Navigation Links
CSL Behring Doses First Patient in Part 3 of Global Phase I/III Pivotal Study of rVIII-SingleChain (Recombinant Single-Chain Factor VIII) for Treatment of Hemophilia A

KING OF PRUSSIA, Pa., June 4, 2013 /PRNewswire/ -- CSL Behring has dosed the first patient in Part 3 of its AFFINITY clinical trial program, which is now in phase III. AFFINITY is an open-label, non-randomized, multi-center study evaluating the efficacy, safety and pharmacokinetics of its novel investigational recombinant coagulation single-chain factor VIII (rVIII-SingleChain) for the treatment of hemophilia A. The patient was dosed in Vienna, Austria and had a successful major surgery.

(Logo: )

The CSL Behring rVIII-SingleChain is a novel recombinant single-chain Factor VIII design that uses a strong, covalent bond that has been shown to improve the stability and half-life of factor VIII (FVIII). A recombinant factor therapy option with a longer half-life could provide a longer dosing interval, leading to less frequent injections. This may have the potential to improve compliance and ease prophylaxis, improving the quality of life for people with hemophilia A.

"We are proud and excited that this recombinant coagulation factor development program is progressing into Phase III clinical studies and look forward to sharing the clinical trial results for our novel rVIII-SingleChain molecule with the hemophilia community in the future," said Russell Basser , M.D., CSL Senior Vice President, Global Clinical Research & Development. "As part of our ongoing commitment to the hemophilia community, we are developing and investigating innovative recombinant factor therapies for the treatment of both hemophilia A and B that have the potential to offer patients and caregivers significant advances in treatment and convenience of factor infusion." CSL Behring, in collaboration with its parent company, CSL Limited (ASX: CSL), is developing rVIII-SingleChain through the AFFINITY clinical trial program.

About the AFFINITY Phase I/III Study 
The Phase I/III study is an open-label, multi-center trial that examines the crossover safety, efficacy and pharmacokinetics of recombinant coagulation single-chain factor VIII compared with recombinant human antihemophilic factor VIII (octocog alpha). 

In Part 1 of the study, 27 subjects received a single infusion of 50 IU/kg body weight (b.w.) of octocog alfa followed by a single infusion of 50 IU/kg b.w. rVIII-SingleChain. Results of Part 1 of the study will be presented at the 2013 International Society on Thrombosis and Haemostasis in Amsterdam, Netherlands on 2 July. In Parts 2 and 3 of the study, subjects will receive infusions of rVIII-SingleChain to prevent and treat bleeding (if required), at a dose and frequency determined by their study doctor (based on the subject's underlying bleeding phenotype). More information about the study design can be found at

Recombinant FVIII molecules so far available consist of a heavy and a light chain. Under certain conditions, these chains can dissociate, resulting in the formation of separated, or "dissociated," rFVIII chains that are not hemostatically active. The CSL Behring rVIII-SingleChain uses a strong, covalent bond that connects the light and heavy chains, thereby creating a stable single chain rFVIII.

In-house studies have shown that the molecular integrity of rVIII-SingleChain is significantly increased using the single-chain design, resulting in a homogenous product that is more stable than currently available FVIII products. In addition, in-vitro studies have shown that rVIII-SingleChain demonstrates a strong affinity for von Willebrand factor (VWF), resulting in a faster and more efficient binding to VWF. The FVIII/VWF complex plays an important role in the physiological activity and clearance of FVIII and has been shown to have an influence on the presentation of FVIII to the immune system.

The research leading to the initiation of the studies that CSL Behring is now conducting is the result of collaboration across the CSL Behring research sites in Marburg, Germany, in King of Prussia, USA, and at laboratories operated by CSL Limited in Melbourne, Australia.

About Hemophilia
Hemophilia is a congenital bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. In nearly all cases, it affects only males. The disease is caused by deficient or defective blood coagulation proteins known as factor VIII or IX (FIX). 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. Hemophilia A affects approximately 1 in 5,000 to 10,000 people. Hemophilia B affects approximately 1 in 25,000 to 50,000 people. The recommended treatment for patients who are factor deficient is to treat by replacement factor therapy.

