2015 News Releases
Global biotherapeutics leader CSL Behring announced today that the company has submitted its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its novel investigational recombinant factor VIII single-chain (rVIII-SingleChain) for the treatment of hemophilia A. In the pivotal clinical trial, rVIII-SingleChain met all primary endpoints.
Global biotherapeutics leader CSL Behring announced today that the company has submitted its new drug application to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for its investigational fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP). rIX-FP is a long-acting recombinant albumin fusion protein for people with hemophilia B, a congenital bleeding disorder characterized by deficient or defective factor IX. Based on the 2011 National Survey of Coagulation Disorder, hemophilia B occurs in approximately 1 to 2 of every 100,000 male births in Japan.
CSL Limited (ASX:CSL) has recorded another strong performance in corporate responsibility, delivering on its promise to responsibly achieve growth through the disciplined execution of its business strategy, investing in research and development to advance therapies for unmet medical needs, growing its core portfolio of products by expanding indications and markets, and through its commitment to productivity and efficiency.
Global biotherapeutics leader CSL Behring today presented data from its Phase III PROLONG-9FP clinical program evaluating the efficacy and long-term safety of its investigational long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP). The data, from an ongoing extension study and two pivotal Phase III studies, assessed rIX-FP for routine prophylaxis in previously-treated adults with hemophilia B, at dosing intervals of up to 14 days. The findings were presented during an oral presentation at the American Society of Hematology’s (ASH) 57th ASH Annual Meeting and Exposition in Orlando, along with a second abstract reporting efficacy and safety results of rIX-FP in patients undergoing surgical procedures.
CSL Behring announced today that it will present several abstracts from its recombinant coagulation factor development programs at the American Society of Hematology’s (ASH) 57th ASH Annual Meeting in Orlando, December 5-8. An oral presentation will focus on the pivotal trial for CSL Behring’s novel, long-acting albumin fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP) for the treatment of hemophilia B.
CSL Behring announced that Tyler Reitano from the Cascade Foundation of Southern Arizona and Maklain Briggs from the Utah Hemophilia Foundation are the national winners of the 2015 Gettin’ in the GameSM Junior National Championship (JNC) program in golf and baseball, respectively. The JNC, launched in 2002, is CSL Behring’s annual baseball and golf competition that encourages kids to remain active despite the challenges a bleeding disorder can pose, while allowing them to develop life-long connections with other members of the community.
CSL Behring today hosted a symposium highlighting an option to slow the progression of emphysema in adults with documented severe alpha-1 antitrypsin deficiency (AATD), during the European Respiratory Society (ERS) International Congress, which is being held from 26-30 September in Amsterdam, Netherlands. AATD is a hereditary condition marked by a lack of the alpha-1 antitrypsin protein, whose main function is to protect the lungs from inflammation
CSL Behring today announced that the Centers for Medicare and Medicaid Services (CMS) again extended the new technology add-on payment (NTAP) for Kcentra® (Prothrombin Complex Concentrate [Human]). The NTAP for Kcentra is available through September 2016 for eligible Medicare beneficiaries treated in the inpatient hospital setting. Kcentra is the first and only non-activated 4-factor prothrombin complex concentrate (4F-PCC) approved by the U.S. Food and Drug Administration (FDA) for the urgent reversal of acquired coagulation factor deficiency induced by Vitamin K antagonist (VKA, e.g., warfarin) therapy in adult patients with acute major bleeding or in need of an urgent surgery or invasive procedure. Kcentra, first approved for use in the U.S. in April 2013, received its NTAP designation effective October 1, 2013.
CSL Behring announced today that the first patient has been enrolled in its Phase II/III clinical study evaluating the pharmacokinetics (PK), efficacy, and safety of the company’s recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP) for on-demand treatment in patients with congenital hemophilia A or B who have developed an inhibitor to factor VIII or factor IX replacement therapy. The study will enroll approximately 54 male patients, the first of whom was enrolled in Malaysia.
Global biotherapeutics leader CSL Behring announced today its latest geographic expansion to provide more patients with greater access to treatment by opening operations in Russia. This is particularly significant in Russia where the healthcare system has some unmet needs for state-of-the-art biotherapies and blood plasma products.
