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Abstract: SA-PO421

Long-Term Renal Outcomes with Migalastat in Patients with Fabry Disease: Results from Phase 3 Trials

Session Information

Category: Genetic Diseases of the Kidneys

  • 1002 Genetic Diseases of the Kidneys: Non-Cystic

Authors

  • Bichet, Daniel, University of Montreal, Montreal, Quebec, Canada
  • Wallace, Eric L., University of Alabama at Birmingham, Birmingham, Alabama, United States
  • Hughes, Derralynn, University College London, Royal Free London NHS Foundation Trust, London, United Kingdom
  • Skuban, Nina, Amicus Therapeutics Inc, Cranbury, New Jersey, United States
  • Lin, Brian, Amicus Therapeutics Inc, Cranbury, New Jersey, United States
  • Feldt-Rasmussen, Ulla, Rigshospitalet, Copenhagen University Hospital, Copenhagen, Denmark
  • Schiffmann, Raphael, Baylor Research Institute, Dallas, Texas, United States
Background

Fabry disease is a multisystem disorder of which nephropathy is an important feature, with untreated male patients experiencing a decline in glomerular filtration rate (GFR) of up to 12.2 mL/min/1.73 m2 per year. Here, we assessed long-term changes in renal function in patients with Fabry disease and amenable mutations who were treated with migalastat in the phase 3 FACETS (NCT00925301) and ATTRACT (NCT01218659) trials.

Methods

In FACETS, enzyme replacement therapy (ERT)-naive patients were randomly assigned to 6 mo of double-blind miglastat 150 mg every other day (QOD) or placebo, followed by an additional 18 mo of migalastat. In ATTRACT, patients receiving ERT were randomly assigned to 18 mo of miglastat 150 mg QOD or continued ERT, followed by an additional 12 mo of miglastat. Patients could continue migalastat in separate long-term extension trials. Renal outcomes were evaluated using the beginning of migalastat treatment as the baseline, and were analyzed by sex, baseline eGFRCKD-EPI, and baseline 24-h urine protein.

Results

Overall, mean (range) duration of migalastat exposure was 4.4 (0.1-7.8) years. Mean (SD) baseline eGFRCKD-EPI (mL/min/1.73 m2) was 93.1 (24.4) in FACETS and 89.0 (20.4) in ATTRACTS. Annualized rates of change in eGFRCKD-EPI by baseline variables are shown in the table.

Conclusion

Data suggest that patients who received long-term treatment with migalastat experienced a rate of eGFR decline comparable to published data with ERT. In addition, starting treatment early may prevent irreversible renal progression.

Funding

  • Commercial Support –