UPDATE 1-Genzyme's Gaucher disease pill promising in trial

By Toni Clarke

BOSTON, May 16 (Reuters) - Genzyme Corp. (GENZ.O: Quote, Profile, Research, Stock Buzz) said on Wednesday that its experimental pill to treat a rare genetic disorder known as Gaucher disease showed promise in a mid-stage clinical trial.

The Cambridge, Massachusetts-based biotechnology company said that, based on results seen so far from a Phase II trial of the drug, Genz-112638, the company plans to meet shortly with regulatory agencies in the United States and Europe to discuss an expedited development strategy.

Gaucher disease is caused by a specific enzyme deficiency in the body caused by a genetic mutation received from both parents. It can cause enlargement of the liver and spleen, anemia, bone pain and fractures.

Currently, Genzyme's own drug, Cerezyme, which is given once every two weeks by infusion, is the standard of care for treating the disease and generates annual sales of more than $1 billion.

But an effective oral drug would be highly desirable and is particularly important for Genzyme since Amicus Therapeutics, a rival company which filed in March to go public, is developing an oral drug that could compete with Cerezyme.

Amicus is also developing oral versions of several other Genzyme drugs, including Fabrazyme, a treatment for the rare genetic disorder Fabry disease and Myozyme, Genzyme's treatment for the rare muscle disorder Pompe disease.

Dr. David Meeker, head of Genzyme's rare genetic diseases unit, said that, while some patients on Cerezyme will likely respond to the new drug, others may not, allowing a role for both drugs and potentially a third if Amicus's drug is approved.