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Miglustat

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Abstract

  • ▴ Miglustat is an orally administered ceramide glucosyltransferase inhibitor which prevents the lysosomal accumulation of glucocerebroside that occurs in patients with Gaucher’s disease.

  • ▴ In noncomparative trials in patients with type 1 Gaucher’s disease, miglustat (50 or 100mg three times daily) for 6–12 months significantly reduced baseline liver and spleen volumes. At both 6 and 12 months, the reductions in organ volumes were greater with the higher dosage.

  • ▴ Miglustat 50 or 100mg three times daily for 6–12 months had no significant effect on haemoglobin concentrations. Baseline platelet counts were not significantly improved by either dosage at 6 months, although the higher dosage significantly increased platelet counts at 12 months.

  • ▴ In an open extension phase, patients continued to show further reductions in organ volume as well as significant improvements in haematological parameters at 24 and 36 months.

  • ▴ In a 6-month randomised study in patients with type 1 Gaucher’s disease who had previously received long-term enzyme replacement therapy (ERT), liver volume reduction was greater with miglustat plus ERT than with ERT alone.

  • ▴ Diarrhoea and weight loss were the most frequent adverse events associated with miglustat therapy. Fine tremor has been reported in approximately 30% of miglustat-treated patients.

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Notes

  1. Use of tradenames is for product identification purposes only and does not imply endorsement.

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Authors and Affiliations

  1. Adis International Limited, 41 Centorian Drive, Private Bag 65901, Mairangi Bay, Auckland, 1311, New Zealand

    Paul L. McCormack & Karen L. Goa

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  1. Paul L. McCormack
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  2. Karen L. Goa
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McCormack, P.L., Goa, K.L. Miglustat. Drugs 63, 2427–2434 (2003). https://doi.org/10.2165/00003495-200363220-00006

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  • DOI: https://doi.org/10.2165/00003495-200363220-00006

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