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Executive Summary

GENZYME's CEREDASE PATIENT POPULATION TO BE DISCUSSED as part of a scheduled FDA advisory committee review on Oct. 22. A preliminary agenda for the Endocrinologic and Metabolic Drugs Advisory Committee meeting asks: "Is it possible to define (for labeling purposes) a subject of the population with Type 1 Gaucher's disease that would benefit the most by receiving treatment with Ceredase?" The advisory committee will meet at the Ramada Inn in Bethesda, Maryland. The orphan product (glucocerebrosidase) is placenta-derived. According to FDA's preliminary questions to the advisory committee, the agency wants to examine the potential of a continuing supply problem for the product if it receives full approval. FDA refers to "the very limited availability of Ceredase at this time." FDA is also planning to seek the advisory committee's guidance on dosing levels. The agency wants to know if a subset of patients could be treated with a smaller dose. Genzyme says it did have a supply difficulty with the product when it first became available under a Treatment IND in late 1989. However, the firm maintains that it currently has the capacity to produce doses for up to 2,000 patients and that supply levels should not present a difficulty. Genzyme is working on producing Ceradex recombinantly, which would significantly increase supply in a few years. The Oct. 22 advisory committee meeting comes after an eleven-month Treatment IND period for the drug. Ceredase was okayed for Treatment IND use in late November 1989. Distribution of the product under the Treatment IND was complicated by protracted discussions with FDA over "reasonable charges." A price range ($20,000 to $60,000 per year) was finally agreed upon between FDA and the company in mid-June of this year. The firm has reportedly treated about five dozen patients during trials and under compassionate and Treatment INDs. The NDA for the product was submitted in January 1990. The supply issue may be clouded by varying estimates of the Gaucher's disease patient population in the U.S. The National Institute of Neurological Disorders and Stroke (NINDS) has extrapolated a potential patient population of 15,000 to 20,000 patients from calculations on the size of the ethnic group that experiences the most cases of Gaucher's disease. Genzyme officials have cited those figures in the past ("The Pink Sheet" May 29, 1989, p. 13) but now put a more conservative figure on the Type 1 patient population: 5,000 total with 2,000-3,000 symptomatic. Ceredase's NDA contains "one single-dose, dose-ranging study that demonstrates lysosomal changes and decreases in glucocerebroside in liver biopsy specimens, and one study of 12 Type 1 Gaucher's patients who were treated with repeated infusions over six months," FDA notes. In the latter study, patients' hemoglobin, hematocrit and organ sizes improved, the agency said. The 12 treated patients were compared to a non-randomized group of 12 adult patients. The advisory committee will be asked: "Are these data from small, non-randomized trials adequate to establish that benefits outweigh the risks for the use of Ceredase in Type 1 Gaucher's patients?"

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