Profile
Fabrazyme® (agalsidase beta) is an enzyme replacement therapy (ERT) indicated for use in adults, children and adolescents aged 8 years and older with Fabry disease.1,2
FIRST prescribed treatment
Fabrazyme® is the first globally prescribed treatment for Fabry disease, chosen for nearly 6000 patients worldwide.2,3
FIRST for
patients
The first globally prescribed treatment, with over 20 years of global real-world experience and a commitment to advancing fabry disease knowledge through the patient-centred Fabry Registry.2,4
FIRST in
evidence
The first treatment to have published long-term efficacy (up to 10 years) from a phase 3 clinical study and real-world evidence in a peer-reviewed international journal.5
Safety profile
Learn more about the safety and tolerability profile for Fabrazyme® for patients living with Fabry disease.
Mechanism of action
Click through the slides below using the arrows or swipe to learn more about how Fabrazyme® works.
Fabrazyme® is a recombinant enzyme, with an amino acid sequence identical to the body’s own native enzyme2
Fabrazyme® binds to the cell surface likely through mannose-6-phosphate, mannose, and asialoglycoprotein receptors, allowing it to be internalized2
Once inside the cell, Fabrazyme® is directly transported to the lysosome2
In the lysosome, Fabrazyme® hydrolyzes GL-3, resulting in clearance of accumulated GL-3 in major organs2
Fabrazyme® is a recombinant enzyme, with an amino acid sequence identical to the body’s own native enzyme2
Fabrazyme® binds to the cell surface likely through mannose-6-phosphate, mannose, and asialoglycoprotein receptors, allowing it to be internalized2
Once inside the cell, Fabrazyme® is directly transported to the lysosome2
In the lysosome, Fabrazyme® hydrolyzes GL-3, resulting in clearance of accumulated GL-3 in major organs2
Usage
Adapt to your patient's life by optimising their infusion time
Fabrazyme® should be administered as an intravenous (IV) infusion.
The initial IV infusion rate should be no more than 0.25 mg/min (15 mg/hour). The infusion rate may be slowed in the event of infusion-associated reactions.
After patient tolerance is well established, the infusion rate may be increased in increments of 0.05 to 0.083 mg/min (increments of 3 to 5 mg/hr) with each subsequent infusion. In clinical trials with classic patients, the infusion rate was increased incrementally to reach a minimum duration of 2 hours. This was achieved after 8 initial infusions at 0.25 mg/min (15 mg/hr), without any IARs, change in infusion rate, or infusion interruption. A further decrease of infusion time to 1.5 hours was allowed for patients without new IARs during the last 10 infusions or reported serious adverse events within the last 5 infusions. Each rate increment of 0.083 mg/min (~5 mg/hr) was maintained for 3 consecutive infusions, without any new IARs, change in infusion rate, or infusion interruption, before subsequent rate increases.
The below protocol is not mandated and is an example for classic patients:*,2
| Examples | |||||||
| Patient weight | 50kg | 70kg | 70kg | ||||
| Rate increase | 5 mg/hr | 5 mg/hr | 3 mg/hr | ||||
| Infusion number | Rate mg/hr | Total infusion time | Rate mg/hr | Total infusion time | Rate mg/hr | Total infusion time | |
| 1-8 | 15 | 3h 20m | 15 | 4h 40m | 15 | 4h 40m | |
| 9 | 20 | 2h 30m | 20 | 3h 30m | 18 | 3h 53m | |
| 10 | 25 | 2h | 25 | 2h 48m | 21 | 3h 20m | |
| 11 | 30 | 1h 40 m | 30 | 2h 20m | 24 | 2h 55m | |
| 12 | 33 | 1h 30m | 35 | 2h | 27 | 2h 35m | |
| 13 | 40 | 1h 45m | 30 | 2h 20m | |||
| 14 | 45 | 1h 33m | 33 | 2h 07m | |||
| 15 | 36 | 1h 57m | |||||
| 16 | 39 | 1h 48m | |||||
| 17 | 42 | 1h 40m | |||||
| 18 | 45 | 1h 33m | |||||
*For patients weighing <30kg: maximum infusion rate should remain 0.25 mg/min (15 mg/hr).
Did you know?
Fabry disease starts early in the kidney and is a silent threat. Progressive accumulation of globotriaocylceramide (GL-3) leads to cellular changes and histological damage.6 Fabry nephropathy shows similarities to other proteinuric nephropathies of metabolic origin.4 Once the mechanisms leading to tissue injury are activated, the progression of Fabry nephropathy becomes irreversible.7,8
References
1. UK(GB) Summary of Product Characteristics (SmPC) - Fabrazyme 35mg - April 2024 (accessed February 2025)
2. https://www.sciencedirect.com/science/article/pii/S1096719222004206#s0100 (accessed February 2025).
3. Sanofi Genzyme. Data on file. Based on estimated patient numbers based on publicly published revenue as of September 2022.
4. Wanner C et al. Mol Genet Metab 2023; 139(3): 107603.
5. Germain D et al. J Med Genet 2015; 52(5): 353–358.
6. Waldek S and Ferriozi S. BMC Nephrol. 2014;15:72.
7. Ortiz A, et al. Mol Genet Metab. 2018;123(4):416–27.
8. Van der Veen SJ, et al. Mol Genet Metab. 2022;135(2):163–69
MAT-GB-2105812 (v7.0) Date of preparation: February 2025