A new study conducted in Japan supports the previously demonstrated safety and efficacy of Hizentra® (Immune Globulin Subcutaneous [Human]) for the treatment of primary immunodeficiency (PID). The data were presented today at the 15th Biennial Meeting of the European Society for Immunodeficiencies (ESID). Hizentra is the first and only 20 percent subcutaneous immunoglobulin (SCIg) therapy in the world for the treatment of PID, a rare and serious group of diseases of the immune system. It is the first-ever SCIg therapy to demonstrate safety and efficacy in Japanese subjects. Based on these excellent results, CSL Behring submitted the new drug application (NDA) for Hizentra to the Pharmaceutical and Medicines Devices Agency (PMDA) in Japan on 28 September, 2012.
The Phase III study, conducted in Japanese patients who converted from intravenous immunoglobulin (IVIG) treatment, found that a dose-equivalent switch to Hizentra therapy maintained serum IgG (immunoglobulin) at a similar level of trough concentration than previous IVIG therapy. Results showed that Hizentra provided effective passive immunity in adults and children to control most recurrent infections and improved patients’ overall quality of life.
“Our study is the first to investigate the safety and efficacy of a subcutaneous immunoglobulin product in Japanese patients,” said Toshio Miyawaki, M.D., Department of Pediatrics, Graduate School of Medicine and Pharmaceutical Sciences, University of Toyama in Japan, and Coordination Investigator of the study. “Our findings substantiate the results obtained in previous European and North American trials, and serve to confirm that Hizentra is an effective and well-tolerated treatment option for primary immunodeficiency.
About Primary Immunodeficiencies
Primary immunodeficiency (PID) is a group of over 150 diseases that affect the cells, tissues and proteins of the immune system. In people with PID, the immune system is either absent or functioning inadequately, leaving them more susceptible to infection. For individuals with PID – many of them children – infections may not improve with treatment as expected and may keep returning. As a result, patients may face repeated rounds of antibiotics and hospitalization for treatment. Repeated infections can lead to organ damage, which over time can become life threatening.
Collectively, PIDs affect an estimated 10 million people worldwide, and the incidence is estimated to be 1 in 10,000. For more information on PID, please visit www.cslbehring.com. For patients with PID, immunoglobulin replacement therapy with a product such as Hizentra can help treat existing or chronic infections and prevent new infections from occurring. No single treatment works for every type of PID, but infusions of replacement antibodies (immunoglobulins) can help supplement the immune system to prevent infection in nearly 75 percent of PID cases that are due to antibody deficiencies.
Immunoglobulin is a blood component that has become standard immune replacement therapy for more people living with PID, and nearly 70 percent of PID patients receive Ig replacement therapy. Since the 1980s, the first-line therapy for most PID patients has been intravenous immunoglobulin (IVIg), which immunoglobulin is delivered through a needle into the vein. Some patients, however, cannot easily tolerate intravenous infusions due to serious side effects or poor venous access. Hizentra allows patients to use a small, portable pump to self-administer their infusions by injection under the skin (subcutaneous administrations).
Study Design and Key Findings
Patients were switched using 1:1 dose conversion. In this prospective multicenter, open-label, single-arm Phase III study of Hizentra, 24 Japanese patients with PID, who required IgG replacement therapy, were evaluated following an IVIG treatment period. Patients received three infusions of IVIG therapy, followed by a 12-week wash-in/wash-out period where treatment was converted to Hizentra and a 12-week efficacy period. The primary endpoint was achievement of the total serum IgG trough levels with SCIg therapy as compared to the preceding IVIG treatment period. Secondary efficacy and safety endpoints included all infections and local reactions to treatment and adverse events (AEs).
Findings demonstrated that Hizentra produced serum IgG trough concentrations higher than those seen during previous IVIG therapy. Mean serum IgG levels increased from 6.51 (1.32) g/L in the IVIG period to 7.28 (1.47) g/L in the SCIg efficacy period. During the efficacy period, no serious bacterial infections were reported; 11 patients experienced a non-serious infection. Ninety-six percent of the patients enrolled experience at least one AE; most common AEs were local reactions of mild intensity. No related serious AEs were reported.
Hizentra (Immune Globulin Subcutaneous [Human]), the first and only 20 percent SCIg developed for subcutaneous use, is already approved in North American and Europe. It is stable at 25 degrees Celcius for at least 24 months due to formulation with L-proline. In the United States, Hizentra is indicated for the treatment of patients with PID, and contraindicated in individuals with a history of anaphylactic or severe systemic response to immune globulin preparations or components of Hizentra, and in persons with selective immunoglobulin A deficiency who have known antibody against IgA and a history of hypersensitivity. For more information, including full U.S. prescribing information, visit http://www.hizentra.com/. In 2011, the European Commission granted marketing authorization for Hizentra for treating patients diagnosed with PID as well as secondary imunodeficiencies. The authorization is valid for all 29 European/European Economic Area member states.
About CSL Behring
CSL Behring is a global leader in the plasma protein biotherapeutics industry. Passionate about improving the quality of patients' lives, CSL Behring manufactures and markets a range of safe and effective plasma-derived and recombinant products and related services. The company's therapies are used in the treatment of immune deficiency disorders, hereditary angioedema, hemophilia, von Willebrand disease, other bleeding disorders and inherited emphysema. Other products are used for the prevention of hemolytic diseases in the newborn, in cardiac surgery, organ transplantation and in the treatment of burns. The company also operates one of the world's largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited, a biopharmaceutical company with headquarters in Melbourne, Australia. For more information, visit www.cslbehring.com
For further information please contact:
Sheila A. Burke
Director, Communications & Public Relations
Worldwide Commercial Operations