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fabry

Fabry

<h2>Design of the Fabry Registry and objectives</h2>
<ul>
 <li>The Fabry Registry is a multicenter, international, longitudinal, observational study open to all patients with a confirmed diagnosis of FD, irrespective of treatment status or therapy administered</li>
 <li>It is designed to collect consistent retrospective and prospective patient data with the primary objectives of exploring and defining the variability in the natural history and clinical characteristics across the disease spectrum,&nbsp;and of tracking and characterizing long-term treatment outcomes in a real-world setting</li>
</ul>

<h2>Enrollment of patients in the Fabry Registry over time</h2>
<ul>
 <li>The Fabry Registry has grown significantly from an initial enrollment of 21 patients with FD at two sites in 2001 to 8,098 patients (43.5% male, 56.5% female) as of October 7, 2022</li>
 <li>The Fabry Registry has a global reach, with a current presence in 44 countries worldwide and 219 participating sites (EMEA 45.7%; North America 40.3%; JAPAC 8.1%; Latin America 5.9%)</li>
 <li>Genotype data are available for 90.3% of patients and, overall, 1,039 GLA variants are represented in the Fabry Registry: classic phenotype (406 variants, 3,532 patients), later-onset phenotype (54 variants, 1,239 patients), and “other” phenotypes* (e.g. 70 variants, 762 patients)</li>
</ul>

Cumulative numbers of participating physicians and patients enrolled in the Fabry Registry by year of enrollment

(122,599 years for males, 188,537 years for females).

(23,983 years for males, 16,436 years for females)

* [Likely] benign, likely classic, likely later-onset, variants of uncertain significance. 
 **Date of treatment initiation (for “ever-treated” patients) or date of most recent data entry (for “never-treated” patients).

<h2>Topics covered by Fabry Registry publications</h2>

<h2>Key contributions from 20+ years of the Fabry Registry</h2>
<ul>
 <li>The Fabry Registry has now been operational for over 20 years and has collected real-world demographic and longitudinal clinical data from more than 8,000 individuals with FD</li>
 <li>Leveraging the accumulating evidence base, multidisciplinary collaborations have resulted in the creation of 32 peer-reviewed scientific publications on the onset and progression of FD, its clinical management, the role of sex and genetics, the outcomes of ERT with Fabrazyme®, and prognostic factors</li>
</ul>

Clinical outcomes of treatment with Fabrazyme®

Continued evolution of data collection

Advancing statistical methodology

<h2>Use of real-world data in global multistakeholder collaborations</h2>
<ul>
 <li>Fabry Registry analyses are used in multistakeholder engagement between healthcare professionals, patients and patient organizations, payors, and healthcare policy makers, and can support decision making by regulatory agencies</li>
 <li>The patient-centered Fabry Registry fosters collaborative research partnerships with the overarching goal of optimizing the clinical management of patients with FD and is well positioned to add to its past achievements</li>
</ul>

<h2>Learn more about Fabrazyme®</h2>

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

Discover&nbsp;safety profile

Find out more about the safety and tolerability profile for Fabrazyme&reg;.

Abbreviations

Explore the advancement of Fabry disease expertise and dissemination of real-world
evidence to the Fabry community.

Prescribing Information GB

Prescribing Information NI

FABRY

RARE-DISEASE

Global reach of over 20 years experience in the patient-centered Fabry Registry

<h2>Fabrazyme® cleared GL-3 in the kidney, heart, and skin in as quickly as 6 months after initiation6</h2>

Adapted from Germain D et al. 2007.6

Study design details

<h2>A potential reduction of life threatening events by 61%3</h2>
61% relative risk reduction of renal, cardiac and cerebrovascular life-threatening events and death in Fabrazyme®-treated patients vs. placebo.3&nbsp;A smaller percentage of Fabrazyme®- treated patients (27%) experienced clinical events vs. placebo (42%).3&nbsp;(Absolute risk reduction = 14%).3
Fabrazyme® substantially lowered the rate of renal, cardiac, and cerebrovascular clinical events among Fabrazyme®-treated vs. placebo-treated patients (risk reduction = 53% ITT population [HR 95% CI: 0.47 (0.21–1.03); p=0.06.3]; risk reduction = 61% per-protocol population [HR 95% CI: 0.39 (0.16–0.93); p=0.034.3]).3
The time-to-event analyses for the composite end point in both treatment groups in the per-protocol population3

Results are from the population of protocol-adherent patients who followed all of the clinical study guidelines after adjustment for baseline proteinuria.3 
 Adapted from Banikazemi M et al. 2007.3

<h2>Protection from long term progression of renal function decline4</h2>
70.9% slower rate of eGFR decrease in Fabrazyme®-treated patients than a comparable untreated patient, after adjusting for the noted imbalances in gender and proteinuria.4 Fabrazyme®-treated classic Fabry disease patients experience a slower median eGFR decrease (2.46 mL/min/1.73m2/year, 95% CI: [0.63–4.29]; p=0.0087) than comparable untreated patients.4
Forest plot comparing the adjusted median eGFR slopes in Fabrazyme®-treated versus untreated patients4

Adjusted median eGFR slopes and IQR for the overall treated and untreated groups and the individual studies. 
 Adapted from Ortiz A et al. 2021.4

<h2>Early initiation with Fabrazyme®, before development of fibrosis, could help improve cardiac morphology, function, and exercise capacity5</h2>
Change in left ventricular mass during 3 years of ERT.5

Adapted from Weidemann F et al. 2009.5

Change in exercise capacity during 3 years of ERT5

Adapted from Weidemann F et al. 2009.5 
 A mild but significant improvement in exercise capacity could be demonstrated for patients 
 with no fibrosis (p=0.014).5

<h2>Did you know?</h2>
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.8 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.9,10

Explore&nbsp;safety profile

Discover more about treatment with Fabrazyme&reg;&nbsp;for patients living with Fabry disease.

