Reduced Incidence of Stroke with Agalsidase beta in Patients with Fabry Disease : An Analysis from Fabry Registry

Background ¹²³

• Fabry disease can cause glycosphingolipid accumulation damages vascular endothelium, heart, kidneys, and brain, creating a strong predisposition to cerebrovascular events including TIA and stroke.

• Recent clinical evidence highlights that Fabry disease is an important, potentially treatable cause of early and cryptogenic stroke, underscoring the need for timely diagnosis and targeted therapy to mitigate longterm cerebrovascular risk.

• Presented below is a summary of the Fabry Registry analysis evaluating how treatment with agalsidase beta influences the incidence and characteristics of stroke in patients with Fabry disease.

Methods⁴

• Analysis of Fabry Registry Data, collected from 2001 to October 2024

• Agalsidase beta-treated and untreated Fabry disease patients matched 1:1 by sex, phenotype, and index age

Incidence of stroke⁴

Agalsidase beta-treated
patients (N=1626)

Untreated patients
(N=1626)

Number of events/person-year

0.61%

1.48%

Median age at first stroke (Years)

54.0

44.3

Median time of follow-up (years)

5.0

2.0

Incidence rate per 1000 person-years

6.19

14.86

New evidence from real-world Fabry registry data suggests⁴

• Strokes are common in Fabry disease, particularly in the classic phenotype, and occur at a younger age in Untreated vs Treated patients

• The use of agalsidase beta for at least one year was significantly associated with a 71% lower risk of stroke in the sample studied.