Positive trial for new Genzyme drug
Genzyme Corp. saw positive results in a small human trial of a new pill designed to fight Gaucher disease, a rare genetic disorder currently treated with Cerezyme, the Cambridge company's expensive intravenous biotechnology drug.
In five patients tested, Genzyme said its experimental pill helped improve the severe spleen and liver swelling that is characteristic of Gaucher disease. The company said one patient suffered a health problem that may be related to the drug. Based on the trial, which will ultimately include at least 19 patients, Genzyme plans to ask federal regulators to speed approval of its new pill.
Genzyme currently makes $1 billion a year treating Gaucher patients with Cerezyme, a replacement enzyme manufactured at its Allston factory, and is trying to develop a next-generation pill that will be easier to administer.
(By Stephen Heuser, Globe staff)







