miglustat (Acetlion, Zavesca, Opfolda)

Introduction

Tradename: Acetlion (FDA approved 2003)

Indications

• Gaucher disease
  • patients who cannot receive enzyme therapy (FDA)
• Pompe disease in combination with cipaglucosidase alfa-atga (Pombiliti)

Adverse effects

GI distress, peripheral neuropathy, weight loss

Mechanism of action

• glucosylceramide synthase inhibitor

More general terms

• enzyme inhibitor
• metabolic agent (metabolic modifier)

Database

• PubChem: http://pubchem.ncbi.nlm.nih.gov/summary/summary.cgi?cid=51634