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Orphan drug performs well in phase II

Genzyme's eliglustat tartrate for the orphan condition Gaucher disease has performed well in the two-year follow-up of its phase II trial

Positive results from the two-year follow-up of Genzyme's phase II trial of its investigational treatment for Gaucher disease, eliglustat tartrate, will be published in an upcoming edition of the journal Blood. The results were previously announced at the Lysosomal Disease Network WORLD Symposium earlier this year.

Gaucher disease is a lysosomal storage disease affecting fewer than 10,000 people worldwide. Patients with Gaucher disease accumulate lipid engorged cells around the body, predominantly in the spleen, liver and bone marrow, which can lead to spleen and liver enlargement, anaemia, excessive bleeding and bruising and bone disease. Current treatment options are administered intravenously, but as an oral capsule eliglustat tartrate would offer sufferers a more convenient treatment option.

Patients were treated with either 50mg or 100mg of eliglustat tartrate twice daily during the one-year, multicentre, open-label phase II study. The study assessed three endpoints: spleen size, haemoglobin and platelet levels. Patients treated with eliglustat tartrate continued to experience clinically significant improvements in all three endpoints and in liver volume after two years.

Genzyme is now enrolling up to 450 patients in a phase III trial programme, which will be the largest ever conducted in Gaucher disease. The three trials – ENCORE, ENGAGE and EDGE – will take place across more than 50 sites in at least 25 countries.

The phase II follow-up results are available to subscribers on the Blood website now.

23rd August 2010

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