Genzyme Corp. will launch Ceredase, an orphan drug that is thecompany's first biotherapeutic product, next Monday, aftergetting a green light from the U.S. Food and DrugAdministration.
The FDA has approved Ceredase to treat Type 1 Gaucher'sdisease, according to Alison Taunton-Rigby, Ph.D., Genzyme'ssenior vice president of biotherapeutics.
Genzyme, based in Boston, estimates that 2,000 to 3,000 peoplein the United States and about 5,000 worldwide are severelyaffected with Type 1 Gaucher's disease. Genzyme expects totreat 500 to 1,000 patients in 1991 and to be able to treat allU.S. patients within two years, said Taunton-Rigby.
Ceredase will be administered intravenously every two weeks.Genzyme estimates the average cost for a year's treatment tobe between $20,000 and $60,000 per patient, depending on theindividual's size and the severity of the disease.
The company expects $25 million to $50 million in revenuesfrom Ceredase in 1991, expanding to between $80 million and$100 million in 1992, Taunton-Rigby said.
Gaucher's patients build up fat deposits in their macrophagewhite blood cells due to the lack of glucocerebrosidase, anenzyme that breaks down fatty acids. Symptoms include anenlarged liver or spleen, anemia, bleeding problems, bone andjoint pain, fatigue, repeated fractures and bone erosion.
Ceredase is a modified form of the enzyme that is recognizedand internalized by the macrophages. Once inside, Ceredasebreaks down the accumulated fatty acids.
Genzyme purifies glucocerebrosidase from human placentasand converts it into Ceredase by placing mannose sugar groupson the protein. The mannose groups target Ceredase to themacrophages that have mannose receptors.
Company scientists are developing a recombinant-derivedCeredase that Taunton-Rigby predicts will be on the market intwo years. The new version will allow Genzyme to expandworldwide treatment of Gaucher's patients.
David Stone, an analyst at Cowen & Co., expects sales ofCeredase to make Genzyme profitable for the first time since itsmerger with Integrated Genetics. He added that Genzyme has a"very solid situation with Ceredase because there is nocompeting product and because the company has lots of otherproducts in the pipeline."
Most analysts will wait one or two quarters to see if Genzymerealizes the profits it expects, he added. Genzyme (NASDAQ:GENZ) stock closed unchanged Monday at $38.50.
-- Carol Talkington Verser, Ph.D. Special to BioWorld
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