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.
Patricia, Ammeris, and Shemary, living with Fabry disease, and their family, CJ, Eric, and Eisha
Review the global reach of over 20 years of experience
- 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 variant1-4
- 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
of patients were alive at the end of the 10-year study period1
of patients did not experience any severe renal, cardiac, or cerebrovascular clinical event1
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
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 patients6
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 characterisitcs
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
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
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
“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.”
Roland, a real Fabrazyme patient for over 20 years
IMPORTANT SAFETY INFORMATION
IMPORTANT SAFETY INFORMATION
|
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate FABRAZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g. anaphylaxis) occurs, discontinue FABRAZYME and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1)]. |
WARNINGS AND PRECAUTIONS
Hypersensitivity Reactions Including Anaphylaxis
In clinical trials and post-marketing experience, approximately 1% of patients developed anaphylactic or severe hypersensitivity reactions, some life-threatening, during Fabrazyme infusion. Reactions have included localized angioedema (including swelling of the face, mouth, and throat), bronchospasm, hypotension, generalized urticaria, dysphagia, rash, dyspnea, flushing, chest discomfort, pruritus, and nasal congestion. Consider pretreating with antihistamines, antipyretics, and/or corticosteroids; however, reactions may still occur.
In Fabrazyme clinical trials, some patients developed IgE antibodies or skin test reactivity specific to Fabrazyme.
- Higher incidences of hypersensitivity reactions were observed in adult patients with persistent anti-Fabrazyme antibodies, and in those with high antibody titers compared with antibody negative adult patients.
- Consider testing for IgE antibodies in patients who experienced suspected hypersensitivity reactions and consider the risks and benefits of continued treatment in patients with anti-Fabrazyme IgE antibodies. Rechallenge of these patients should only occur under the direct supervision of qualified personnel, with appropriate medical support measures readily available.
Infusion-Associated Reactions
In Fabrazyme clinical trials, 59% of patients experienced infusion-associated reactions (IARs), some of which were severe. IARs are defined as those occurring on the same day as the infusion. The incidence of these reactions was higher in patients who were positive for anti-Fabrazyme antibodies than those negative for anti-Fabrazyme antibodies.
- Consider pretreatment with antipyretics, antihistamines, and/or corticosteroids to reduce the risk of IARs; however, they may still occur.
- If a mild or moderate IAR occurs, consider holding the infusion temporarily, decreasing the infusion rate, and/or reducing the Fabrazyme dosage. If a severe IAR occurs, discontinue Fabrazyme immediately and initiate appropriate medical treatment as needed. Assess the risks and benefits of readministering Fabrazyme and monitor patients closely if readministering.
- Patients with advanced Fabry disease may have compromised cardiac function, which may predispose them to a higher risk of severe complications from IARs. Closely monitor patients with compromised cardiac function receiving Fabrazyme.
Common Adverse Reactions
Adverse reactions reported (≥20%) were upper respiratory tract infection, chills, pyrexia, headache, cough, paresthesia, fatigue, peripheral edema, dizziness, and rash.
Please see full Prescribing Information, including Boxed WARNING
IMPORTANT SAFETY INFORMATION
Show moreIMPORTANT SAFETY INFORMATION
|
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate FABRAZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g. anaphylaxis) occurs, discontinue FABRAZYME and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1)]. |
WARNINGS AND PRECAUTIONS
Hypersensitivity Reactions Including Anaphylaxis
In clinical trials and post-marketing experience, approximately 1% of patients developed anaphylactic or severe hypersensitivity reactions, some life-threatening, during Fabrazyme infusion. Reactions have included localized angioedema (including swelling of the face, mouth, and throat), bronchospasm, hypotension, generalized urticaria, dysphagia, rash, dyspnea, flushing, chest discomfort, pruritus, and nasal congestion. Consider pretreating with antihistamines, antipyretics, and/or corticosteroids; however, reactions may still occur.
In Fabrazyme clinical trials, some patients developed IgE antibodies or skin test reactivity specific to Fabrazyme.
- Higher incidences of hypersensitivity reactions were observed in adult patients with persistent anti-Fabrazyme antibodies, and in those with high antibody titers compared with antibody negative adult patients.
- Consider testing for IgE antibodies in patients who experienced suspected hypersensitivity reactions and consider the risks and benefits of continued treatment in patients with anti-Fabrazyme IgE antibodies. Rechallenge of these patients should only occur under the direct supervision of qualified personnel, with appropriate medical support measures readily available.
Infusion-Associated Reactions
In Fabrazyme clinical trials, 59% of patients experienced infusion-associated reactions (IARs), some of which were severe. IARs are defined as those occurring on the same day as the infusion. The incidence of these reactions was higher in patients who were positive for anti-Fabrazyme antibodies than those negative for anti-Fabrazyme antibodies.
- Consider pretreatment with antipyretics, antihistamines, and/or corticosteroids to reduce the risk of IARs; however, they may still occur.
- If a mild or moderate IAR occurs, consider holding the infusion temporarily, decreasing the infusion rate, and/or reducing the Fabrazyme dosage. If a severe IAR occurs, discontinue Fabrazyme immediately and initiate appropriate medical treatment as needed. Assess the risks and benefits of readministering Fabrazyme and monitor patients closely if readministering.
- Patients with advanced Fabry disease may have compromised cardiac function, which may predispose them to a higher risk of severe complications from IARs. Closely monitor patients with compromised cardiac function receiving Fabrazyme.
Common Adverse Reactions
Adverse reactions reported (≥20%) were upper respiratory tract infection, chills, pyrexia, headache, cough, paresthesia, fatigue, peripheral edema, dizziness, and rash.
Please see full Prescribing Information, including Boxed WARNING
INDICATION AND USAGE
Fabrazyme® is indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease.
References: 1. Germain DP et al. J Med Genet. 2015;52(5):353-358. 2. Fabrazyme® (agalsidase beta). Prescribing Information 2024. 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.