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news.Pfizer has reported positive results from its multi-center, extension Phase III trial evaluating the long-term efficacy and safety of Elelyso (taliglucerase alfa) for injection in pediatric patients with Type 1 Gaucher disease who were treatment-naïve or previously treated with imiglucerase.
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A total of 15 patients aged two to 18 years from two groups were included in this pediatric-specific extension trial.
The first group included ten previously treatment-naïve pediatric patients and they were treated with Elelyso for up to 36 months at 30 units per kg or 60 units per kg.
The trial’s second group included five pediatric patients who were previously switched from imiglucerase and were given Elelyso for a total of 33 months at the same dose as each patient’s previous imiglucerase dose.
At the end of the trial, treatment-naïve patients treated with Elelyso for 36 months showed clinical improvements, as measured by a decrease in spleen and liver volume and an increase in platelet count.
The company said that in patients previously switched from imiglucerase to Elelyso, mean spleen and liver volume, platelet count and hemoglobin value remained stable through 33 months of treatment with Elelyso.
Sha’are Zedek Medical Center head of Gaucher Clinic and lead trial investigator Ari Zimran said: "The data presented at the WORLDSymposium contribute to our understanding of Elelyso as a long-term enzyme replacement therapy for patients with a confirmed diagnosis of Type 1 Gaucher disease.
"This study further underscores the clinical benefit of ongoing treatment with ELELYSO in pediatric patients with Type 1 Gaucher disease."
In the US, Elelyso is approved for long-term enzyme replacement therapy (ERT) for adult and pediatric patients with a confirmed diagnosis of Type 1 Gaucher disease.
The recommended dosage of Elelsyo for treatment-naïve adult and pediatric patients four years of age and older is 60 units per kg of body weight administered every other week as a 60 to 120 minute intravenous infusion, the company noted.
Image: Pfizer world headquarters. Photo: courtesy of Jim.henderson.