Kidney Week

Abstract: SA-PO0152

followME Fabry Pathfinders Registry: ≥Five-Year Kidney, Cardiac, and Multisystem Effectiveness in Patients on Migalastat

Session Information

• Tubular, Metabolic, and Systemic Noncystic Genetic Kidney Diseases
  October 24, 2026 | Location: Exhibit Hall A, Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: Genetic Diseases of the Kidneys

• 1202 Genetic Diseases of the Kidneys: Non-Cystic (Complex and Non-Cystic Monogenic)

Authors

• Lawson, Lee Ann, Amicus Therapeutics, Inc., Princeton, New Jersey, United States

Lee Ann Lawson, APRN, DNP, RN

• Azevedo, Olga, Cardiology Department, Reference Center of Lysosomal Storage Disorders, MetabERN member, Hospital Senhora da Oliveira, Guimarães, Portugal

Olga Azevedo, MD, PhD

• Linhart, Ales, Second Department of Internal Cardiovascular Medicine, First Faculty of Medicine, Charles University and General University Hospital, Prague, Czechia

Ales Linhart, MD

• Pisani, Antonio, Department of Public Health, Nephrology Unit, Federico II University Hospital, Naples, Italy

Antonio Pisani, MD, PhD

• Hughes, Derralynn, Royal Free London NHS Foundation Trust, and University College London, London, United Kingdom

Derralynn Hughes

• Yang, Haichen, Amicus Therapeutics, Inc., Princeton, New Jersey, United States

Haichen Yang, MD, MA, MBA

• Hiros, Jennifer, Amicus Therapeutics, Inc., Princeton, New Jersey, United States

Jennifer Hiros, MS

• Jain, Vipul, Amicus Therapeutics, Inc., Princeton, New Jersey, United States

Vipul Jain, MS, MBA

• Veleva-Rotse, Biliana O., Amicus Therapeutics, Inc., Princeton, New Jersey, United States

Biliana O. Veleva-Rotse, PhD

• Nordbeck, Peter, Internal Medicine I, University Hospital Würzburg, Würzburg, Germany

Peter Nordbeck, MD

Background

The ongoing, real-world followME Fabry Pathfinders registry (EUPAS20599) is evaluating natural history and treatment outcomes in patients (pts) with Fabry disease (FD). Here, we present ≥5-year effectiveness and safety data for migalastat.

Methods

Data were collected from pts ≥12 years of age with confirmed FD, migalastat-amenable GLA variants, an estimated glomerular filtration rate (eGFR) ≥30mL/min/1.73m² and who had received migalastat for ≥4.9 years.

Results

As of December 2025, 156 pts (64.1% male) were included (median migalastat exposure 6.5 years [range 4.9–8.7]). At enrollment, median age was 56.0 years (range 16–77). At baseline, 56 (35.9%) pts had eGFR (mL/min/1.73m²; Chronic Kidney Disease Epidemiology Collaboration equation) ≥90, 54 (34.6%) ≥60–<90, 10 (6.4%) ≥30–<60 and 36 (23.1%) had missing values; 39 had a proteinuria category ≥1, and 26 had missing values. Median albumin creatinine ratio was 26.8mg/g (95% confidence interval [95%CI] 17.7, 38.0; n=74). In females, baseline median eGFR was 90.8mL/min/1.73m² (n=43; range 57.7–128.1), with median annualized rate of change (ARC) −1.32mL/min/1.73m²/year (n=55; 95%CI −1.90, −0.65); baseline median left ventricular mass index (LVMi) was 85.6g/m² (n=34; range 47.0–232.5) and median LVMi ARC was −0.67g/m²/year (n=47; 95%CI −2.01, 1.64). In males, baseline median eGFR was 85.8mL/min/1.73m² (n=77; range 37.8–133.1), with median ARC −1.57mL/min/1.73m²/year (n=99; 95%CI −2.18, −1.14); baseline median LVMi was 132.3g/m² (n=55; range 54.3–296.2) and median LVMi ARC was −1.55g/m²/year (n=68; 95%CI −2.87, 0.23). Combined, median eGFR ARC was −1.52mL/min/1.73m²/year (n=154; 95%CI −2.01, −1.06) and median LVMi ARC was −0.95g/m²/year (n=115; 95%CI −2.01, 0.23). The incidence of renal, cerebrovascular, cardiac and composite Fabry-associated clinical events (FACEs) was 2.9, 8.8, 77.1 and 88.8 per 1000 patient-years, respectively. Correlations between baseline characteristics and FACEs will be investigated. Two pts (1.3%) had any treatment-related treatment-emergent serious adverse event; neither discontinued migalastat.

Conclusion

Long-term, real-world effectiveness of migalastat was demonstrated through stable renal function, preserved LVMi and low FACE incidence over median 6.5 years, with no new safety findings reported. These results are consistent with prior findings from followME and the migalastat clinical program.

Funding

• Commercial Support – This study was supported by Amicus Therapeutics, Inc. Medical writing/editorial assistance was provided by Lauren Wardle, MBio, at Amiculum, and was funded by Amicus Therapeutics, Inc.