Interim data available from 16 women and four men between the ages of 18 and 63 demonstrated that Plicera was generally well-tolerated at all doses evaluated. GCase (the enzyme deficient in Gaucher patients) activity as measured in white blood cells was increased in 15 of the 20 patients. The five patients without a clear increase were in either the lowest dose cohort or the cohort dosed least frequently.
The primary objective of this study is to evaluate safety and tolerability of different doses and dosing regimens of Plicera. The secondary objective is to evaluate certain pharmacodynamic measures of treatment, including effects on GCase levels in white blood cells.
Thirty individuals with Gaucher disease currently receiving enzyme replacement therapy were enrolled in this study. The protocol required that patients temporarily stop enzyme replacement therapy with Cerezyme (imiglucerase) to receive Plicera for four weeks. The patients had 10 different mutations and on average were on enzyme replacement therapy with Cerezyme for 10 years prior to the study.
John Crowley, president and CEO of Amicus Therapeutics, said: “We are very encouraged by these early results in this first study of Plicera in patients with Gaucher disease who have switched off of Cerezyme to Plicera.”