This website contains promotional content and is intended for Healthcare Professionals based in the United Kingdom only. Some content is only relevant to HCPs practising in Great Britain (England, Scotland, Wales). This website is optimised for desktop use, and some features may perform differently on mobile devices.

Adverse event reporting can be found at the bottom of the page.

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

In an open-label, phase III extension study, early initiation of Fabrazyme was shown to maintain renal function over 54 months1


  • A 54-month observational study of Fabrazyme in 58 Classic Fabry patients (56 male, 2 female) with a mean age of 31 years at baseline showed:1
    • Median eGFR (-1.0mL/min per 1.73 m2/yr) was maintaned within the normal range over 54 months
    • Patients without significant renal involvment (proteinuria ≤ 1g/day) (n=42) at baseline benefited the most

Key data

Sustained, long-term renal stabilisation (media eGFR) after 54 months of Fabrazyme therapy in patients with Fabry disease.

Adapted from Germain DP et al. Am Soc Nephrol. 2007.

Relevance to clinical practice

  • Timely initiation of Fabry-specific treatment, such as Fabrazyme, prior to the development of significant organ damage may help to optimise patient outcomes
  • Fabry disease is progressive and often life-limiting,2-4 ensuring the long-term efficacy of the selected Fabry-specific therapy is essential


  1. Germain DP et al. J Am Soc Nephrol.2007;18(5);1547-1557. 
  2. Germain DP. J Am Soc Nephrol. 2002;13(suppl 2):S150–S153.  
  3. Desnick RJ, Loannou YA, Eng CM. Chapter 150: α-Galactosidase A Deficiency: Fabry Disease. In: Valle D, Beaudet AL, Vogelstein B, eds. The Online Metabolic and Molecular Basis of Inherited Disease. New York, NY: McGraw Hill; 2014. Available at: sectionid=225546984&bookid=2709 Accessed: January 2022.
  4. Ortiz A, Germain DP, Desnick RJ, et al. Mol Genet Metab. 2018;123(4):416–427. 

MAT-XU-2301503 (v2.0) Date of preparation: October 2023