The European Community's Committee for Proprietary MedicinalProducts (CPMP) has recommended approval of Genzyme Corp.'sCeredase enzyme-replacement therapy for patients with Type IGaucher's disease, the company said Tuesday.Genzyme of Cambridge, Mass., said it expects individual EC countriesto approve Ceredase within a few months. The company said it nowwill actively pursue marketing and reimbursement approval in ECcountries.About half of the 5,000 to 6,000 Gaucher's patients worldwide are inEurope, said Steve Push, Genzyme's vice president of corporatecommunications.Greg Phelps, the company's senior vice president, told BioWorld thatGenzyme is just beginning to open discussions in Europe concerningthe recombinant version of Ceredase, recombinant glucocerebosidase(r-GCR).Genzyme also said Tuesday that it is expecting FDA approval by Juneto market r-GCR in the U.S. In a Phase III trial, rGCR showed nosignificant differences to Ceredase (the natural version of the enzyme,which is derived from human placenta) in its ability to decrease hepaticor splenic volume observed or in the improvement of anemia orthrombocytopenia.Ceredase is often cited by members of Congress as an example of anexcessively priced breakthrough drug. The price of treatment is notexpected to go down significantly, if at all, in the early stages of thedrug's recombinant version. Phelps said the worldwide average of theannual cost of therapy is about $140,000. After patients get to amaintenance dose, the annual cost drops to between $20,000 and$60,000, he said."We have discouraged people from expecting price reductions at thisstage," Phelps said, adding that the cost of producing the drug at itssmall plant in Framingham, Mass., prohibits cost cutting.But Genzyme said it is hoping to get regulatory approval on a largerplant it is readying in Boston. It hopes to produce and sell r-GCR fromthe new plant in 1995. Phelps said the worldwide supply of the drugcould be produced there and cost reductions realized at that point.

-- Jim Shrine

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