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

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

Background

  • 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

Objective

  • To evaluate the efficacy of agalsidase‑α treatment relative to Fabrazyme treatment in treatment naïve patients with Fabry disease

Methods

  • Study design Head‑to‑head, prospective, multicentre study in Canada
  • Treatment regimen Treatment-naïve patients who met treatment guidelines / criteria for ERT initiation were randomly assigned to either agalsidase‑α or Fabrazyme
  • Patient numbers 132 (56 Fabrazyme, 76 agalsidase alpha), split of male and female not disclosed but no significant difference in baseline characteristics including gender was shown
  • Treatment duration CFDI is a study of ERT use in Fabry disease in Canada over 10 years
    • Patients were followed for a median time of 99 (5–123) months
  • Outcomes renal / cardiovascular / neurological events and death
    • Safety outcomes were not reported

Key data

There were more renal events in males receiving agalsidase‑α than males receiving Fabrazyme.

  • The rate of decline of estimated glomerular filtration rate (eGFR) tended to be lower in male patients on Fabrazyme than those on agalsidase-α (-1.98 vs. - 4.15 ml/min/ 1.73m2/year; p=0.09)
  • No difference in renal events or eGFR was seen between groups in females

 

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,2-4ensuring the long-term efficacy of the selected Fabry-specific therapy is essential

References

  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: https://ommbid.mhmedical.com/content.aspx? sectionid=225546984&bookid=2709 Accessed: January 2022.
  4. Ortiz A, Germain DP, Desnick RJ, et al. Mol Genet Metab. 2018;123(4):416–427
Fabrazyme API

MAT-BH-2300650 V1 NOV 23