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Zavesca (MIGLUSTAT)

Zavesca works by inhibiting the enzyme glucosylceramide synthase, which is involved in the production of a toxic lipid that accumulates in cells of patients with certain lysosomal storage disorders.

Zavesca (MIGLUSTAT) is a small molecule glucosylceramide synthase inhibitor developed by ACTELION PHARMS LTD and currently owned by Actelion. It targets lysosomal alpha-glucosidase and is used to treat various lysosomal storage disorders, including Gaucher's disease, Late-onset Pompe disease, and Niemann-Pick disease type C. Zavesca was FDA-approved in 2003 and is now available as a generic medication. The commercial status of Zavesca is off-patent, with four generic manufacturers available. Key safety considerations include monitoring for gastrointestinal side effects and potential interactions with other medications.

At a glance

Mechanism of action

Type Gaucher disease is caused by functional deficiency of glucocerebrosidase, the enzyme that mediates the degradation of the glycosphingolipid glucosylceramide.Miglustat functions as competitive and reversible inhibitor of the enzyme glucosylceramide synthase, the initial enzyme in series of reactions which results in the synthesis of most glycosphingolipids.miglustat helps reduce the rate of glycosphingolipid biosynthesis so that the amount of glycosphingolipid substrate is reduced to level which allows the residual activity of the deficient glucocerebrosidase enzyme to be more effective (substrate reduction therapy). In vitro and in vivo studies have shown that miglustat can reduce the synthesis of glucosylceramide-based glycosphingolipids.

Approved indications

• Glucosylceramide beta-glucosidase deficiency
• Lateonset Pompe disease
• Niemann-Pick disease, type C

Common side effects

• Diarrhea
• Weight loss
• Abdominal Pain
• Flatulence
• Nausea
• Vomiting
• Headache
• Tremor
• Dizziness
• Leg cramps
• Visual Disturbance
• Thrombocytopenia

Key clinical trials

• A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease (PHASE2)
• A Global Prospective Observational Registry of Patients With Pompe Disease
• A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) (PHASE3)
• A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 (PHASE3)
• Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation (PHASE2,PHASE3)
• ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD (PHASE3)
• First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 (PHASE1,PHASE2)
• Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) (PHASE3)

Patents

Primary sources

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Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Zavesca CI brief — competitive landscape report
• Zavesca updates RSS · CI watch RSS
• AstraZeneca portfolio CI