Fabry disease is a progressive, X-linked lysosomal disorder caused by reduced or absent α-galactosidase A activity due to GLA variants. The effects of migalastat were examined in a cohort of 125 Fabry patients with migalastat-amenable GLA variants in the followME Pathfinders registry (EUPAS20599), an ongoing, prospective, patient-focused registry evaluating outcomes for current Fabry disease treatments. We report annualised estimated glomerular filtration rate (eGFR) and Fabry-associated clinical events (FACEs) in a cohort of patients who had received ≥3 years of migalastat treatment in a real-world setting. As of August 2022, 125 patients (60% male) had a mean migalastat exposure of 3.9 years. At enrolment, median age was 58 years (males, 57; females, 60) with a mean eGFR of 83.7 mL/min/1.73 m2 (n = 122; males, 83.7; females, 83.8) and a median left ventricular mass index of 115.1 g/m2 (n = 61; males, 131.2; females, 98.0). Mean (95% confidence interval) eGFR annualised rate of change in the overall cohort (n = 116) was -0.9 (-10.8, 9.9) mL/min/1.73 m2/year with a similar rate of change observed across patients with varying levels of kidney function at enrolment. Despite population age and baseline morbidity, 80% of patients did not experience a FACE during the mean 3.9 years of migalastat exposure. The incidence of renal, cardiac, and cerebrovascular events was 2.0, 83.2, and 4.1 events per 1000 patient-years, respectively. These data support a role of migalastat in preserving renal function and multisystem effectiveness during ≥3 years of migalastat treatment in this real-world Fabry population.

2nd Department of Internal Cardiovascular Medicine 1st Faculty of Medicine Charles University and General University Hospital Prague Czech Republic

Amicus Therapeutics Inc Princeton New Jersey USA

Cardiology Department Reference Center on Lysosomal Storage Disorders Hospital Senhora da Oliveira Guimarães Portugal

Department of Endocrinology and Clinical Nutrition University Hospital Zurich and University of Zurich Zurich Switzerland

Department of Medical Genetics University of British Columbia Vancouver British Columbia Canada

Department of Medicine Dalhousie University Halifax Nova Scotia Canada

Department of Medicine Hôpital du Sacré Coeur University of Montréal Montréal Quebec Canada

Department of Public Health Nephrology Unit Federico 2 University Hospital Naples Italy

Division of Nephrology and Dialysis Department of Medicine 3 Medical University of Vienna Vienna Austria

Inherited Kidney Diseases Nephrology Department Fundació Puigvert Institut d'Investigacions Biomèdiques Department of Medicine Universitat Autònoma de Barcelona Barcelona Spain

Internal Medicine D Department of Nephrology Hypertension and Rheumatology Interdisciplinary Fabry Center Münster University Hospital Münster Münster Germany

Lysosomal Storage Disorders Unit Royal Free London NHS Foundation Trust and University College London London UK

M A G 1 C Clinic Ltd Calgary Alberta Canada

Northern Care Alliance NHS Foundation Trust Salford UK

University Hospital Würzburg Würzburg Germany

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