Fabrazyme is the first and only Fabry treatment approved for ages 2 and up regardless of genetic variant, disease severity, or gender, with over 20 years of real-world experience1-5
Fabrazyme is the first and only ERT indicated for patients aged 2 years and older with confirmed Fabry disease, with efficacy and safety outcomes in pediatric patients consistent with those seen in adults.1
Fabrazyme has over 20 years of global real-world experience, being chosen for over 6000 patients worldwide regardless of their genotype, gender, or disease severity.1,6,7
Fabrazyme showed a beneficial effect in females with Fabry disease and maintained normal kidney function as shown in a novel long-term trial of Fabrazyme outcomes in a large cohort (n=86) of females with Fabry disease.8,a
Fabrazyme maintained efficacy regardless of presence of antibodies.9 Approximately 83% (110 of 133) of adult patients receiving Fabrazyme developed antibodies:
- 77% (102/133) of patients developed NAb that inhibited in vitro Fabrazyme catalytic activity, which declined over time
- 6% (8/133) of patients developed NAb that inhibited cellular uptake1
Over 90% of adult and pediatric patients achieved and maintained normal plasma GL-3 levels irrespective of developing antibodies.1
In a retrospective study of 134 clinical trial patients, no correlation was found between titers of anti-α-GAL IgG antibodies and the onset of clinical events or rate of change in eGFR during treatment.9
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Michelle, a real Fabrazyme patient
“At the time of my diagnosis, some of the medical field considered women to be only carriers of Fabry and usually asymptomatic. All these years later, now we know that women are not just carriers; many of us have multiple symptoms.”
Indication
References: 1. Fabrazyme (agalsidase beta). Prescribing Information. Sanofi. 2. Vedder A et al. Genet Metab. 2008;94(3):319–325. 3. Lee K et al. Glycobiology. 2003;13(4):305-313. 4. Elfabrio (pegunigalsidase alfa-iwxj). Prescribing Information. Chiesi Farmaceutici S.p.A. 5. Galafold (migalastat). Prescribing Information. Amicus Therapeutics, Inc. 6. Data on file. Genzyme Corporation. 7. Data on file. Sanofi. Based on estimated patient numbers based on publicly published revenue as of September 2022. 8. Wanner C et al. ESC Heart Failure. 2020;7:825–834. 9. Bénichou B et al. Mol Genet Metab. 2009;96(1):4-12.
