Uplyso taliglucerase alfa: Additional Phase III data

Additional data from a double-blind, international Phase III trial in 31 treatment-naïve patients showed that 30 and 60 U/kg IV Uplyso every 2 weeks significantly reducing spleen volume from baseline by 26.9% and 38%, respectively, at 9 months (p<0.0001 for both). Low- and high-dose Uplyso also significantly increased hemoglobin levels from baseline by 14.8% and 22.2%, respectively, (p=0.001 and p<0.0001), and significantly decreased liver size from baseline by 10.5% and 11.1%, respectively, at 9 months (p=0.0041 and p<0.0001). Uplyso increased platelet counts by 13.7% and 72.1% from baseline in the low- and high-dose groups, respectively (p=0.046 and p=0.0031). Uplyso was well tolerated with no serious or severe adverse events reported. Data were presented at the Lysosomal Disease Network World Symposium in Miami. An open-label extension study of the trial, which has an SPA from FDA, is ongoing. ...