Miglustat Side Effects

Medically reviewed by Drugs.com. Last updated on Aug 27, 2024.

Applies to miglustat: oral capsule.

Precautions

It is very important that your doctor check your progress at regular visits to make sure that this medicine is working properly. Blood and nerve tests (neurological exam) before and during treatment may be needed to check for unwanted effects.

Using this medicine while you are pregnant can harm your unborn baby. If you are a woman who can get pregnant, your doctor may do tests to make sure you are not pregnant before receiving this medicine. Use an effective form of birth control during treatment and for at least 60 days after the last dose. If you think you have become pregnant while using this medicine, tell your doctor right away.

Check with your doctor right away if you are having burning, numbness, tingling, or painful sensations in the arms, hands, legs, or feet. These could be symptoms of a condition called peripheral neuropathy.

Taking this medicine may decrease in the number of platelets in the blood. Your doctor may monitor this periodically during therapy.

This medicine may cause or worsen hand tremors (trembling or shaking of the hands). Tell your doctor right away if you develop new hand tremors or worsening of existing hand tremors while using this medicine.

Diarrhea is the most common side effect for people taking miglustat. Your doctor may give you another medicine (anti-diarrheal) to help treat diarrhea if it is a problem for you. Your doctor may also recommend changes to your diet. You may also lose weight when you start treatment with miglustat.

Some men and women who use this medicine have become infertile (unable to have children). Talk with your doctor before using this medicine if you plan to have children.

Serious side effects of miglustat

Along with its needed effects, miglustat may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur while taking miglustat:

Other side effects of miglustat

Some side effects of miglustat may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects.

Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

For healthcare professionals

Applies to miglustat: oral capsule.

General adverse events

The most commonly reported adverse reactions included diarrhea, flatulence, abdominal pain, weight loss, and tremor; the most common serious adverse reaction was peripheral neuropathy; diarrhea and tremor were the side effects that most commonly required intervention.^[Ref]

Gastrointestinal

• Very common (10% or more): Diarrhea (including osmotic diarrhea; up to 100%), abdominal pain (up to 67%), flatulence (up to 50%), nausea (up to 22%), vomiting (up to 11%)
• Common (1% to 10%): Constipation, dry mouth, dyspepsia, epigastric pain not food-related, bloating, stomach pain, gas, abdominal distension/discomfort
• Frequency not reported: Dysphagia, indigestion, dry mouth^[Ref]

Osmotic diarrhea appears to be due to inhibitory activity of miglustat on intestinal disaccharidases leading to reduced absorption of dietary disaccharides in the small intestine. Diarrhea decreased over time with continued therapy.^[Ref]

Metabolic

• Very common (10% or more): Weight decrease (up to 67%)
• Common (1% to 10%): Anorexia^[Ref]

Weight loss has been observed in approximately 55% to 65% of patients. Peak weight loss occurs between 6 and 12 months, with a mean weight loss of 6% to 7% of body weight. It is unclear if weight loss results from the diarrhea and associated gastrointestinal complaints, a decrease in food intake, or a combination of these or other factors. There is a trend to return to baseline weight after the first year.^[Ref]

Nervous system

• Very common (10% or more): Tremor/exacerbation of existing tremor (about 30%), headache (up to 22%), tremor (up to 17%), dizziness (up to 11%)
• Common (1% to 10%): Unsteady gait, paresthesia, migraine, peripheral neuropathy, ataxia, hypoesthesia
• Frequency not reported: Gait abnormal, gait spastic, hyperreflexia^[Ref]

The most common serious adverse reaction in clinical trials was peripheral neuropathy. Tremor, and worsening tremor were very commonly reported. Compared to the general population, patients with type 1 Gaucher disease are more likely to have peripheral neuropathy. A patient should be evaluated for tremor and neuropathy prior to starting therapy.^[Ref]

Other

• Very common (10% or more): Generalized weakness (up to 17%)
• Common (1% to 10%):Fatigue, asthenia, chills, malaise, pain
• Frequency not reported: Fatigue, lacerations^[Ref]

Ocular

• Very common (10% or more): Visual disturbances (up to 17%)^[Ref]

Musculoskeletal

• Very common (10% or more): Cramps (up to 11%)
• Common (1% to 10%): Muscle spasms
• Frequency not reported: Leg cramps, muscle cramps, back pain, heaviness in arms and legs^[Ref]

Psychiatric

• Common (1% to 10%): Memory loss
• Frequency not reported: Insomnia, decreased appetite, depression, decreased libido^[Ref]

Hematologic

• Common (1% to 10%): Thrombocytopenia
• Frequency not reported: Mild reduction in platelet count^[Ref]

Genitourinary

• Common (1% to 10%): Menstrual disorder^[Ref]

Respiratory

• Frequency not reported: Nasopharyngitis, cough, sinusitis^[Ref]

See also:

Further information

Miglustat side effects can vary depending on the individual. Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Note: Medication side effects may be underreported. If you are experiencing side effects that are not listed, submit a report to the FDA by following this guide.

Medical Disclaimer