Elelyso Gains Pediatric Gaucher Disease Indication

Elelyso Gains Pediatric Gaucher Disease Indication
Elelyso Gains Pediatric Gaucher Disease Indication

Pfizer and Protalix BioTherapeutics announced that the Food and Drug Administration (FDA) has approved Elelyso (taliglucerase alfa) for injection for pediatric patients.

Elelyso, a lysosomal glucocerebroside-specific enzyme, is now 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 were assessed in 14 pediatric patients with Type 1 Gaucher disease in 2 clinical trials. The first trial consisted of 9 patients in a 12-month, multi-center, double-blind, randomized study in treatment-naïve patients aged 2–13 years. At the end of the study, Elelyso demonstrated a decrease in spleen and liver volume and an increase in platelet count.

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The second trial was a 9-month, multi-center, open-label, single-arm study that consisted of 5 pediatric patients aged 6–16 years who were switched from imiglucerase (Cerezyme; Genzyme) to Elelyso. Elelyso was administered for 9 months at the same dose as the patient's previous imiglucerase dose. Results showed mean spleen and liver volume, platelet count, and hemoglobin value remained stable through the 9 months of Elelyso treatment.

Elelyso for injection is available as a lyophilized powder containing 200 units per vial.

For more information call (855) 353-5976 or visit Elelyso.com.

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