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Fabrazyme® is an enzyme replacement therapy (ERT) indicated for use in adults, children and adolescents aged 8 years and older with Fabry disease.1 In this page we specifically explore the effect of Fabrazyme on renal manifestations in Fabry disease

Background

Fabry disease starts early in the kidney and is a silent threat. Progressive accumulation of globotriaocylceramide (GL-3) leads to cellular changes and histological damage.2 Fabry nephropathy shows similarities to other proteinuric nephropathies of metabolic origin.3 Once the mechanisms leading to tissue injury are activated, the progression of Fabry nephropathy becomes irreversible.4,5

Treatment with Fabrazyme has been shown to preserve renal function in patients and reduce the risk of adverse renal outcomes in patients with Fabry disease

  • In a meta‑analysis of data from ten studies, Fabrazyme was shown to preserve renal function in patients with classic Fabry disease compared to untreated patients6
  • 10-year outcomes data from the Canadian Fabry Disease Initiative (CFDI) showed a numerical difference in renal events and rate of renal decline between Fabrazyme (1.0mg/kg EOW) and agalsidase-alfa (0.2mg/kg EOW)7
  • Early initiation of Fabrazyme has been shown to maintain long-term renal function over 54 months8,9

Fabrazyme has a well characterised safety and tolerability profile

  • The most common adverse events reported were mild-to-moderate infusion-associated reactions. At least one infusion-associated reaction was experienced by 67% of patients1
  • Percentage of patients experiencing infusion-associated reactions (IARs) decreased from 19/29 during the Phase III trial to 7/51 after 30–36 months of treatment10

Quantifying the effect of Fabrazyme (agalsidase beta) on the preservation of renal function in Fabry disease

In a meta‑analysis of data from ten studies, Fabrazyme was shown to preserve renal function in patients with classic Fabry disease compared to untreated patients1

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Learning from the Canadian Fabry Disease Initiative

10-year outcomes data from the Canadian Fabry Disease Initiative (CFDI) showed a numerical difference in renal effects and rate of renal decline between Fabrazyme (1.0mg/kg EOW) and agalsidase-alfa (0.2mg/kg EOW)1

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Understanding the impact of Fabrazyme (agalsidase beta) on and long-term renal function

Early initiation of Fabrazyme has been shown to maintain long-term renal function over 54 months1,2

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References

  1. FABRAZYME Greater Gulf Summary of Product Characteristics Dec 2018.
  2. Waldek S and Ferriozi S. BMC Nephrol. 2014;15:72.
  3. Ortiz A and Sanchez-Niño MD. Port J Nephrol Hypert. 2017;31(3):200–6.
  4. Ortiz A, et al. Mol Genet Metab. 2018;123(4):416–27.
  5. Van der Veen SJ, et al. Mol Genet Metab. 2022;135(2):163–69
  6. Ortiz A, et al. Clin Kidney J. 2020;14(4):1136–46
  7. Sirrs SM, et al. J Inherit Metab Dis. 2018;41(Suppl 1):S37–S219
  8. Wanner et al. Clin J Am Soc Nephrol. 2010 Dec;5(12):2220-228
  9. Germain DP et al. J Am Soc Nephrol. 2007;18(5);1547-1557.
  10. Wilcox WR, et al. Am J Hum Genet. 2004;75(1):65–74.
Fabrazyme API

MAT-BH-2300650 V1 Nov 23