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Fabrazyme® (agalsidase beta) prescribing information GB
Fabrazyme® (agalsidase beta) prescribing information NI

A meta‑analysis of data from ten studies suggests that Fabrazyme preserves renal function in patients with classic Fabry disease compared to untreated patients1


  • Glomerular filtration rate (GFR) decline indicates the progression of nephropathy in Fabry disease
  • The effect of Fabrazyme treatment on GFR decline is not well established as the strength of the data from individual studies is limited by their small populations
  • This publication reports results from a meta‑analysis of ten studies on classic Fabry disease using patient‑level data, which allows inclusion of patients on an individual basis and the adjustment for patient characteristics across settings


  • To determine the long‑term effect of Fabrazyme on eGFR 


  • Four clinical trials and six studies from a systematic literature review (SLR). The SLR were restricted to patients with classic Fabry disease who met the eligibility criteria from phase III and phase IV Fabrazyme trials
  • 315 patients were included in total, including 161 patients treated with Fabrazyme
  • Analysis was undertaken with summary measures approach (SMA)
    • Step 1: an estimated rate of change in eGFR per year was obtained using linear regression on each patient individually
    • Step 2: the estimated slope coefficients from Step 1 were modelled using quantile regression with covariates of interest
  • ERT was given for a median of 65 months (range, 13–69 months)

Key data

Fabrazyme‑treated patients experienced a slower median eGFR decrease than untreated patients.

Relevance to clinical practice

  • This meta‑analysis of 10 studies on patients with classic Fabry disease suggests that treatment with Fabrazyme slows down renal decline
  • Patients conserved their renal function better than untreated patients as assessed by eGFR and uPCR
  • Starting treatment earlier may delay the time to end stage renal disease compared with patients who are left untreated


  1. Ortiz A, et al. Clin Kidney J. 2020;14(4):1136–46.

MAT-XU-2301502 (v2.0) Date of preparation: October 2023