Sanofi and Genzyme report positive Gaucher disease drug trial data

Sanofi and Genzyme have reported positive clinical trial data from a pair of new phase III clinical trials of their investigational therapy for Gaucher disease type 1, eliglustat tartrate.

According to the results of the Encore and Engage trials, the developmental compound is a safe and effective therapy for this rare condition, helping to reduce spleen size among patients while delivering a non-inferior performance to Cerezyme.

This data will form the basis of a registration package for the new oral therapy, with the clinical development programme involving around 400 patients and representing the largest ever carried out for this condition.

Genzyme's head of rare diseases Dr Rogerio Vivaldi said: "We are excited about this therapy's potential and are making excellent progress in our robust development plan for bringing eliglustat tartrate to the market."

This comes after Genzyme announced last month that Kynamro, the therapy for homozygous familial hypercholesterolaemia it has developed alongside Isis Pharmaceuticals, has been approved by US regulators.