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 manufactures and markets a range of plasma-derived and recombinant therapies worldwide.

CSL Behring therapies are used around the world to treat coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease, and neurological disorders in certain markets. The company's products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic diseases in the newborn. 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

Media Contact:

Sheila Burke
Director, Public Relations & Communications
Worldwide Commercial Operations
1 610-878-4209

Copyright©2012 PR Newswire.
All rights reserved

Related medicine technology :

1. CSL Behring Parent Company, CSL Limited, Issues Fourth Corporate Responsibility Report
2. CSL Behring Receives FDA Orphan Drug Designation for rIX-FP, a Novel Therapy in Development to Treat Congenital Factor IX Deficiency (Hemophilia B)
3. CSL Behring Announces FDA Filing of Biologics License Application for 4-Factor PCC for Urgent Reversal of Vitamin K Antagonist Anticoagulant Therapy
4. CSL Behring Initiates Study of Subcutaneous Administration of C1-esterase Inhibitor in Patients with Hereditary Angioedema
5. Summit Yields One-of-a-Kind Opportunity To Reduce Prescription Drug Overdoses
6. CLAAD Warns of Increase in Prescription Drug Overdoses
7. AcelRx Pharmaceuticals Doses the First Patients in ARX-04 Phase 2 Clinical Study
8. As Prescription Painkiller Overdoses Mount, Researchers Outline Effective Approaches to Curb Epidemic
9. DebMed to Display Clients Increased Hand Hygiene Compliance Rates and Worlds First Monitored Point-of-Care Dispenser at APIC
10. Cochlear Americas Receives FDA Approval for the First and Only Ear Level Accessory for Waterproof Hearing with Cochlear Implants
11. MEDIFIRST SOLUTIONS, INC. To Exhibit At The Orlando International Beauty Show
Post Your Comments:
(Date:6/23/2016)... , June 23, 2016 Research ... MEMS Devices Medical Market Analysis 2016 - Forecast to 2022" ... The report contains up to date financial data ... analysis. Assessment of major trends with potential impact on the ... analysis of market segmentation which comprises of sub markets, regional ...
(Date:6/23/2016)... INDIANAPOLIS , June 23, 2016 If ... Leaders Scholarship is any indication, the future is in ... at by the Diabetes Scholars Foundation ... the way of academic and community service excellence. ... since 2012, and continues to advocate for people with ...
(Date:6/23/2016)... 2016 Research and Markets has ... Global Analysis (United States, China, Japan, Brazil, United Kingdom, ... their offering. Surgical ... business planners, provides surgical procedure volume data in a ... an in-depth analysis of growth drivers and inhibitors, including ...
Breaking Medicine Technology:
(Date:6/25/2016)... ... ... closing of Bruton Memorial Library on June 21 due to a possible lice infestation, as ... of head lice: the parasite’s ability to live away from a human host, and to ... one in the event that lice have simply gotten out of control. , As lice ...
(Date:6/25/2016)... ... June 25, 2016 , ... As a lifelong Southern Californian, ... and his M.D from the David Geffen School of Medicine at UCLA. He trained ... Angeles to complete his fellowship in hematology/oncology at the UCLA-Olive View-Cedars Sinai program where ...
(Date:6/25/2016)... ... 2016 , ... Conventional wisdom preaches the benefits of moderation, whether it’s a ... the bar too high can result in disappointment, perhaps even self-loathing. However, those who ... , Research from reveals that behind the tendency to set ...
(Date:6/24/2016)... (PRWEB) , ... June 24, 2016 , ... June 19, ... the dangers associated with chronic pain and the benefits of holistic treatments, Serenity ... who are suffering with Sickle Cell Disease. , Sickle Cell Disease (SCD) is a ...
(Date:6/24/2016)... ... ... Global law firm Greenberg Traurig, P.A. announced that 20 Florida attorneys are ... for this recognition are considered among the top 2 percent of lawyers practicing within ... of this year’s Legal Elite Hall of Fame: Miami Shareholders Mark D. Bloom, ...
Breaking Medicine News(10 mins):