Positive phase 3 data for Genzyme rare disease drug

Hannah Blake


Sanofi’s Genzyme has announced that phase 3 clinical trials of its investigational oral therapy for Gaucher disease type 1 met their primary endpoints.

The two phase 3 studies ENGAGE and ENCORE were evaluating the safety and efficacy of eliglustat tartrate, which is being developed to provide a convenient treatment alternative for rare disease patients with Gaucher disease type 1 and to provide a broader range of treatment options for patients and physicians. Both sets of results were presented last week at the 9th Annual Lysosomal Disease Network WORLD Symposium in Orlando, Fla.

Both studies met their primary efficacy endpoints and together will

form the basis of Genzyme’s registration package for eliglustat tartrate.

“The data presented at this year’s WORLD symposium reinforce our confidence that eliglustat tartrate may become an important oral option for patients with Gaucher disease. 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.”

Genzyme’s Head of Rare Diseases, Rogerio Vivaldi MD.

Genzyme’s clinical development program for eliglustat tartrate represents the largest clinical program ever focused on Gaucher disease type 1 with approximately 400 patients treated in 30 countries.

Gaucher disease is an inherited condition affecting fewer than 10,000 people worldwide and is therefore classed as a rare disease.


Related news:

Sanofi says Gaucher pill studies meet goals (Reuters)

Reference links:

Genzyme press release

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