UPDATE 1-Genzyme's Gaucher disease pill promising in trial
By Toni Clarke
BOSTON, May 16 (Reuters) - Genzyme Corp. (GENZ.O) 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.
"In the end, which drug comes out on top will all depend on safety and efficacy," he said.
Genz-112638 works through a different mechanism than Cerezyme. Whereas Cerezyme replaces an enzyme that is needed to break down excess fats in the cells and tissues of patients with Gaucher disease, the new drug blocks the enzyme that makes the fats in the first place, Meeker said in an interview.
Initial data came from the first five patients in the trial, which enrolled 20 people. Full results of the trial will be available in mid-2008.
"The number is small but in the rare disease world it is a meaningful number," Meeker said.
In addition, he said, the efficacy results "are in the range of what we have seen with Cerezyme."
About 10,000 people suffer from Gaucher disease worldwide, of which about 5,000 are treated with Cerezyme, Meeker said.
Genzyme said the side effects seen with its new drug were generally mild. However, one patient experienced an abnormal heart rhythm. Meeker said the company is looking into whether the abnormality was caused by the drug or whether it was already part of the patient's background physical makeup.
((Reporting by Toni Clarke, editing by Andre Grenon; Reuters Messaging, toni.clarke.reuters.com@reuters.net +1-617-367- 4165)) Keywords: GENZYME/
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