Arimoclomol

(ar im″ oh kloe′ mol)

Brand Name(s): Miplyffa^®

WHY is this medicine prescribed?

Arimoclomol is used in combination with miglustat to treat neurologic symptoms in adults and children 2 years of age and older 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). Arimoclomol is in a class of medications called heat shock protein activators. It is not known exactly how arimoclomol works to treat 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?

Arimoclomol comes as a capsule to take by mouth. It is usually taken with or without food three times a day. Take arimoclomol 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 arimoclomol exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.

Swallow the capsules whole; do not chew or crush them.

If you are unable to swallow capsules whole, you may open the capsules and sprinkle the contents over one tablespoonful (15 mL) of water, apple juice, or a soft food such as applesauce, pudding, or yogurt in a small cup. Stir the mixture with a spoon for 15 seconds and swallow the mixture immediately. Do not store the mixture for future use.

If you have a nasogastric (NG) tube, your doctor or pharmacist will explain how to prepare arimoclomol to give through an NG tube.

Arimoclomol controls Niemann-Pick disease but does not cure it. Continue to take arimoclomol even if you feel well. Do not stop taking arimoclomol 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 arimoclomol,

• tell your doctor and pharmacist if you are allergic to arimoclomol, any other medications, or any of the ingredients in arimoclomol capsules. 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 while taking arimoclomol. Your doctor may need to change the doses of your medications or monitor you carefully for side effects.

• tell your doctor if you have or have ever had kidney disease.

• tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding. Your doctor may tell you to use birth control to prevent pregnancy while you are taking arimoclomol. If you become pregnant while taking arimoclomol, call your doctor. Arimoclomol may harm the fetus.

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?

Arimoclomol may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:

• diarrhea
• weight loss
• decreased appetite
• nasal congestion, runny nose, sneezing, sore or scratchy throat, cough, or fever
• headache
• uncontrollable shaking of a part of the body

Some side effects can be serious. If you experience any of these symptoms, stop taking arimoclomol and call your doctor immediately or get emergency medical treatment:

• hives; rash; itching; swelling of the face, eyes, mouth, throat, tongue, or lips; difficulty breathing or swallowing; dizziness
• seizure

Arimoclomol 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 light, excess heat and moisture (not in the bathroom).

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.

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.