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

Get in touch with us

<h3>&nbsp;</h3>
<h4>References</h4>
<ol>
 <li>
 Eng CM et al. N Engl J Med 2001; 345(1): 9–16.</li>
 <li>
 Thurberg B et al. Circulation 2009; 119(19): 2561–2567.</li>
 <li>
 Banikazemi M et al. Ann Intern Med 2007; 146: 77–86.</li>
 <li>
 Ortiz A et al. Clinical Kidney Journal 2021; 14 (4): 1136–1146.</li>
 <li>
 Weidemann F et al. Circulation 2009; 119(4): 524–529.</li>
 <li>
 Germain D et al. J Am Soc Nephrol 2007; 18(5): 1547–1557.</li>
 <li>
 Eng CM et al. N Engl J Med 2001; 345(1): 9–16.</li>
 <li>
 Waldek S and Ferriozi S. BMC Nephrol. 2014;15:72.</li>
 <li>
 Ortiz A, et al. Mol Genet Metab. 2018;123(4):416–27.</li>
 <li>
 Van der Veen SJ, et al. Mol Genet Metab. 2022;135(2):163–69</li>
</ol>

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

Fabrazyme® (agalsidase beta) evidence

<h2>Summary of safety information</h2>
Please refer to the <a href="https://www.medicines.org.uk/emc/product/15500/smpc" target="_blank">Summary of Product Characteristics</a> before prescribing.
<h2>Dosage and administration1</h2>
Treatment with Fabrazyme® should be supervised by a physician experienced in the management of patients with Fabry disease or other inherited metabolic diseases.
The recommended dose of Fabrazyme® is 1mg/kg body weight administered once every 2 weeks as an intravenous infusion.
<ul>
</ul>

Special populations

<h3> Administration</h3>
<h4>Intravenous infusion</h4>
Initial IV infusion rate: no more than 0.25 mg/min (15 mg/h)
After patient tolerance is well established, the infusion rate may be increased in increments of 0.05 to 0.083 mg/min (3 to 5 mg/hr) with each subsequent infusion. In the event of infusion-associated reactions, the infusion rate may be slowed.
<h2>Contraindications1</h2>
Life threatening hypersensitivity to agalsidase beta or to any of the excipients of this medicine (listed in section 6.1 of the Summary of Product Characteristics).
Fabrazyme® should not be administered with chloroquine, amiodarone, benoquin or gentamycin due to a theoretical risk of inhibition of intra-cellular α-galactosidase A activity.
<h2>Precautions and Warnings1</h2>
Immunogenicity 
 As agalsidase beta is a recombinant protein, the development of IgG antibodies is anticipated in patients with little or no residual enzyme activity. The majority of patients developed IgG antibodies to agalsidase beta, typically within 3 months of the first Fabrazyme® infusion.
Infusion associated reactions
Patients with antibodies to agalsidase beta have a higher risk of experiencing infusion-associated reactions, defined as any adverse event occurring on the infusion day. These patients should be treated with caution when re-administering agalsidase beta to these patients, and their antibody status should be regularly monitored.
Hypersensitivity
A small number of patients have experienced reactions suggestive of immediate (Type I) hypersensitivity.&nbsp;As with any intravenous protein medicinal product, allergic-type hypersensitivity reactions are possible. If severe allergic or anaphylactic-type reactions occur, immediate discontinuation of the administration of Fabrazyme® should be considered and appropriate treatment initiated.
Patients with advanced renal disease
The effect of Fabrazyme® treatment on the kidneys may be limited in patients with advanced renal disease.
Sodium
This medicinal product contains less than 1 mmol sodium per vial, that is to say essentially 'sodium-free'.
Traceability
To enhance traceability, it is recommended to clearly record the name and batch number of the administered product

<h2>Adverse reactions1</h2>
Adverse reactions are listed by system organ class and frequency in the list below. Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness.1

Very common (≥1/10)

Common (≥1/100 to &lt; 1/10)

Uncommon (≥1/1000 to &lt; 1/100)

Not known (cannot be estimated by data)

The list presents adverse reactions reported from clinical trials (168 patients [154 males, 14 females] treated with Fabrazyme® 1 mg/kg EOW for ≥1 infusion up to a maximum of 5 years).1 Frequencies (very common ≥ 1/10; common ≥ 1/100 to &lt; 1/10 and uncommon ≥ 1/1000 to &lt; 1/100). Adverse reactions reported in post-marketing period are indicated as frequency “not known” (cannot be estimated from data).1 Adverse reactions were mostly mild or moderate in severity.1 Adverse reaction terminology is based upon the Medical Dictionary for Regulatory Activities (MedDRA).1

<h2>Did you know?</h2>
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.2 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.3,4

Explore the results and evidence supporting Fabrazyme&reg;&nbsp;and how it was studied through several clinical trials.

<h3>&nbsp;</h3>
<h4>References</h4>
<ol>
 <li>
 Fabrazyme®. Summary of Product Characteristics 2024.</li>
 <li>
 Waldek S and Ferriozi S. BMC Nephrol. 2014;15:72.</li>
 <li>
 Ortiz A, et al. Mol Genet Metab. 2018;123(4):416–27.</li>
 <li>
 Van der Veen SJ, et al. Mol Genet Metab. 2022;135(2):163–69</li>
</ol>

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

Fabry

Latest Articles

Global reach of over 20 years experience in the patient-centered Fabry Registry

Fabrazyme® (agalsidase beta) evidence

Fabrazyme® (agalsidase beta) safety profile