Lecture Notes

Overview

• Generic Name: Miglustat

• Trade Name: Zavesca

• Drug Class: Substrate reduction therapy (SRT); glucosylceramide synthase inhibitor

• Administration: Oral capsules

Indications

• Gaucher disease type 1 (mild to moderate severity) for patients unable or unwilling to receive enzyme replacement therapy (ERT).

• Niemann–Pick Disease Type C (NPC), to slow neurological progression.

Mechanism of Action

• Miglustat is an iminosugar (N-alkylated imino sugar) derivative.

• Acts by competitively inhibiting glucosylceramide synthase enzyme.

• Reduces glycosphingolipid accumulation by decreasing substrate production.

Pharmacokinetics

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