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Executive Summary

Genzyme expects soon to add "a dozen" Gaucher patients to Ceredase (alglucerase) treatment following the product's final clearance by FDA on April 5. Those patients had not been able to receive reimbursement for the drug under Treatment IND distribution initiated in November 1989, but Genzyme said that they have been precleared for reimbursement from their insurers following FDA approval. Genzyme has been treating 60-70 patients under the Treatment IND. The company has an additional 20-30 patients "in the reimbursement cycle," the company noted. The insurers for those patients have already been contacted about use of the drug. Altogether, the firm appears close to having 100 patients on the chronic treatment regimen. The total patient population is estimated to be 2,000-3,000. The company requires preclearance from an insurer before it will begin shipping Ceredase for use by a patient. However, "uninsurable" patients will still have access to the drug, the company maintained. The reimbursement issue is crucial to Genzyme because of the high cost of the product and the chronic use pattern of treatment. The company estimates that drug costs for patients on maintenance therapy with alglucerase will be between $ 20,000 and $ 60,000 per patient per year. The drug price is $ 3.50 per unit, Genzyme said, with 400 units in each 5 ml vial. Dosage varies, with initial doses ranging up to 60 units/kg and then gradually declines to the maintenance dose, which could be as low as 1 unit/kg, the approved label states. Administration is by I.V. infusion about once every two weeks. The new price reflects a 17% increase over the Treatment IND charges: the cost of the drug had been $ 3 per unit. That price had been set last June after discussions with FDA about a "reasonable charge" ("The Pink Sheet" July 2, T&G-1). The increase will cover sales and marketing costs and ensure that the company receives a return on its investment, Genzyme said. The approved product will begin reaching hospitals on April 15. Genzyme said it will guarantee the $ 3.50 per unit price by covering any markups in the distribution of the product. In an April 8 HHS press release announcing the approval, FDA Commissioner Kessler said "alglucerase shows how an important drug can successfully be made available early to desperately ill people and then be reviewed by FDA in less than a year." Genzyme's "rolling" NDA was officially filed April 14, 1990. Ceredase was recommended for approval at an Oct. 22 Endocrinologic & Metabolic Drugs Advisory Committee meeting, just 10 months after Genzyme began submitting data for its NDA ("The Pink Sheet" Oct. 29, p. 13). Final approval, however, took another five-and-a-half months. The delay may have been attributable to an inspection of the sterile filing and bottling facilities for Ceredase at the Albuquerque, N.M. plant of Adria Labs. An FDA inspection at Adria was recently completed. Ceredase will be marketed by Genzyme's eight-person sales team and six- to eight-person marketing and reimbursement group, Genzyme said. More people are being added to the reimbursement group. Genzyme does not appear to face too difficult a challenge with acceptance of the product, despite the high annual cost. Blue Cross/Blue Shield noted, for example, that its policy is to reimburse drugs with FDA approved indications regardless of cost. The Health Insurance Association of America (HIAA) said that its member insurers generally have the same policy and would consider cost only in cases where competing therapies existed. One possible long-term complication, HIAA noted, is that most plans include a maximum lifetime payment to any individual, usually $ 1 mil. Considering the total cost of therapy in addition to just drug costs and the young age of most Gaucher patients, that limit could be reached very quickly. Ultimately, both HIAA and Blue Cross/Blue Shield noted, as expensive drugs like Ceredase become more prevalent, premium increases may be tied to their coverage. That would put the decision for coverage on corporate/employer clients. The Ceredase approval and a pending public offering by Enzon dramatize the increasingly high cost of biotech treatments. Enzon disclosed in its recent filing with the Securities & Exchange Commission that drug costs for Adagen will exceed $ 100,000 per patient per year (see related story, p. 14). Amgen's Neupogen, as an adjunct for a six-cycle course of chemotherapy, is estimated to cost $ 6,500-$ 14,500 ("The Pink Sheet" Feb. 25, p. 5). Genentech reportedly has led the investment community to believe that several of its next generation of products (such as DNase for cystic fibrosis) would cost over $ 5,000 per year. Ceredase approved labeling indicates the product "for use as a long-term enzyme replacement therapy for patients with a confirmed diagnosis of Type I Gaucher disease." It is only to be used for patients "who exhibit signs and symptoms that are severe enough" to result in one or more of four conditions: moderate-to-severe anemia, thrombocytopenia with bleeding tendency, bone disease, or significant hepatomegaly or splenomegaly, the label says. There are no contraindications. The listing of specific symptoms in the indication stems from the Oct. 22 advisory committee meeting, where FDA requested that the committee help to define specific subsets of the total Gaucher population that need treatment. The genetic disorder may be present in 10,000-15,000 people in the U.S., but only 2,000-3,000 are symptomatic. The National Gaucher Foundation (NGF) expressed concern that the drug will not be available to asymptomatic patients, both because of the label and the high price. The disease can strike asymptomatic patients at any time, and low dose Ceredase treatment could be valuable for preventing the onset of symptoms, NGF said. NGF noted that the National Institutes of Health is conducting a minimum dosage study with Ceredase. While "thrilled" by the approval, NGF expressed mixed emotions about the drug's price. Patients "will be okay for a year," NGF said, but then will begin to exhaust their insurance. Severely ill patients may pay $ 200,000 per year or more in drug costs alone, NGF said, and so will reach their insurance caps very quickly. The Oct. 22 advisory committee also expressed concern about the possible transmission of viruses, especially Creutzfeldt-Jakob Disease (CJD), by the placenta-derived product. Genzyme told the committee that the risk is only "theoretical" and that a recombinant product is being developed. Approved labeling notes that "the risk of contamination from slowly acting or latent viruses, including the Creutzfeldt-Jakob disease agent, is believed to be remote but has not been tested. Accordingly, the benefits and the risks of treatment with this product should be assessed prior to use." FDA's approval letter says that Genzyme has "committed to redoing the pharmacokinetic study with the marketed formulation as part of . . . post-marketing studies." The original pharmacokinetic studies were performed at NIH, which uses a different assay than the one used at Genzyme. The company will redo those studies as part of the clinicals for the recombinant form of the product, Genzyme said. Genzyme also is committed "to submit, when available, the information regarding the analytical methods" used in the production of Ceredase, FDA's letter says. As a "post-marketing study," Genzyme is tracking all of its Gaucher patients through the International Collaborative Gaucher Study, which will supply data to physicians to guide in dosing their patients. That study is ongoing, the company said. Ceredase's approval follows a $ 124 mil. public offering completed by Genzyme March 22. Initially intended as a 3 mil. share offering, it was expanded to 3.5 mil. shares at $ 35.50 "due to strong investor demand," the company said.

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