Generic Name: Miglustat Brand Name(s): Generics only. No brands available. Avability: Rx Pregnancy Category: C


What is Miglustat used for?

Miglustat is used for the long-term treatment of a certain rare genetic problem (Gaucher’s disease). It works by decreasing the amount of a certain natural fatty substance (glucosylceramide) that builds up due to this condition. This can help improve symptoms of Gaucher’s disease, such as tiredness, pain in the joints/bones, and a swollen liver or spleen.

How should I take Miglustat?

Take this medication by mouth with or without food as directed by your doctor, usually 3 times a day. The dosage is based on your medical condition and response to treatment.

Take this medication regularly to get the most benefit from it. To help you remember, take it at the same times each day.

Tell your doctor if your condition does not improve or if it worsens.

How do I store Miglustat?

Miglustat is best stored at room temperature away from direct light and moisture. To prevent drug damage, you should not store Miglustat in the bathroom or the freezer. There may be different brands of Miglustat that may have different storage needs. It is important to always check the product package for instructions on storage, or ask your pharmacist. For safety, you should keep all medicines away from children and pets.

You should not flush Miglustat down the toilet or pour them into a drain unless instructed to do so. It is important to properly discard this product when it is expired or no longer needed. Consult your pharmacist for more details about how to safely discard your product.

Precautions & warnings

What should I know before using Miglustat?

Before taking miglustat, tell your doctor or pharmacist if you are allergic to it; or if you have any other allergies. This product may contain inactive ingredients, which can cause allergic reactions or other problems. Talk to your pharmacist for more details.

Before using this medication, tell your doctor or pharmacist your medical history, especially of: kidney disease, numbness/tingling of the hands/feet, stomach/intestinal problems (such as inflammatory bowel disease).

This drug may make you dizzy. Alcohol or marijuana can make you more dizzy. Do not drive, use machinery, or do anything that needs alertness until you can do it safely. Limit alcoholic beverages. Talk to your doctor if you are using marijuana.

Before having surgery, tell your doctor or dentist about all the products you use (including prescription drugs, nonprescription drugs, and herbal products).

During pregnancy, this medication should be used only when clearly needed. Discuss the risks and benefits with your doctor.

It is unknown if this medication passes into breast milk. Because of the possible risk to the infant, breast-feeding while using this drug is not recommended. Consult your doctor before breast-feeding.

Is it safe during pregnancy or breast-feeding?

There are no adequate studies in women for determining risk when using this Miglustat during pregnancy or while breastfeeding. Please always consult with your doctor to weigh the potential benefits and risks before taking Miglustat. Miglustat is pregnancy risk category C according to the US Food and Drug Administration (FDA).

FDA pregnancy risk category reference below:

  • A=No risk,
  • B=No risk in some studies,
  • C=There may be some risk,
  • D=Positive evidence of risk,
  • X=Contraindicated,
  • N=Unknown

Side effects

What side effects can occur from Miglustat?

Nausea, vomiting, stomach/abdominal pain, gas, diarrhea, weight loss, headache, muscle cramps, dizziness, or weakness may occur. If any of these effects persist or worsen, tell your doctor or pharmacist promptly.

To reduce diarrhea, your doctor may tell you to take miglustat between meals, recommend that you change your diet to avoid high-carbohydrate foods, or prescribe a drug (such as loperamide) to treat diarrhea. Talk to your doctor or pharmacist for more details.

Remember that your doctor has prescribed this medication because he or she has judged that the benefit to you is greater than the risk of side effects. Many people using this medication do not have serious side effects.

Tell your doctor right away if you have any serious side effects, including: numbness/tingling/weakness/pain of the hands or feet, new/worsening shaking or tremor of the hands, vision changes, new/worsening bruising/bleeding.

A very serious allergic reaction to this drug is rare. However, get medical help right away if you notice any symptoms of a serious allergic reaction, including: rash, itching/swelling (especially of the face/tongue/throat), severe dizziness, trouble breathing.

Not everyone experiences these side effects. There may be some side effects not listed above. If you have any concerns about a side-effect, please consult your doctor or pharmacist.


What drugs may interact with Miglustat?

Miglustat may interact with other drugs that you are currently taking, which can change how your drug works or increase your risk for serious side effects. To avoid any potential drug interactions, you should keep a list of all the drugs you are using (including prescription drugs, nonprescription drugs and herbal products) and share it with your doctor and pharmacist. For your safety, do not start, stop, or change the dosage of any drugs without your doctor’s approval.

Does food or alcohol interact with Miglustat?

Miglustat may interact with food or alcohol by altering the way the drug works or increase the risk for serious side effects. Please discuss with your doctor or pharmacist any potential food or alcohol interactions before using this drug.

What health conditions may interact with Miglustat?

Miglustat may interact with your health condition. This interaction may worsen your health condition or alter the way the drug works. It is important to always let your doctor and pharmacist know all the health conditions you currently have.


The information provided is not a substitute for any medical advice. You should ALWAYS consult with your doctor or pharmacist before using this Miglustat.

What is the dose of Miglustat for an adult?

Usual Adult Dose for Gaucher Disease

Usual dose: 100 mg orally 3 times a day at regular intervals


-Decreasing consumption of foods high in disaccharides (e.g., lactose or sucrose) and not taking this drug with food has been shown to reduce the risk and/or intensity of gastrointestinal adverse events.

-Dose reduction to 100 mg orally once or twice a day may be necessary due to adverse reactions such as tremor or diarrhea.

Use: As monotherapy for the treatment of mild to moderate type 1 Gaucher disease when enzyme replacement therapy is not a therapeutic option (e.g., due to allergy, hypersensitivity, or poor venous access)

Renal Dose Adjustments

CrCl 50 to 70 mL/min/1.73 m2: Initial dose: 100 mg orally twice a day.

CrCl 30 to 50 mL/min/1.73 m2: Initial dose: 100 mg orally once a day.

CrCl less than 30 mL/min/1.73 m2: Not recommended.

Dose Adjustments

Dose Reductions to 100 mg once or twice a day may be desired in patients experiencing side effects, such as diarrhea or tremor.

Loperamide has been shown to be effective in patients experiencing diarrhea.

Other Comments

Administration advice:

-Take orally at regular intervals

-A missed dose should be taken at the next scheduled time


-Therapy should be guided by physicians experienced in managing Gaucher disease patients.


-Assess platelet count at baseline and as clinically indicated.

-Neurological evaluation at baseline and every 6 months

-Vitamin B12 levels in patients with type 1 Gaucher disease

Patient Advice:

-Patients should be instructed to report: numbness, tingling, pain, or burning in hands or feet.

-Patients should be instructed to report new or worsening tremor.

-Patients should be advised to adhere to dietary instructions and report diarrhea and/or excessive weight loss.

-Advise patient to speak to physician or health care professional if pregnant, intend to become pregnant, or are breastfeeding.

What is the dose of Miglustat for a child?

The dosage has not been established in pediatric patients. It may be unsafe for your child. It is always important to fully understand the safety of the drug before using. Please consult with your doctor or pharmacist for more information.

How is Miglustat available?

Miglustat is available in the following dosage forms and strengths:

  • Oral capsule.

What should I do in case of an emergency or overdose?

In case of an emergency or an overdose, call your local emergency services or go to your nearest emergency room.

What should I do if I miss a dose?

If you miss a dose of Miglustat, take it as soon as possible. However, if it is almost time for your next dose, skip the missed dose and take your regular dose as scheduled. Do not take a double dose.

Hello Health Group does not provide medical advice, diagnosis or treatment.

Review Date: August 10, 2018 | Last Modified: August 10, 2018