Factor VIII--Baxter: rAHF-PFM, recombinant anti-haemophilic factor--protein-free method, recombinant factor...

Baxter Healthcare is developing a next-generation recombinant factor VIII for the treatment of haemophilia A that is produced using a totally protein-free manufacturing process. By excluding proteins or raw materials derived from human or animal sources in the final product, the risk of transmission of potentially infectious agents is removed. All of the recombinant factor VIII products currently on the market incorporate proteins or raw materials derived from either human or animal sources, as part of the nutrients required for the cells to produce the protein. Baxter has exclusive rights to Quadrant Healthcare's factor VIII stabilisation technology. Quadrant received 1 million US dollars under the agreement, and was to receive milestone payments in excess of 2 million US dollars, together with royalties on sales should a new factor VIII product using Quadrant's technology reach the market. In December 2000, Quadrant Healthcare was acquired by Elan Drug Delivery (Elan Corporation), which continued to develop the proprietary formulation and stabilisation technologies of Quadrant. In July 2003, the newly formed company Quadrant Technologies acquired Elan Drug Delivery from Elan Corporation and renamed it Quadrant Drug Delivery. In July 2003, the US FDA approved the use of ADVATE for the prevention and control of bleeding episodes in people with haemophilia A. At the Bear Stearns 16th Annual Healthcare Conference held in September 2003, Baxter reported that ADVATE was launched in August 2003 in the US. A pivotal phase III clinical trial of this recombinant factor VIII therapy, initiated in 89 previously untreated patients with haemophilia A, has been completed at sites in the US, UK, Sweden, France, Belgium, Germany, Denmark and Italy. This trial compared the pharmacokinetics, tolerability and immunogenicity of this therapy with Baxter's currently marketed recombinant factor VIII preparation, Recombinate trade mark. The first clinical data that were presented on this agent at the XXV International Congress of the World Federation of Hemophilia meeting indicate that Baxter's next-generation recombinant factor VIII is bioequivalant with Recombinate trade mark. The two agents should have comparable efficacy. A Marketing Authorisation Application (MAA) was submitted to the EU EMEA and regulatory filings were made in Canada in September 2002. The submission was made via the centralised procedure. Should the new-generation product reach the market, Baxter will continue to offer Recombinate trade mark as well as plasma-derived factor VIII products. Preliminary study results announced in July 2003 show that factor VIII therapy was effective in controlling bleeding episodes in patients with severe and moderately severe haemophilia A who were previously treated, or had undergone surgery.