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Think Fabrazyme first, 20+ years of trusted treatment
Our understanding of Fabry disease has changed dramatically over the past 20 years.
Our commitment to providing a trusted treatment hasn’t.
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Patricia, Ammeris, and Shemary, living with Fabry disease, and their family, CJ, Eric, and Eisha
Since the launch of Fabrazyme over 20 years ago, our understanding of Fabry disease has advanced significantly. These advancements have shed light on how various patient subgroups can be affected by Fabry disease and how treatment with Fabrazyme can help patients, regardless of disease severity, enzyme activity, or genetic variant.1-4
We're not done yet: Real-world data collection remains underway as we continue to gain a deeper understanding of Fabry disease and how we can further improve patient outcomes.
Think Fabrazyme first, think a long-term reduction in severe clinical events1
In a 10-year follow-up study, 52 patients with Fabry disease were evaluated on the basis of severe clinical events, renal function, and cardiac structure1:
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of patients were alive at the end of the 10-year study period1
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of patients did not experience any severe renal, cardiac, or cerebrovascular clinical event1
10 patients (19%) reported 16 severe clinical events, 8 during the clinical trial (initial 54 months) and 8 during the long-term observation interval. The most common severe event reported was stroke, followed by severe renal events and cardiac events.
The safety of Fabrazyme has been assessed in 4 clinical trials involving 162 patients with over 473 patient-years of experience2,5
Meta-analysis: Effect of Fabrazyme on annualized change in eGFR6
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The meta-analysis evidence base included 4 Sanofi studies and 6 studies from a systematic literature review, restricted to patients with classic Fabry disease meeting the eligibility criteria from Phases 3 and 4 trials.6
Linear regression of eGFR of patient-level data was used to derive annualized eGFR slopes: Patients treated with Fabrazyme vs untreated patients.6
315 total classic phenotype patients with Fabry disease:
- 153 untreated patients
- 133 Fabrazyme-treated patients
- 29 patients untreated for 6 months then switched to Fabrazyme treatment
Baseline patient characteristics
| Treated Patients | Untreated Patients | |
| Mean Age (years) | 39.9 | 34.6 |
| Males (%) | 82.7 | 92.9 |
| Females (%) | 17.3 | 7.1 |
| Mean eGFR (ml/min/1.73m2) | 85.5 | 88.7 |
| Mean UPCR (g/g) | 1.18 | 0.82 |
eGFR=estimated glomerular filtration rate; UPCR=urine protein/creatinine ratio.
Meta-analysis results
Principal analyses using linear regression of eGFR of patient-level data showed a difference in the mean slope of eGFR among Fabrazyme-treated vs untreated patients6,7
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Unadjusted Model6
ERT-treated patients (n=161):
-2.43 mL/min/1.73 m2/year
Untreated patients (n=182):
-3.47 mL/min/1.73 m2/year
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Adjusted Model (proteinuria, gender)6
ERT-treated patients (n=161):
-1.01 mL/min/1.73 m2/year
Untreated patients (n=182):
-3.47 mL/min/1.73 m2/year
When adjusted for imbalances in gender and proteinuria, Fabrazyme-treated patients experienced a slower median eGFR decrease than comparable untreated patients:
- 2.46 mL/min/1.73 m2/year slower [95% CI 0.63–4.29; p=0.0087]6
- eGFR decline in a normal population: -1.0 mL/min/1.73 m2/year7
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Roland, a real Fabrazyme patient for over 20 years
“Having been diagnosed more than [41] years ago, I’d like to think I have acquired a little wisdom about managing this disease to pass along. First, patients shouldn’t delay in dealing with Fabry. From my experience, the best way to face this disease is by being proactive as early as possible.”
Indication
References: 1. Germain DP et al. J Med Genet. 2015;52(5):353-358. 2. Fabrazyme (agalsidase beta). Prescribing Information. Sanofi. 3. Hopkin RJ et al. Pediatr Res. 2008;64(5):550-555. 4. Wilcox WR et al. Mol Genet Metab. 2008;93(2):112-128. 5. Data on file. Sanofi. 6. Ortiz A et al. Clin Kidney J. 2020;14(4):1136-1146. 7. Baba M et al. PLoS One. 2015;10(6):e0129036.
