. does not endorse or recommend the use of any drug. The information is not a substitute for medical care.
AHFS® Patient Medication Information™ Copyright, 2025. The American Society of Health-System Pharmacists®, 4500 East-West Highway, Suite 900, Bethesda, Maryland. All Rights Reserved. Duplication for commercial use must be authorized by ASHP.
Selected Revisions: November 15, 2024.