Miglustat

How it works

It inhibits glucosylceramide synthase, reducing the synthesis of glycosphingolipids so that less accumulates within cells when the degrading enzyme is deficient.

Prescribing in practice

• Diarrhoea and weight loss are very common and can be limiting, often improved by dietary measures and dose adjustment.
• Peripheral neuropathy and tremor have been reported, so neurological function should be assessed before and during treatment.
• It may reduce sperm parameters, so men should use reliable contraception during and for a period after treatment.

Monitoring

Monitor body weight, gastrointestinal tolerance, neurological status including for peripheral neuropathy, and platelet counts where relevant.

Counselling the patient

• Gastrointestinal upset such as diarrhoea is common and may be eased by adjusting your diet.
• Report any numbness, tingling or tremor to your specialist.
• Men should use effective contraception during and after treatment as advised.

Evidence & guidelines

Miglustat is an established substrate reduction therapy for type 1 Gaucher disease and for neurological involvement in Niemann-Pick disease type C.