Drug Index


Mechanism :

Miglustat competitively and reversibly inhibits the enzyme needed to produce glycosphingolipids and decreases the rate of glycosphingolipid glucosylceramide formation.

Indication :

  • Gaucher disease (adults only)
  • Off-label: Niemann-Pick Type C disease

Contraindications :

Hypersensitivity to miglustat or any component of the formulation; women who are or may become pregnant.

Dosing :

<12 years
BSA >1.25 m²: 200 mg 3 times daily.
BSA >0.88 to 1.25 m²: 200 mg 2 times daily.
BSA >0.73 to 0.88 m²: 100 mg 3 times daily.
BSA >0.47 to 0.73 m²: 100 mg 2 times daily.
BSA ≤0.47 m²: 100 mg once daily.
≥12 years
200 mg 3 times daily.

Adverse Effect :

Headache, dizziness, weight loss, diarrhea, abdominal pain, vomiting, tremor, weakness, leg cramps, visual disturbance, unsteady gait, paresthesia, abdominal distension, thrombocytopenia, back pain.

Interaction :

No known significant interactions.

Hepatic Dose :

No dose adjustments are recommended.
02/16/2021 04:43:21 Miglustat
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