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Think Fabrazyme first for pediatric patients with Fabry1
In Fabry disease, organ involvement can begin during infancy or early childhood
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Arrhythmias, most commonly bradycardia, are one of the earliest cardiac manifestations of Fabry disease, beginning in childhood in both males and females.2
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GL-3 inclusions have been found in fetal podocytes.3,4
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Neuropathic pain is the most frequent symptom reported by children and adolescents, both male and female.5
The first signs of Fabry appear in childhood, but diagnosis is often delayed6
- Chronic pain
- Angiokeratomas
- Hypohidrosis
- Heat and cold intolerance
- Gastrointestinal symptoms
In a pediatric study of 352 children with Fabry disease, 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])5
- Moreover, some may have early severe complications5
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 normalized1
- At baseline: All male patients had elevated plasma GL-3 (i.e., >7.03 μg/mL)1
- After treatment: Plasma GL-3 levels fell within the normal range (i.e., ≤7.03 μg/mL) over1:
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6 months
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12 months
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24 months
16 pediatric patients with Fabry disease, aged 8–16 years, were evaluated in an open-label, uncontrolled study1
Before treatment with Fabrazyme
2 of 16 patients had normal levels of GL-3 in the blood
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After 48 weeks of Fabrazyme treatment
100% patients had normal levels of GL-3 in the blood
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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).1
Study Dose: Fabrazyme 1 mg/kg every 2 weeks for up to 48 weeks.1
Baseline Characteristics: All 14 males had elevated plasma GL-3 levels (i.e., >7.03 μg/mL), whereas the 2 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 2 females had no GL-3 inclusions at baseline.1
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Roland, a real Fabrazyme patient for over 20 years
“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.”
Indication
References: 1. Fabrazyme (agalsidase beta). Prescribing Information. Sanofi. 2. Wilson HC et al. Am J Cardiol. 2017;120:251–255. 3.Vaisbich MH et al. J Bras Nefrol. 2022;44(2):268-280. 4. Tøndel C et al. Am J Kidney Dis. 2008;51(5):767-76. 5. Hopkin et al. Pediatr Res. 2008;64(5):550-555. 6. Desnick RJ. Ann Intern Med. 2003;138(4):338-346. 7. Ellaway C. Transl Pediatr. 2016;5(1):37-42. 8. Germain DP. Orphanet J Rare Dis. 2010;5(30):1-49.
