Elelyso taliglucerase alfa: Phase IV data

The double-blind, international Phase IV PB-06-005 trial in 11 treatment-naïve pediatric patients showed that 30 and 60 U/kg Elelyso increased mean hemoglobin concentration, the primary endpoint, from baseline at month 12 by 13.8% and 15.8%, respectively. In anemic patients (n=7), Elelyso increased mean hemoglobin concentration from baseline at month 12 by 4.3 g/dL for the 30 U/kg dose and by 1.7 g/dL for the 60 U/kg dose. On secondary endpoints, low- and high-dose Elelyso led to reductions from baseline at month 12 of 28.6% and 41.1% in spleen volume, respectively, and of 6.3% and 14%, respectively, in liver volume. Additionally, Elelyso reduced chitotriosidase activity from baseline at month 12 by 58% at the low dose and by 66% at the high dose. Furthermore, low- and high-dose Elelyso increased platelet counts by 30.9% and 73.7%, respectively. There was 1 treatment-related serious adverse event of gastroenteritis. Data were presented at the European Working Group on Gaucher Disease meeting in Paris. ...