Kidney Week

Abstract: INFO16-FR

followME Registry Trial in Progress: A Prospective Observational Study to Evaluate Treatment Effects on Long-Term Safety, Effectiveness, and Quality of Life in Adults with Worsening Fabry Disease

Session Information

• Informational Posters - 2
  October 23, 2026 | Location: Exhibit Hall A, Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: Genetic Diseases of the Kidneys

• No subcategory defined

Authors

• Mehta, Ankit, Baylor University Medical Center at Dallas, Dallas, Texas, United States

Ankit Mehta, MD

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

Jennifer Hiros, MS

• Wade, Alyson, Amicus Therapeutics, Inc., Princeton, New Jersey, United States

Alyson Wade

• Goker-Alpan, Ozlem, Lysosomal and Rare Disorders Research and Treatment Center, Inc., Fairfax, Virginia, United States

Ozlem Goker-Alpan, MD

• Rutecki, Jasmine L., Amicus Therapeutics, Inc., Princeton, New Jersey, United States

Jasmine L. Rutecki

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

Biliana O. Veleva-Rotse, PhD

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

Haichen Yang, MD, MA, MBA

Description

Migalastat is an oral small molecule chaperone therapy approved for treatment of adults with Fabry disease (FD) and amenable GLA variants in the US. The global, ongoing, real-world followME Fabry Pathfinders registry (AT1001-030; EUPAS205999) is designed to evaluate natural history and current real-world treatments in patients with FD, focusing on migalastat. This separate, prospective, multicenter observational study (AT1001-030X; NCT06906367), focusing on adults with worsening FD, aims to evaluate natural history and treatment outcomes, including long-term effectiveness, safety and health-related quality of life (HRQoL).

Patients (≥18 years) with amenable GLA variants and estimated glomerular filtration rate (eGFR) ≥30mL/min/1.73m² are eligible to enroll into migalastat, enzyme replacement therapy (ERT) or untreated groups. Enrollment is open to patients experiencing decline based on any of the following: annualized rate of decline in eGFR calculated by the Chronic Kidney Disease Epidemiology equation (CKD-EPI) ≥2mL/min/1.73m² for ≤2 years prior to enrollment, microalbuminuria/macroalbuminuria/proteinuria any time prior to or at enrollment, or males with the classic FD phenotype. Patients in the migalastat or ERT groups initiated treatment ≤24 months prior to enrollment. All patients will be followed for up to 5 years after enrollment. Treatment switching is permitted; patients in the untreated group who switch to migalastat or ERT <6 months from enrollment will be considered part of the respective treatment group.

The primary endpoint evaluates annualized rate of change (ARC) eGFR_CKD-EPI over time, from enrollment, in migalastat-treated vs untreated patients who have risk factors for eGFR decline. Secondary effectiveness endpoints include time from enrollment to first Fabry-associated clinical event (FACE; cardiac, cerebrovascular and renal events) and death due to FACEs between migalastat-treated and untreated patients, and ARC eGFR_CKD-EPI over time from treatment start between migalastat-treated and ERT-treated patients. Safety endpoints include incidence of serious adverse events. HRQoL will be assessed using patient-reported outcomes and health preference measures.

AT1001-030X enrollment is ongoing.

Acknowledgment

Medical writing/editorial assistance was provided by Lauren Wardle, MBio, at Amiculum, and was funded by Amicus Therapeutics, Inc. As of April 27, 2026, Amicus Therapeutics, Inc. is a wholly owned subsidiary of BioMarin Pharmaceutical Inc.

Funding

• This study was supported by Amicus Therapeutics, Inc. As of April 27, 2026, Amicus Therapeutics, Inc. is a wholly owned subsidiary of BioMarin Pharmaceutical Inc.