This website contains promotional content and is intended for Healthcare Professionals based in the United Kingdom only. Some content is only relevant to HCPs practising in Great Britain (England, Scotland, Wales). This website is optimised for desktop use, and some features may perform differently on mobile devices.

Adverse event reporting can be found at the bottom of the page.

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

FIRST prescribed treatment

Fabrazyme® is the first globally prescribed treatment for Fabry disease, chosen for nearly 6000 patients worldwide.1,2

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.1,3
 

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.4

 

 

Evidence 

Discover the results and evidence supporting Fabrazyme® and how it was studied through several clinical trials.


 


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 enzyme1

Fabrazyme® binds to the cell surface through mannose-6-phosphate, mannose, and asialoglycoprotein receptors, allowing it to be internalized1

Once inside the cell, Fabrazyme® is directly transported to the lysosome1

In the lysosome, Fabrazyme® hydrolyzes GL-3, resulting in clearance of accumulated GL-3 in major organs1

1/4

Fabrazyme® is a recombinant enzyme, with an amino acid sequence identical to the body’s own native enzyme1

Fabrazyme® binds to the cell surface through mannose-6-phosphate, mannose, and asialoglycoprotein receptors, allowing it to be internalized1

Once inside the cell, Fabrazyme® is directly transported to the lysosome1

In the lysosome, Fabrazyme® hydrolyzes GL-3, resulting in clearance of accumulated GL-3 in major organs1

1/2

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:*,1

Examples
Patient weight50kg70kg70kg
Rate increase5 mg/hr5 mg/hr3 mg/hr
Infusion number Rate mg/hrTotal infusion timeRate mg/hrTotal infusion timeRate mg/hrTotal infusion time
1-8153h 20m154h 40m154h 40m
9202h 30m203h 30m183h 53m
10252h252h 48m213h 20m
11301h 40 m302h 20m242h 55m
12331h 30m352h272h 35m
13  401h 45m302h 20m
14  451h 33m332h 07m
15    361h 57m
16    391h 48m
17    421h 40m
18    451h 33m

*For patients weighing <30kg: maximum infusion rate should remain 0.25 mg/min (15 mg/hr), infusion time of no more than 2 hours.

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.5 Fabry nephropathy shows similarities to other proteinuric nephropathies of metabolic origin.3 Once the mechanisms leading to tissue injury are activated, the progression of Fabry nephropathy becomes irreversible.6,7

Resources

Quantifying the effect of Fabrazyme® on the preservation of renal function in Fabry disease
L

Learning from the Canadian Fabry Disease Initiative
L
L

Understanding the impact of Fabrazyme® on long-term renal function
L

Learn more about Fabrazyme®

Fabrazyme® evidence

Explore the results and evidence supporting Fabrazyme® and how it was studied through several clinical trials.

Fabrazyme® safety profile

Find out more about the safety and tolerability profile for Fabrazyme®.

1/2

Fabrazyme® evidence

Explore the results and evidence supporting Fabrazyme® and how it was studied through several clinical trials.

Fabrazyme® safety profile

Find out more about the safety and tolerability profile for Fabrazyme®.

 

 

Get in touch with us

Do you have questions or need support? We are here to help you.

 

 

References

1. Fabrazyme® (agalsidase beta) Summary of Product Characteristics. 2024.

2. Sanofi Genzyme. Data on file. Based on estimated patient numbers based on publicly published revenue as of September 2022.

3. Wanner C et al. Mol Genet Metab 2023; 139(3): 107603.

4. Germain D et al. J Med Genet 2015; 52(5): 353–358.

5. Waldek S and Ferriozi S. BMC Nephrol. 2014;15:72.

6.  Ortiz A, et al. Mol Genet Metab. 2018;123(4):416–27.

7. Van der Veen SJ, et al. Mol Genet Metab. 2022;135(2):163–69

MAT-GB-2105812 (v6.0) Date of preparation: March 2024