Think Fabrazyme first for pediatric patients with Fabry
In Fabry disease, organ involvement can begin during infancy or early childhood
Arrhythmias, most commonly bradycardia, are one of the first cardiac manifestations for Fabry disease, beginning in childhood. The frequency of arrhythmias did not differ by sex.1
GL3 inclusions have been found in fetal podocytes2,3
Neuropathic pain is the most frequent symptom reported by children and adolescents, both male and female4
The first signs of Fabry appear in childhood, but diagnosis is often delayed5
- Chronic pain
- Angiokeratomas
- Hyperhidrosis
- Heat and cold intolerance
- Gastrointestinal symptoms
In a pediatric study based on the Fabry Registry:
- Boys and girls with Fabry disease begin developing symptoms at an early age (median age of 6 years for boys [n=194] and 9 years for girls [n=158])7
- Moreover, some may have early severe complications7
Pain is one of the earliest symptoms of Fabry, affecting 60% to 80% of classically affected boys and girls8
Think Fabrazyme, think proven efficacy and safety in pediatric patients
In an analysis of 24 Fabrazyme-treated pediatric patients with Fabry disease aged 2 to <8 years, plasma GL-3 levels were normalized9
At baseline
- All male patients had elevated plasma GL-3 (i.e., >7.03 μg/mL)9
After treatment
- Plasma GL-3 levels fell within the normal range (i.e., ≤7.03 μg/mL) over:9
16 pediatric patients with Fabry disease, aged 8–16 years, were evaluated in an open-label, uncontrolled study9
Before treatment with Fabrazyme
2 of 16 patients had normal levels of GL-3 in the blood
After 5.5 months and 11 months of Fabrazyme treatment
100% patients had normal levels of GL-3 in the blood
Study 3: Open-label, single-arm, multinational, multicenter study in 16 pediatric patients with Fabry disease (14 males, 2 females), aged 8 to 16 years (median 12 years). Histological evaluation of the capillary endothelium, deep vessel endothelium, deep vessel smooth muscle cells, and perineurium of biopsied skin was conducted using histochemistry with light microscopy. Scoring was on a scale of 0 (defined as none) to 3 (severe).9
Study Dose: Fabrazyme® 1 mg/kg every 2 weeks for up to 48 weeks.9
Baseline Characteristics: All 14 males had elevated plasma GL-3 levels (i.e., >7.03 μg/mL),whereas the two female patients had normal plasma GL-3 levels. 12 of the 14 males had GL-3 inclusions present on skin biopsy (scores 1, 2, or 3), whereas the two females had no GL-3 inclusions at baseline.9
“My earliest symptom that I can link to Fabry was neuropathy in my hands and feet at age 10 or 11. I had pain but nothing else, so it was difficult back then to understand what was causing it.”
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. Wilson HC, et al. Am J Cardiol 2017;120:251–255. 2. Vaisbich MH et al. J Bras Nefrol. 2022 Apr-Jun;44(2):268-280. 3. Tøndel C et al. Am J Kidney Dis. 2008. 4. Hopkin et al. Pediatr Res. 2008 Nov;64(5):550-5. 5. Desnick RJ. Ann Intern Med. 2003;138(4):338-346. 6. Ellaway C. Paediatric Fabry disease. Transl Pediatr 2016;5(1):37-42. 7. Hopkin RJ, et al. Pediatr Res. 2008;64(5):550-555. 8. Germain DP. Orphanet J Rare Dis. 2010;5(30):1-49. 9. Fabrazyme® (agalsidase beta). Prescribing Information 2024.