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Miglustat oral

Updated 2 Feb 2023 | Gaucher's disease


Oral formulations of miglustat.

Drugs List

  • miglustat 100mg capsules
  • YARGESA 100mg capsules
  • ZAVESCA 100mg capsules
  • Therapeutic Indications


    Treatment of neurological manifestations in Niemann-Pick type C disease
    Treatment of Type 1 Gaucher disease in patients for whom ERT is unsuitable


    Therapy should be directed by physicians who are knowledgeable in the management of Gaucher disease or Niemann-Pick type C disease.

    A dose reduction may be necessary in some patients if miglustat is not tolerated.


    Type 1 Gaucher disease
    100mg three times daily.

    Dose reduction to 100mg once or twice daily if necessary.

    Niemann-Pick type C disease
    200mg three times daily.


    Niemann-Pick C disease
    Children aged 12 to 18 years:200mg three times daily.

    Children aged 4 to 12 years: Adjust according to body surface area as below:

    Body surface area greater than 1.25 metre square: 200mg three times a day.
    Body surface area greater than 0.88 to 1.25 metre square: 200mg twice a day.
    Body surface area greater than 0.73 to 0.88 metre square: 100mg three times a day.
    Body surface area greater than 0.47 to 0.73 metre square: 100mg twice a day.
    Body surface area lower than or equal to 0.47 metre square: 100mg once a day.

    Patients with Renal Impairment

    In patients with an adjusted creatinine clearance of 50 to 70ml/min/1.73 square metres, the recommended starting dose is 100mg twice daily in patients with type 1 Gaucher disease and 200mg twice daily (adjusted for body surface area in patients below the age of 12) in patients with Niemann-Pick type C disease.

    In patients with an adjusted creatinine clearance of 30 to 50ml/min/1.73 square metres, the recommended starting dose is 100mg once daily in patients with type 1 Gaucher disease and 100mg twice daily (adjusted for body surface area in patients below the age of 12) in patients with Niemann-Pick type C disease.


    Children under 4 years
    Renal impairment - creatinine clearance below 30ml/minute/1.73m sq

    Precautions and Warnings

    Children under 18 years
    Hepatic impairment
    Renal impairment - creatinine clearance between 30-70ml/minute/1.73m sq

    Severe Gaucher's disease: Safety & efficacy not established
    Advise patient dizziness may affect ability to drive or operate machinery
    Treatment to be initiated and supervised by a specialist
    Pre-treatment neurological examination recommended
    Monitor linear growth in children
    Monitor platelets
    Monitor vitamin B12 levels regularly
    Periodic neurological examination
    Reduce dose or discontinue treatment if tremor occurs or worsens
    Female: Ensure adequate contraception during treatment
    Male: Use barrier contraception during and for 3 months after treatment

    Enzyme replacement therapy (ERT) is the standard of care in patients who require treatment for type 1 Gaucher disease. In treatment-naive patients with type 1 Gaucher disease there is no evidence of an efficacy or safety advantage of miglustat over ERT.

    There is an increased risk of peripheral neuropathy in patients with type 1 Gaucher disease.

    Animal studies have shown adverse effects on spermatogenesis and reduced fertility.

    Incidents of gastrointestinal events, mainly diarrhoea, have been reported in more than 80% of patients taking miglustat, and the majority of cases would be expected to resolve spontaneously. In clinical practice, diarrhoea may respond to anti-diarrhoeal medicinal products (e.g. loperamide), taking miglustat away from meals, and/or diet modification (reduction of lactose and carbohydrate intake). Temporary dose reduction may be necessary. If patients with chronic gastrointestinal symptoms do not respond to interventions, investigate with accordance to clinical practice. Miglustat has not been evaluated in patents with a history of significant gastrointestinal disease e.g. inflammatory bowel disease.

    The benefit of treatment for neurological function in patients with Niemann-Pick type C disease should be evaluated on a regular basis such as every 6 months. Continuation of miglustat should be re-appraised after at least 1 year of treatment.

    Mild reductions in platelet counts without association with bleeding has been observed in some patients with Niemann-Pick type C disease and type 1 Gaucher disease. In Niemann-Pick type C disease patients included in the clinical trial, 40 to 50% of patients had platelet counts below the lower limit of normal at baseline.

    Pregnancy and Lactation


    Miglustat is contraindicated during pregnancy.

    The manufacturer does not recommend using miglustat during pregnancy. Animal studies have shown reproductive toxicity, including dystocia. At the time of writing there is no adequate data available from the use of miglustat during human pregnancy. Miglustat is known to cross the placenta. Risks are unknown.


    Miglustat is contraindicated during breastfeeding.

    The manufacturer recommends that breastfeeding is discontinued during treatment with miglustat. It is unknown if miglustat is excreted in human breast milk. A risk to the neonate cannot be excluded.

    Side Effects

    Abdominal discomfort
    Abdominal distension
    Abdominal pain
    Cognitive impairment
    Decreased appetite
    Impaired co-ordination
    Muscle spasm
    Muscle weakness
    Peripheral neuropathy
    Reduced libido
    Weight loss


    It is strongly recommended that the UK National Poisons Information Service be consulted on cases of suspected or actual overdose where there is doubt over the degree of risk or about appropriate management.

    The following number will direct the caller to the relevant local centre (0844) 892 0111

    Information may be obtained if you have access to ToxBase the primary clinical toxicology database of the National Poisons Information Service. This is available via password on the internet ( ) or if this is unavailable at the backup site ( ).

    Further Information

    Last Full Review Date: September 2020

    Reference Sources

    Summary of Product Characteristics: Yargesa (miglustat) 100mg Hard Capsules. Piramal Critical Care Ltd. Revised January 2020.

    Summary of Product Characteristics: Zavesca (miglustat) 100mg Hard Capsules. Janssen-Cilag Ltd. Revised February 2020.

    NICE Evidence Services Available at: Last accessed: 8 September 2020

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