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Pfizer announced that FDA approved ELELYSO for treatment of Type 1 Gaucher disease in adult and pediatric patients.
Pfizer Inc. announced on Aug. 28 that FDA approved ELELYSO for injection in pediatric patients. ELELYSO is indicated for long-term enzyme replacement therapy (ERT) for adult and pediatric patients with a confirmed diagnosis of Type 1 Gaucher disease.
The safety and efficacy of ELELYSO in treating Type 1 Gaucher disease was assessed in two clinical trials consisting of pediatric patients. The first trial tested nine patients in a 12-month, multi-center, double-blind, randomized study in treatment-naïve patients aged 2-13 years. At the end of the 12-month study, therapeutic efficacy of ELELYSO was demonstrated, as measured by a decrease in spleen and liver volume and an increase in platelet count. The second trial consisted of five pediatric patients aged 6-16 years who were switched from imiglucerase to ELELYSO in a nine-month, multi-center, open-label, single-arm study in patients who had been receiving treatment with imiglucerase at dosages ranging from 9.5 units/kg to 60 units/kg every other week for a minimum of two years. ELELYSO was administered for nine months at the same dose as each patient’s previous imiglucerase dose, with relevant adjustments to maintain stability. Mean spleen and liver volume, platelet count, and hemoglobin value remained stable through nine months of ELELYSO treatment.
The recommended dosage of ELELSYO for treatment-naïve adult and pediatric patients four years of age and older is 60 units/kg of body weight administered every other week as a 60 to 120 minute intravenous infusion.