miglustat at a glance
Medicine class
Substrate-reduction therapy; an inhibitor of glucosylceramide synthase.
Licensed uses
Selected adults with type 1 Gaucher disease, and adults or children with progressive neurological manifestations of Niemann–Pick type C disease.
Administration
Swallow the capsule whole with water. It may be taken with or without food.
Specialist treatment
Treatment should be directed by a clinician experienced in Gaucher disease or Niemann–Pick type C disease.
What is miglustat used for?
The selected UK product is licensed for:
Oral treatment of mild to moderate type 1 Gaucher disease in adults, only when enzyme replacement therapy is unsuitable.
Treatment of progressive neurological manifestations of Niemann–Pick type C disease in adults and children.
How does miglustat work?
Miglustat inhibits glucosylceramide synthase, reducing the first step in the production of many glycosphingolipids. This limits formation of materials that accumulate abnormally in Gaucher disease and Niemann–Pick type C disease.
miglustat preparations and strengths
Capsule
Route: Oral
Strengths: 65 mg, 100 mg
How to take miglustat
Follow the specialist’s instructions and do not alter or stop treatment without discussing it with them.
Swallow capsules whole with a glass of water.
Capsules may be taken with or without food.
Taking miglustat between meals and adjusting dietary carbohydrates may help gastrointestinal effects, but changes should be agreed with the clinical team.
miglustat side effects
Common or expected effects
- Diarrhoea, wind and abdominal pain
- Reduced appetite and weight loss
- Tremor
- Nausea, vomiting, indigestion or constipation
- Headache or dizziness
- Tiredness, muscle spasms or muscle weakness
Get urgent medical advice
- New tingling, numbness, reduced sensation, weakness or coordination problems suggesting peripheral neuropathy
- Low platelet count, particularly if accompanied by unusual bleeding or bruising
- Persistent diarrhoea or abdominal pain that does not improve, which requires assessment for another cause
miglustat in pregnancy
Miglustat crosses the placenta and should not be used during pregnancy. Anyone who is pregnant, planning pregnancy or who could become pregnant should discuss contraception and treatment with the specialist team; male patients also require reliable contraception during treatment and for three months after stopping.
miglustat while breastfeeding
Miglustat should not be taken while breastfeeding because it is unknown whether it passes into human milk. Discuss feeding options with the specialist team.
miglustat interactions
Tell the specialist or pharmacist about all prescribed, non-prescribed and herbal products. The selected SmPC identifies limited formal interaction data.
Imiglucerase enzyme replacement therapy
Concurrent use may modestly reduce exposure to miglustat; the clinical team should review the treatment combination.
Common questions about miglustat
Answers are fully visible for fast scanning and source review.
What is miglustat used for?
The selected product treats mild to moderate type 1 Gaucher disease in adults when enzyme replacement therapy is unsuitable, and progressive neurological manifestations of Niemann–Pick type C disease in adults and children.
Is miglustat an enzyme replacement therapy?
No. It is substrate-reduction therapy that decreases production of certain glycosphingolipids.
Can miglustat be taken with food?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.