Levacetylleucine

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.

Are there OTHER USES for this medicine?

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

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.

What SPECIAL PRECAUTIONS should I follow?

Before taking levacetylleucine,

• tell your doctor and pharmacist if you are allergic to levacetylleucine, any other medications, or any of the ingredients in levacetylleucine oral suspension. Ask your pharmacist for a list of the ingredients.

• tell your doctor and pharmacist what prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking or plan to take.

• tell your doctor if you have or have ever had any other medical conditions.

• tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding. You should not become pregnant while you are taking levacetylleucine. If you are female, you will need to have a pregnancy test before you start treatment and use birth control to prevent pregnancy during your treatment with levacetylleucine and for 1 week after your final dose. If you become pregnant while taking levacetylleucine, call your doctor immediately. Levacetylleucine can cause fetal harm.

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:

• nasal congestion, runny nose, sneezing, sore or scratchy throat, or cough
• stomach pain
• vomiting
• difficulty swallowing
• redness or pimples on the face

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 (https://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

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).

Unneeded medications should be disposed of in special ways to ensure that pets, children, and other people cannot consume them. However, you should not flush this medication down the toilet. Instead, the best way to dispose of your medication is through a medicine take-back program. Talk to your pharmacist or contact your local garbage/recycling department to learn about take-back programs in your community. See the FDA's Safe Disposal of Medicines website (https://goo.gl/c4Rm4p) for more information if you do not have access to a take-back program.

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 https://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 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.

It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.