miglustat: clinical use

Prescribing considerations

• Restrict use in type 1 Gaucher disease to adults with mild to moderate disease for whom enzyme replacement therapy is unsuitable.
• Use under specialist supervision and review clinical benefit regularly in Niemann–Pick type C disease.
• Anticipate frequent gastrointestinal intolerance; consider individualised dietary measures, administration between meals and symptomatic management.
• Assess renal function because systemic exposure rises as renal clearance falls; use in severe renal impairment is not recommended.
• Evidence is limited in hepatic impairment and in very young children with Niemann–Pick type C disease.

Contraindications and cautions

• Hypersensitivity to miglustat or any capsule excipient.
• Avoid during pregnancy and breastfeeding.
• Severe renal impairment is not a formal section 4.3 contraindication, but use is not recommended.

Monitoring

• Baseline and repeat neurological examination for peripheral neuropathy.
• Platelet count in type 1 Gaucher disease and Niemann–Pick type C disease.
• Vitamin B12 in type 1 Gaucher disease.
• Weight and persistent gastrointestinal symptoms.
• Growth in children and adolescents with Niemann–Pick type C disease.
• Renal function and continued clinical benefit.

Clinical pharmacology

Miglustat inhibits glucosylceramide synthase. It is rapidly absorbed, does not bind to plasma proteins and is eliminated mainly unchanged in urine; renal impairment therefore increases exposure.

Formulation and product differences

• The selected product is a hard gelatin capsule intended to be swallowed whole.
• It is essentially sodium-free.
• The selected capsule is licensed for Gaucher disease and Niemann–Pick type C disease; other miglustat-branded products may have different formulations and licensed roles and should not be assumed interchangeable.

Capsule

Route: Oral

Strengths: 65 mg, 100 mg