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Executive Summary

The approval of Baxter and Genetics Institute's Recombinate antihemophilic factor on Dec. 10 makes it the first recombinant Factor VIII blood clotting agent licensed by the agency. Recombinate is indicated in "hemophilia A (classical hemophilia) for the prevention and control of hemorrhagic episodes" and in "the perioperative management of patients with hemophilia A," labeling states. Recombinate also was approved by Canada's Health Protection Branch on the same day, becoming the second drug product in two weeks to be approved under joint, integrated review by FDA and its Canadian counterpart. Burroughs Wellcome's Mepron (atovaquone) was approved by the two government regulatory agencies on Nov. 25 ("The Pink Sheet" Dec. 7, T&G-4). Baxter submitted a PLA for Recombinate in May 1990. Baxter is taking orders for the rAHF product "immediately" and will begin shipping Recombinate on Dec. 21. The average wholesale price per unit of Recombinate is $1.18, with an estimated annual cost of treatment for an adult with "severe" hemophilia A ranging from $50,000-$70,000, Baxter said. The price range depends on factors including the cost of home health care services, the number of bleeding episodes each patient experiences and how often bleeding episodes are treated with rAHF. Recombinate will be available under Baxter's "Factor Plus" program for indigent patients "only in cases of medical emergency," the firm noted. However, Recombinate will not be available under the "Factor Assist" program for people whose insurance coverage has lapsed. Baxter's Hemophil M monoclonally- purified Factor VIII product is available under both programs. Recombinate will be manufactured under a 10-year R&D collaboration agreement between Baxter's Hyland Division and Genetics Institute. Under the agreement, Genetics Institute, majority-owned by American Home Products, will manufacture the bulk product while Baxter will make the final dosage form and market rAHF. Recombinate is Genetics Institute's first product to reach the market. The rAHF product is likely to be the centerpiece of Baxter's Biotech Group, which was formed earlier this year when Baxter renamed its Blood Group -- comprised of the Hyland and Fenwal Automated Systems divisions -- and added an Immunotherapy Division. The Biotech Group is headed by former Fenwal Division President Tim Anderson. Other projects for the Biotech Group include bone marrow transplant products. Under a separate cross-licensing agreement, Baxter/Genetics Institute avoid potential patent disputes over the rAHF technology with Genentech and its licensee, Miles ("The Pink Sheet" March 27, 1989, p. 5). Miles/Genentech's KoGENate recombinant Factor VIII product PLA is still pending at FDA. In December 1991, FDA's Blood Products Advisory Committee unanimously recommended approval of Recombinate despite reservations about data presented by Baxter on the rAHF. The advisory group noted that Recombinate data on 109 previously- treated patients from 26 centers in the U.S., Canada and Europe were not as complete as those presented by Miles for its product ("The Pink Sheet" Dec. 16, 1991, p. 14). Specifically, advisory committee members determined that data were inconclusive regarding Recombinate's safety in previously- untreated patients; Miles' KoGENate was deemed by the advisory committee to be safe for use in previously-untreated patients. Both Baxter and Miles were asked by the committee to conduct Phase IV studies to pinpoint the incidence and prevalence of inhibitors to their respective products and characterize the specific inhibitors to recombinant Factor VIII to determine if they have the potential to reduce the efficacy of Factor VIII products. Baxter says Phase IV studies for Recombinate have begun and will continue for either up to five years or 100 "exposure days," defined as one or more treatments within a 24-hour period. Prior to Recombinate approval, people with hemophilia A were often treated with Factor VIII concentrates made from human plasma, which in some cases transmitted HIV and hepatitis viruses to recipients. Noting these incidents, FDA Commissioner Kessler remarked in a Dec. 10 agency press release that Recombinate's approval "is a milestone in the history of treatment of hemophilia" and "eliminates even the theoretical possibility of the transmission of viruses from plasma." Since the beginning of clinical trials for Recombinate in 1987, the product has been evaluated in approximately 150 patients "with no reported cases of viral transmission or any unexpected complications," Baxter declared. The rAHF product also "has demonstrated a level of clotting activity comparable to that of blood-derived products and has been well-tolerated in patients," the firm added.

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