Pfizer reports positive clinical data for Elelyso in paediatric patients

Pfizer has reported positive clinical trial data that add to the body of evidence supporting the safety and efficacy of the Gaucher disease treatment Elelyso.

Data from a phase III multi-centre extension trial has been presented at the WORLDSymposium demonstrating the long-term effectiveness of Elelyso in paediatric patients with type 1 Gaucher disease who were treatment-naive or previously treated with imiglucerase.

It was shown that treatment-naive patients receiving Elelyso for 36 months demonstrated clinical improvements, as measured by a decrease in spleen and liver volume and an increase in platelet count. 

Meanwhile, those that switched from imiglucerase to Elelyso saw their spleen and liver volume, platelet count and haemoglobin value remain stable for 33 months of treatment.

Dr Ari Zimran of the Gaucher Clinic at Jersualem's Sha'are Zedek Medical Center 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."

The company also recently announced that the US Food and Drug Administration has accepted its new pain relief therapy  ALO-02 for regulatory review.