Elelyso can now be used as a long-term enzyme replacement therapy (ERT) for the treatment of these patients.
Pfizer Global Innovative Pharma Business senior vice-president of Global Medical Affairs Rory O’Connor said the approval of Elelyso to treat pediatric patients with Type 1 Gaucher disease provides physicians another treatment option for this rare and potentially debilitating disease.
"This pediatric indication, along with the recent announcement that ELELYSO received kosher certification by the Orthodox Union (OU), reinforces the ongoing commitment of Pfizer to addressing the needs of the Gaucher community," O’Connor said.
Two clinical trials have been carried out to evaluate the safety and efficacy of Elelyso in fourteen pediatric patients with Type 1 Gaucher disease.
Around nine treatment-naïve patients aged two to 13 years were included in the 12-month, multi-center, double-blind, randomized first trial.
At the end of the trial period, Elelyso showed therapeutic efficacy, as measured by a decrease in spleen and liver volume and an increase in platelet count.
The second trial was a nine-month, multi-center, open-label, single-arm study that enrolled five pediatric patients aged six to 16 years who were switched from imiglucerase to Elelyso.
The trial included 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.
In this trial, the vaccine was administered for nine months at the same dose as each patient’s previous imiglucerase dose.
The company said that 60 units per kg of body weight every other week as a 60 to 120 minute intravenous infusion is the recommended dosage of Elelsyo for treatment-naïve adult and pediatric patients four years of age and older.
The company said that Elelyso for injection is supplied as 200 units per vial and is available by prescription only.
Image: Safety and efficacy of Elelyso for injection was evaluated in two trials in fourteen pediatric patients with Type 1 Gaucher disease. Photo: courtesy of Baitong333/ freedigitalphotos.net.