CSL Behring announced today the start of the global clinical trial program SWIFT (Studies with von Willebrand factor [VWF]/Factor VIII) to evaluate the pharmacokinetics, efficacy and safety of Biostate®, a low-volume, highly active, plasma-derived VWF/FVIII concentrate for the treatment of von Willebrand disease (VWD) and hemophilia A. The methodology behind the multi-center trials was detailed in a presentation at the 3rd Annual Congress of the European Association of Haemophilia and Allied Disorders (EAHAD) today.
“The management of patients with bleeding disorders like VWD and hemophilia A continues to be challenging, and we welcome new therapeutic options,” said Professor Toshko Lissitchkov, M.D. of Specialized Haematological Hospital Joan Pavel. “Our goal with the SWIFT program is to evaluate the safety and efficacy of Biostate, a factor concentrate with high specific activity that has the potential to benefit patients.”
The SWIFT program, which will include centers in Europe, North America and South America, comprises four open-label studies in adults/adolescents and children with VWD and hemophilia A. CSL Behring is performing the SWIFT studies to meet the regulatory requirements for making Biostate available in multiple countries. Biostate has been available in Australia since 2003 where it is indicated for the treatment of von Willebrand disease and hemophilia A.
“CSL Behring has a heritage of innovation in developing bleeding disorder therapies and making a difference in patients’ lives,” said Russell Basser, M.D., Senior Vice President, Global Clinical Research, CSL Behring. “CSL Behring’s commitment to the hemophilia and von Willebrand disease community will be further strengthened by Biostate’s development and approval. It will provide physicians and patients with another option in the management of bleeding disorders, joining CSL Behring’s Haemate® P/Humate-P® and other therapies.”
About the SWIFT Program
The SWIFT (Studies with von Willebrand factor [VWF]/Factor VIII) program comprises four open-label, multicenter studies to evaluate the pharmacokinetics, efficacy and safety of Biostate®, a low-volume, highly-active, VWF/FVIII concentrate for the treatment of von Willebrand disease (VWD) and hemophilia A in both adult and pediatric patients. Patient criteria for all four studies are as follows:
- SWIFT-VWD will include patients at least 12 years of age diagnosed with von Willebrand disease (VWF: RCo less than 15 percent) and who require a VWF product to manage their VWD.
- SWIFTLY-VWD will include pediatric patients (0-11 years of age) for whom treatment with a VWF replacement therapy is required for prophylactic therapy, hemostatic control during surgery or control of a non-surgical, spontaneous or traumatic bleeding event. Patients who are diagnosed with type 1, 2A or 3 VWD and a VWF: RCo level less than 15 percent are eligible.
- SWIFT-HA will include patients at least 12 years of age diagnosed with hemophilia A, including previously treated male patients with severe hemophilia (FVIII: C activity less than 1 percent).
- SWIFTLY-HA will include pediatric patients with hemophilia A, including previously treated patients with severe hemophilia A (FVIII: C activity less than 1 percent).
About von Willebrand Disease
Von Willebrand disease is caused by a deficiency or abnormality of von Willebrand factor (VWF), a protein in the blood that is necessary for normal blood clotting. VWD is classified by types, ranging from Type 1 (the most common and usually mild) to Type 3 (the most severe). Patients with Type 2 VWD have VWF that does not work properly, while patients with Type 1 or Type 3 do not have enough VWF to produce a normal clot. Women with VWD are more likely to experience heavy, prolonged menstruation. Other common symptoms of VWD include frequent nosebleeds and easy bruising. Bleeding can be mild or serious and can occur as a result of injury, or without any obvious cause. More serious symptoms include bleeding into joints and internal organs. VWD patients may require special care during dental procedures, surgery and childbirth.
Hemophilia is an inherited bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. The disease is caused by deficient or defective blood coagulation proteins known as factor VIII or IX. The most common form of the disease is hemophilia A, or classic hemophilia, in which the clotting factor VIII is either deficient or defective. Hemophilia B is characterized by deficient or defective factor IX.
Biostate is a low-volume, highly active, plasma-derived VWF/FVIII concentrate produced by a sophisticated manufacturing process that uses two virus inactivation steps. Biostate contains high molecular weight multimers of VWF. It has been shown that high molecular weight multimers are associated with effective hemostasis and play a role in platelet adhesion, binding more efficiently to platelets than do low molecular weight VWF multimers. Biostate has a VWF:FVIII ratio of approximately 2.2:1. Biostate is not currently licensed in Europe or the United States.
About CSL Behring
CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufacturers and markets a range of plasma-derived and recombinant therapies worldwide. CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies and inherited respiratory disease. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic diseases in newborns. CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited (ASX: CSL), a biopharmaceutical company headquartered in Melbourne, Australia. For more information, visit www.cslbehring.com.
Sheila A. Burke, Director,
Communications & Public Relations
Worldwide Commercial Operations
MCS Healthcare Public Relations