Pfizer and Protalix BioTherapeutics have secured approval from the US Food and Drug Administration (FDA) for Elelyso (taliglucerase alfa) to treat paediatric patients with type 1 Gaucher disease.
Elelyso will be provided in the form of injection for paediatric patients.
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Pfizer global medical affairs and global innovative pharma business senior vice-president Rory Connor said: “The approval of Elelyso to treat paediatric patients with type 1 Gaucher disease provides physicians another treatment option for this rare and potentially debilitating disease.
“This paediatric indication, along with the recent announcement that Elelyso received kosher certification by the Orthodox Union, reinforces the ongoing commitment of Pfizer to addressing the needs of the Gaucher community.”
Elelyso’s safety and efficacy has been evaluated in 14 paediatric patients with type 1 Gaucher disease in two clinical trials.
Nine treatment-naive patients aged two to 13 years were included in a 12-month, multi-centre, double-blind and randomised first trial.
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By GlobalDataElelyso showcased therapeutic efficacy at the end of the 12-month study, which was measured by a decrease in spleen and liver volume and an increase in platelet count.
The second trial included five paediatric patients aged six to 16 years, and was a nine-month multi-centre, open-label and single-arm study.
All patients in this trial were switched from imiglucerase to Elelyso.
The patients in the study have received treatment with imiglucerase at dosages ranging from 9.5 units/kg to 60 units/kg every other week for a minimum of two years, while Elelyso has been administered for nine months at the same dose as each patient’s previous imiglucerase dose.
In the nine months Elelyso treatment, the mean spleen and liver volume, platelet count and haemoglobin value remained stable, according to Pfizer.
Pfizer noted that Elelyso for injection is supplied as 200 units per vial and is available by prescription only.
Image: Micrograph of Gaucher disease and necrotic bone, H&E stain. Photo: courtesy of Nephron.