CSL Behring announced today that Swissmedic has accepted for review a Marketing Authorization Application (MAA) for the company’s investigational long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP) for the prophylaxis and treatment of bleeding episodes in people with Hemophilia B. Upon Swissmedic approval, rIX-FP will provide hemophilia B patients with a long-acting treatment option with dosing intervals up to 14 days.
CSL Behring announced today that the U.S. Food and Drug Administration has accepted for review the company’s Biologics License Application (BLA) for its novel investigational recombinant factor VIII single-chain (rVIII-SingleChain) for the treatment of hemophilia A. In the pivotal clinical trial, rVIII-SingleChain met all primary endpoints.
CSL Behring announced today that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorization for Respreeza®, a highly purified alpha-1 protein derived from human plasma, indicated to treat patients with alpha-1 antitrypsin deficiency (AATD). AATD is a hereditary condition marked by a lack of the alpha-1 antitrypsin protein, whose main function is to protect the lungs from inflammation. Respreeza® replaces the protein that these patients are missing and raises the alpha-1 antitrypsin levels in their blood, which can help to protect the lungs from damage due to inflammation.
CSL Behring announced today that Mohammad Alsharabati, MD, University of Alabama at Birmingham, United States, is the recipient of the 2015 Interlaken Leadership Award for original research in the field of neuroimmunology. This annual global awards program provides monetary grants and/or product supply for investigational use to support research focusing on the potential role of immunoglobulin (Ig) therapy in the treatment of neurological disorders. The global review committee seeks proposals likely to advance innovative medical research and knowledge about the potential role of Ig therapy to improve the lives of patients who have disabling neurological conditions.
CSL Behring today presented data from a Phase I/III study on the efficacy and safety of its novel investigational recombinant factor VIII single chain (rVIII-SingleChain) in adolescents and adults with hemophilia A during a late breaking abstract session at the 2015 International Society on Thrombosis and Haemostasis (ISTH) Congress. Overall, patients using rVIII-SingleChain to prevent bleeding (prophylaxis) were well controlled with two to three infusions per week and developed no inhibitors.
CSL Behring today presented data from Phase III studies evaluating the efficacy and long-term safety of its investigational long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP). The data, shared in three separate oral presentations at the 2015 International Society on Thrombosis and Haemostasis (ISTH) Congress in Toronto, support the use of rIX-FP for routine prophylaxis, dosed once up to every 14 days, and for on-demand treatment of bleeding episodes in previously-treated adults and children with hemophilia B. The findings also include efficacy and safety results supporting the use of rIX-FP in patients undergoing surgical procedures.
The cost of medicines in general and biopharmaceutical therapies used to treat rare diseases in particular has faced increased attention from payers and other stakeholders in recent years.
CSL Behring announced today it will present more than 20 abstracts, including five oral presentations, from across its hematology portfolio of investigational and branded products at the 2015 International Society on Thrombosis and Haemostasis (ISTH) Congress, being held in Toronto June 20-25. The presentations will include pivotal trial data for two of its late-stage recombinant products – its novel recombinant factor VIII SingleChain (rVIII-SingleChain) compound for hemophilia A and its long-acting recombinant factor IX albumin fusion protein (rIX-FP) for hemophilia B.
Findings from CSL Behring’s RAPID study, the largest placebo-controlled trial ever conducted in patients with alpha-1 antitrypsin deficiency (AATD), demonstrate that the use of Alpha1-Proteinase Inhibitor therapy may slow the progressive loss of lung tissue experienced by these critically ill patients. According to findings of RAPID (Randomized, Placebo-controlled Trial of Augmentation Therapy in Alpha-1 Proteinase Inhibitor Deficiency), published by The Lancet, patients with AATD treated with Alpha1-Proteinase Inhibitor therapy exhibited a lower annual rate of lung density decline compared to placebo, when measured using chest computed tomography, at full inspiration.
Scientific advancements, more readily available medical information and emerging technologies are driving greater patient engagement, improving diagnosis and treatment, and enabling people with life-threatening diseases to live longer and healthier lives, said CSL Limited CEO and Managing Director Paul Perreault. CSL is the parent company of CSL Behring.