Genzyme Corp. said Monday that recombinant beta-glucocerebrosidase (r-GCR) has received orphan designationfrom the Food and Drug Administration to treat patients withtypes I, II and III Gaucher disease. The Cambridge, Mass.,company plans to begin Phase I trials in the first quarter.
Gaucher disease is a genetic disorder caused by insufficientactivity of the beta-glucocerebrosidase enzyme. Genzyme'sCeredase, which is derived from human tissue, received FDAmarketing approval in April for Type I Gaucher disease. Thegenetically engineered form will provide an alternative source.
Genzyme shares (NASDAQ:GENZ) rose $2.50, closing at $47.25.
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