Baxter Presents Interim Phase 1 Safety and Tolerability Data at American Society of Hematology Meeting for Its Investigational Treatment for von Willebrand Disease
European Commission and U.S. Food and Drug Administration Grant Orphan Drug Designation for Recombinant von Willebrand Factor
ORLANDO, Fla.--(BUSINESS WIRE)--Dec 6, 2010 - Recombinant von Willebrand factor (rVWF) may be safe and well tolerated in patients with type 3 and severe type 1 von Willebrand disease, according to interim data from a Phase 1 multicenter, international clinical study presented today at the 52nd Annual Meeting of the American Society of Hematology.1 The interim data from 22 patients in the 32-patient study also suggest rVWF has a pharmacokinetic profile that is comparable to plasma-derived von Willebrand factor (pdVWF),1 the current standard for treatment for patients with the disease. The condition is associated with prolonged bleeding times, and the resulting complications can, in some cases, be considered life-threatening.2
“Baxter is applying its scientific leadership in hemophilia to improve care for patients with other bleeding disorders,” said Bruce Ewenstein, M.D., Ph.D., vice president, clinical affairs at Baxter. “Providing patients and physicians with a plasma- and albumin-free, recombinant treatment option for a condition that is as challenging to treat as von Willebrand disease is an important focus for Baxter's clinical research and development efforts.”
About the Phase 1 clinical study
Safety, tolerability and pharmacokinetics of rVWF combined with rFVIII at a fixed ratio were investigated in the Phase 1 multicenter, international clinical study in 32 patients with type 3 von Willebrand disease and severe type 1 von Willebrand disease.1 Four concentrations of rVWF (2, 7.5, 20 and 50 IU VWF:RCo/kg) were administered in a dose-escalating manner in separate cohorts.1 Pharmacokinetics of rVWF/rFVIII compared with pdVWF/pdFVIII were evaluated in a sub-group of 22 patients using a randomized, cross-over design (eight-day minimum washout period).1 There were no thrombotic events, von Willebrand factor inhibitors or other serious adverse reactions observed.1 Final results from the Phase 1 clinical study will be available in 2011.
rVWF granted EC and FDA orphan designation
The European Commission granted orphan designation for Baxter's rVWF in November 2010. The European Medicines Agency's Committee for Orphan Medical Products adopted a positive opinion in September 2010, recommending the granting of this designation.2 Additionally, the U.S. Food and Drug Administration granted orphan designation for Baxter's rVWF in November 2010.3
Baxter's investigational rVWF concentrate was developed using a plasma- and albumin-free manufacturing method, and represents the largest protein ever produced through recombinant technology.1 This technology does not use blood components and may therefore address the safety concerns regarding the potential risk for transmission of pathogens in blood based additives. Currently available treatments for von Willebrand disease are derived from plasma. Baxter's rVWF is the only recombinant replacement protein in clinical development for von Willebrand disease.
About von Willebrand disease
Von Willebrand disease is the most common inherited bleeding disorder worldwide.9 People with von Willebrand disease have a deficiency in or impairment of a protein called von Willebrand factor, an important component in the blood clotting process.10 As a result, it takes longer for blood to clot and for bleeding to stop in people with von Willebrand disease.9 It is estimated that up to 1 percent of the world's population suffers from von Willebrand disease, but only a small percentage of people living with the condition have been diagnosed.9
Other rVWF data at ASH
Four additional posters related to the rVWF clinical program also were presented by Baxter at the Annual Meeting of the American Society of Hematology. Data from the studies described in the posters demonstrate that rVWF is structurally similar, and it is able to bind FVIII in a manner like that of pdVWF.5,6 These data lay a foundation for future clinical trials of rVWF in patients with von Willebrand disease.
- “Binding of FVIII to rVWF”5
- “Investigation of the morphology of rVWF by two high-resolution microscopic techniques”6
- “Toxico-kinetics of rVWF in non-human primates and rabbits”7
- “Cleavage of recombinant and plasma-derived VWF by human ADAMTS13”8
About Baxter International Inc.
Baxter International Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with hemophilia, immune disorders, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions. As a global, diversified healthcare company, Baxter applies a unique combination of expertise in medical devices, pharmaceuticals and biotechnology to create products that advance patient care worldwide.
This release includes forward-looking statements concerning the company's clinical efforts related to the use of recombinant von Willebrand factor in the treatment of von Willebrand disease. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: additional clinical results demonstrating the safety, tolerability and pharmacokinetics of recombinant von Willebrand factor; satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; and other risks identified in the company's most recent filing on Form 10-K and other SEC filings, all of which are available on the company's website. The company does not undertake to update its forward-looking statements.
1 Suiter T, et al. Recombinant human von Willebrand factor (rhVWF): first-in-human study evaluating pharmacokinetics, demonstrating safety and tolerability in type 3 von Willebrand disease. Oral presentation at 52nd Annual Meeting of the American Society of Hematology on Monday, December 6, 2010 at 7:30 a.m. EST. Orlando, Fla. December 4-7, 2010.
2 European Medicines Agency. Opinion of the Committee for Orphan Medicinal Products on orphan medicinal product designation. 9 September 2010.
3 Department of Health and Human Services, U.S. Food and Drug Administration. Designation Request #10-3222. 23 November 2010.
4 National Hemophilia Foundation. Von Willebrand Disease. Available at: http://hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=182&contentid=47&rptname=bleeding. Accessed on 30 November 2010.
5 Schrenk G. et al. Binding of FVIII to recombinant VWF. Abstract presented at American Society of Hematology Annual Meeting. December 2010.
6 Rottensteiner H, et al. Investigation of the morphology of recombinant von Willebrand factor by two high-resolution microscopic techniques. Abstract presented at American Society of Hematology Annual Meeting. December 2010.
7 Muchitsch EM, et al. Toxico-kinetics of recombinant VWF in non-human primates and rabbits. Abstract presented at American Society of Hematology Annual Meeting. December 2010.
8 Rottensteiner H, et al. Cleavage of recombinant and plasma-derived VWF by human ADAMTS13. Abstract presented at American Society of Hematology Annual Meeting. December 2010.
9 World Federation of Hemophilia. What is von Willebrand disease (VWD)? Available at: http://www.wfh.org/2/1/1_2_VWD_What-is-VWD.htm. Accessed on 10 November 2010.
10Mayo Foundation for Medical Education and Research (MFMER). Definition of von Willebrand Disease. Available at: http://www.mayoclinic.com/health/von-willebrand-disease/DS00903. Accessed on 3 December 2010.
Contact: Baxter International Inc.
Marie Kennedy, (805) 372-3543
Doreen Eaton, (805) 372-3417
Mary Kay Ladone, (847) 948-3371
Clare Trachtman, (847) 948-3085
Posted: December 2010