Miglustat
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 :
Oral:
<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.