Genzyme Corp. today planned to announce a program to helpType 1 Gaucher disease victims obtain Ceredase, the company'streatment that reverses symptoms of the inherited disorder ata cost of $50,000 to $100,000 per year.

Gaucher patients build up fat deposits in their macrophagewhite blood cells because they lack glucocerebrosidase (GCR),an enzyme that breaks down fatty acids. Symptoms includeanemia, enlarged liver or spleen, bleeding problems, bone andjoint pain, fatigue, and repeated fractures.

A 12-month clinical trial, reported in today's New EnglandJournal of Medicine, showed that Ceredase reduced anemia, aswell as liver and spleen enlargement associated with thedisease. Preliminary data suggested that Ceredase, a modifiedform of GCR, also improved skeletal problems caused by boneerosion.

The 12 patients, who are still undergoing treatment, "are doingmagnificently," Dr. Norman Barton told BioWorld. "Each has hada dramatic reversal of disease," he said. Barton and Dr. RoscoeBrady at the National Institutes of Health conducted the study.

About 10,000 Americans have inherited the defect, but manyhave no symptoms. Up to 3,000 people have moderate or severeforms of the disease. In April, Genzyme received Food and DrugAdministration approval and orphan drug status to treat thesepatients.

Genzyme estimates the cost of maintenance treatments at$50,000 a year and initial treatments for severe cases at$100,000 a year.

The clinical trial patients will be part of a study to identify alower dose of Ceredase that will prevent symptoms fromreoccurring.

Brady, a co-developer of the drug, said asymptomatic peoplealso should be treated with a maintenance dose to preventaccumulation of the fatty deposits that cause the symptoms.

A maintenance dose will cost only 10 percent to 20 percent asmuch as the dose used to reverse severe symptoms, hepredicted. He said the cost of treating adults in the clinicalstudy was more than $300,000 for the first year.

The company set up the patient assistance program to providefree treatments to patients who are uninsurable and cannotafford the drug. The program also helps patients obtainreimbursement and includes an education program to identifypatients with less severe Gaucher disease who can begintreatments at lower doses, said spokeswoman Jennifer Pierce.

Company scientists are developing a recombinant-derivedCeredase that Pierce predicted will be on the market in twoyears. It is too early to predict if the new drug will be lessexpensive.

Prudential Bache analyst Joe Edelman said that Wall Streetassumes the cost of Ceredase is $50,000. Investors do notrealize there is no proof yet that patients can be treated at themaintenance dose, he said.

Genzyme (NASDAQ:GENZ) closed Wednesday at $37, up $1.25.

-- Carol Talkington Verser, Ph.D. Special to BioWorld

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