Generic name: arimoclomol [ ar-IM-oh-KLOE-mol ]
Brand name: Miplyffa
Dosage form: oral capsule (124 mg; 47 mg; 62 mg; 93 mg)
Drug class: Miscellaneous metabolic agents

Medically reviewed by Drugs.com on Oct 7, 2024. Written by Cerner Multum.

What is arimoclomol?

Arimoclomol is used together with miglustat to treat neurological problems associated with Niemann-Pick disease type C (NPC) in adults and children at least 2 years old.

Arimoclomol may also be used for purposes not listed in this medication guide.

Arimoclomol side effects

Get emergency medical help if you have signs of an allergic reaction: hives, difficult breathing, swelling of your face, lips, tongue, or throat.

Your treatment may be permanently discontinued if you have certain side effects.

Common side effects of arimoclomol may include:

• decreased weight;

• cold symptoms such as stuffy nose, sneezing, sore throat; or

• diarrhea.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Related/similar drugs

Warnings

Use only as directed. Tell your doctor if you use other medicines or have other medical conditions or allergies.

Before taking this medicine

Tell your doctor if you have or have ever had:

• kidney disease.

May harm an unborn baby. Do not use if you are pregnant. Use effective birth control while using arimoclomol to prevent pregnancy. Tell your doctor if you become pregnant or think you may be pregnant.

Ask a doctor if it is safe to breastfeed while using arimoclomol.

Arimoclomol may affect fertility in men or women. Pregnancy could be harder to achieve while either parent is using this medicine.

How should I take arimoclomol?

Follow all directions on your prescription label and read all medication guides or instruction sheets. Use the medicine exactly as directed.

Arimoclomol is usually taken twice per day. You may take arimoclomol with or without food. Follow your doctor's instructions.

Doses are based on weight. Your dose may change if you gain or lose weight.

Swallow the capsule whole and do not crush, chew or break.

Read and carefully follow the Instructions for Use provided with this medicine on how to open an arimoclomol capsule to prepare a mixture if you cannot swallow the capsule whole.

Arimoclomol can also be given through a feeding tube. Do not store the mixture for later use. Ask your doctor or pharmacist if you don't understand these instructions.  

You may need certain medical tests to check your kidney function.

Store in the original container at room temperature away from moisture, heat, and light.

Arimoclomol dosing information

Usual Adult Dose for Niemann-Pick Disease Type C:

Weight 8 to 15 kg: 47 mg orally 3 times a day
Weight greater than 15 to 30 kg: 62 mg orally 3 times a day
Weight greater than 30 to 55 kg: 93 mg orally 3 times a day
Weight greater than 55 kg: 124 mg orally 3 times a day

Comments:
-Dosage is based on actual body weight.

Use: In combination with miglustat, for the treatment of neurological symptoms of Niemann-Pick disease type C (NPC)

Usual Pediatric Dose for Niemann-Pick Disease Type C:

2 years and older:
-Weight 8 to 15 kg: 47 mg orally 3 times a day
-Weight greater than 15 to 30 kg: 62 mg orally 3 times a day
-Weight greater than 30 to 55 kg: 93 mg orally 3 times a day
-Weight greater than 55 kg: 124 mg orally 3 times a day

Comments:
-Dosage is based on actual body weight.

Use: In combination with miglustat, for the treatment of neurological symptoms of NPC

What happens if I miss a dose?

Skip the missed dose and use your next dose at the regular time. Do not use two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

What should I avoid while taking arimoclomol?

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

What other drugs will affect arimoclomol?

Other drugs may affect arimoclomol, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all other medicines you use.

Further information

Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Medical Disclaimer

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