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ALIMENTARY TRACT AND METABOLISM › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › Various alimentary tract and metabolism products
miglustat
A16AX06
Adult Dosing
- Type 1 Gaucher disease: 100 mg orally three times daily
- Niemann-Pick type C disease: 200 mg orally three times daily
- Renal impairment adjustment (CrCl 30-50 mL/min): 100 mg twice daily for Gaucher, 200 mg twice daily for Niemann-Pick type C
- Renal impairment adjustment (CrCl < 30 mL/min): Not recommended
Pediatric Dosing
- Type 1 Gaucher disease (pediatric patients 12 years and older): 100 mg orally three times daily
- Niemann-Pick type C disease (pediatric patients): Dosing adjusted for body surface area (BSA) relative to the adult 200 mg three times daily dose
Indications
- Treatment of mild-to-moderate type 1 Gaucher disease for whom enzyme replacement therapy is unsuitable or not therapeutic
- Progressive neurological manifestations in adult and pediatric patients with Niemann-Pick type C disease
Mechanism of Action
- Inhibits glucosylceramide synthase, the enzyme responsible for the first committed step in glycosphingolipid biosynthesis
- Acts as a substrate reduction therapy to decrease the rate of glycosphingolipid synthesis and accumulation
Contraindications
- Hypersensitivity to miglustat or any of its excipients
- Pregnancy
Adverse Reactions
- Diarrhea
- Weight loss
- Flatulence
- Abdominal pain
- Tremor
- Peripheral neuropathy
- Thrombocytopenia
Drug Interactions
- Coadministration with loperamide or anti-diarrheal agents may be required to manage treatment-induced diarrhea
- Potential additive effect in causing peripheral neuropathy or tremor with other neurotoxic medications
- No significant pharmacokinetic drug-interactions observed with CYP450 substrates
Curated Content: Needs Vetting Before Put to Clinical Use