Genzyme Corporation has reported positive three-year follow-up data from a Phase II clinical trial of eliglustat tartrate, an investigational oral therapy for Gaucher disease type 1.
The data revealed that sustained or further improvements were observed across all endpoints at the follow up.
Genzyme previously reported that the Phase II trial of eliglustat tartrate had met its primary endpoint at one year, and the data demonstrated continued improvement through two years.
The primary composite endpoint was a clinically meaningful response in at least two of three endpoints: improvements in spleen size, haemoglobin and platelet levels.
The study reviewed 19 patients through three years and the extension phase of this trial is still ongoing.
The three-year follow-up data showed that the spleen volume of people received eliglustat tartrate decreased from baseline by a mean of 61%, while liver volume decreased from baseline by 29%.
Their haemoglobin level increased from baseline by a mean of 2.6g/dl, while the platelet count increased from baseline by a mean of 91%.
Genzyme is currently enrolling patients in three global multi-centre Phase III trials of eliglustat tartrate, across 50 sites in more than 25 countries.
Gaucher disease is an inherited condition characterised by spleen and liver enlargement, anaemia, excessive bleeding and bruising and bone disease. It affects fewer than 10,000 people worldwide.
