Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study

Clin Exp Nephrol. 2020 Feb;24(2):157-166. doi: 10.1007/s10157-019-01810-w. Epub 2019 Dec 30.


Background: Fabry disease is a progressive X-linked lysosomal disorder. In this subgroup analysis of the global phase III ATTRACT study, the efficacy and safety of oral migalastat, a pharmacologic chaperone, were investigated in Japanese patients with Fabry disease.

Methods: Patients were randomly assigned to receive migalastat (150 mg every other day) or to continue biweekly enzyme replacement therapy infusions (ERT; agalsidase alfa 0.2 mg/kg or agalsidase beta 1.0 mg/kg) for 18 months followed by a 12-month open-label extension during which all patients received migalastat. End points included glomerular filtration rate (estimated and measured), left ventricular mass index (LVMi), composite clinical outcomes, leukocyte alpha-galactosidase A activity, plasma globotriaosylsphingosine (lyso-Gb3), and safety.

Results: Data from 7 Japanese patients (migalastat, 5; ERT, 2), mean age 55 years, with high disease burden, were analyzed. All patients in the migalastat group completed the open-label comparison and extension periods. At 18 months, efficacy in the Japanese patient population was similar to that in the overall ATTRACT population. Migalastat treatment increased leukocyte alpha-galactosidase A activity, stabilized renal function, and decreased LVMi. Plasma lyso-Gb3 levels remained low and stable. Additionally, the long-term extension study showed that efficacy of migalastat was maintained for up to 48 months. Migalastat was safe and well tolerated in the Japanese patients, as in the overall ATTRACT population.

Conclusion: Migalastat can be used to treat Japanese patients with Fabry disease with GLA mutations amenable to migalastat according to the dosage and administration approved in other countries.

Trial registration numbers: ClinicalTrials.gov, NCT01218659 and NCT02194985.

Keywords: Alpha-galactosidase; Enzyme replacement therapy; Fabry disease; Migalastat; Mutation; Pharmacologic chaperone.

Publication types

  • Clinical Trial, Phase III
  • Multicenter Study
  • Randomized Controlled Trial

MeSH terms

  • 1-Deoxynojirimycin / administration & dosage
  • 1-Deoxynojirimycin / adverse effects
  • 1-Deoxynojirimycin / analogs & derivatives*
  • Administration, Oral
  • Adult
  • Fabry Disease / drug therapy*
  • Fabry Disease / enzymology
  • Fabry Disease / genetics
  • Female
  • Genetic Predisposition to Disease
  • Humans
  • Japan
  • Male
  • Middle Aged
  • Mutation
  • Prospective Studies
  • Time Factors
  • Treatment Outcome
  • alpha-Galactosidase / genetics
  • alpha-Galactosidase / metabolism


  • 1-Deoxynojirimycin
  • migalastat
  • GLA protein, human
  • alpha-Galactosidase

Associated data

  • ClinicalTrials.gov/NCT01218659
  • ClinicalTrials.gov/NCT02194985