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Miglustat: A Review of Its Use in Niemann-Pick Disease Type C

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Abstract

Miglustat (Zavesca®, Brazaves®), a small iminosugar molecule that reversibly inhibits glycosphingolipid synthesis, is the only disease-specific drug approved for the treatment of progressive neurological manifestations of Niemann-Pick disease type C (NP-C) in adult and paediatric patients. NP-C is a rare, autosomal-recessive lipid storage disorder characterized by impaired intracellular lipid trafficking and progressive neurological symptoms leading to premature death. In a randomized clinical trial, long-term extension studies and a retrospective observational cohort study, treatment with oral miglustat stabilized key neurological manifestations of NP-C (including horizontal saccadic eye movement peak velocity, ambulation, manipulation, language and swallowing) in paediatric and adult patients with the disease. The therapeutic effects of miglustat in stabilizing or slowing disease progression have been confirmed in other reports in the clinical experience setting. The primary tolerability issues associated with miglustat are mild to moderate gastrointestinal effects (e.g. diarrhoea, flatulence and abdominal pain/discomfort) and weight loss, which usually occur during initial therapy and are generally manageable. In the absence of a cure, miglustat is a valuable agent to reduce the progression of clinically relevant neurological symptoms in paediatric and adult patients with NP-C, which is considered a significant achievement in the treatment of this disease.

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Disclosure

The preparation of this review was not supported by any external funding. During the peer review process, the manufacturer of the agent under review was offered an opportunity to comment on this article. Changes resulting from comments received were made by the author on the basis of scientific and editorial merit.

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    Katherine A. Lyseng-Williamson

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Correspondence to Katherine A. Lyseng-Williamson.

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The manuscript was reviewed by: B. Bembi, Regional Coordinator Centre for Rare Diseases, University Hospital Santa Maria della Misericordia, Udine, Italy; W.L. Hwu, Department of Pediatrics and Medical Genetics, National Taiwan University Hospital and National Taiwan University College of Medicine, Taipei, Taiwan; M. Pineda, Pediatric Neurology Centre Medic, Hospital Sant Joan de Deu i Clinica Teknon, Barcelona, Spain; M. Walterfang, Melbourne Neuropsychiatry Centre, University of Melbourne and Melbourne Health, Parkville, Australia.

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Lyseng-Williamson, K.A. Miglustat: A Review of Its Use in Niemann-Pick Disease Type C. Drugs 74, 61–74 (2014). https://doi.org/10.1007/s40265-013-0164-6

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