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Learning from the Canadian Fabry Disease Initiative

Long‑term real‑world evidence confirms that Fabrazyme (agalsidase beta) reduces the risk of renal adverse outcomes related to Fabry disease in males1

Background

  • Conducting and analysing clinical studies in a rare disease is met with several challenges, including small patient numbers and short trial durations. In the case of Fabry disease, use of surrogate endpoints and the lack of records regarding supportive therapies such as, angiotensin converting enzyme inhibitors (ACEI), acetylsalicylic acid (ASA) and statin agents
  • The Canadian Fabry disease initiative (CFDI), which monitors outcomes of patients with Fabry disease who are treated with enzyme replacement therapy (ERT) provides valuable real‑world data to complement phase III trial data

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

  • In males with Fabry disease, Fabrazyme treatment is associated with fewer renal events over 99 months than agalsidase-α
  • Compared to agalsidase‑α, the use of Fabrazyme 1.0 mg/kg is associated with:
    • A trend of slower rate of decline in renal function in males
    • Significantly fewer renal events over 99 months of follow-up

Reference

  1. Sirrs SM, et al. Differential effects of agalsidase alfa and agalsidase beta in Fabry outcomes: 10 year outcomes from the Canadian Fabry Disease Initiative. J Inherit Metab Dis. 2018;41(Suppl 1):S37–219.
Fabrazyme API

MAT-BH-2300650 V1 NOV 23