Genzyme touts 2-year results for oral Gaucher drug

Genzyme touted the two-year results for its mid-stage study of a new oral pill for Gaucher disease, saying it looks comparable to its blockbuster Cerezyme.

Investigators say that eliglustat tartrate (formerly GENZ-112638) demonstrated its ability to strengthen bone, increase blood count and shrink bloated organs significantly. Swollen spleens decreased in size by an average of 52 percent with swollen livers shrinking 24 percent.

"This is not a head-to-head comparison, but based on the historical experience we've seen with Cerezyme the results look comparable," Genzyme Senior Vice President Geoff McDonough tells Reuters. Genzyme has begun enrolling patients for two late-stage trials of the therapy.

Genzyme has a lot riding on the oral therapy. Cerezyme is an infused product and a pill for the disease would quickly seize a significant share of the market at a time competitors like Protalix and Shire are introducing competitive therapies. Cerezyme costs about $200,000 a year per patient.

- check out the press release
- here's the story from Reuters