CSL Behring announced today that the European Commission has granted marketing authorization for Hizentra® (Human Normal Immunoglobulin), 20 percent solution for subcutaneous injection, for treating patients diagnosed with primary immunodeficiency (PI) as well as secondary immunodeficiencies. This authorization is valid for all 29 European/European Economic Area member states.
CSL Behring is a subsidiary of CSL Limited (ASX:CSL). Hizentra is currently marketed in the United States. It received U.S. Food and Drug Administration approval in March 2010.
"Hizentra is an exciting new offering in Europe that represents an effective, convenient choice of at-home subcutaneous Ig therapy for people with primary and secondary immunodeficiencies," said Paul Perreault, Executive Vice President, Worldwide Commercial Operations, CSL Behring. “Because it is ready-to-use, Hizentra enables patients to infuse the product where and when it suits them, and physicians now have another product to select to best meet the individual needs of their patients. As a leader in developing safe, high-quality, effective Ig therapies for use around the world, CSL Behring is proud to add this important addition to our expanding product portfolio."
This high-concentration product is stabilized with L-proline, a naturally occurring amino acid. L-proline allows Hizentra to be stored at room temperature (up to 25°C [77°F]). The European Commission has cleared Hizentra with an initial storage shelf life of two years. Because no refrigeration is necessary, Hizentra is ready to use, offering patients and physicians convenience and portability.
As the first 20 percent subcutaneous immunoglobulin (Ig), Hizentra provides lower infusion volumes, combined with consistent and steady levels of immunoglobulin resulting in effective protection against infection.
Hizentra is part of the CSL Behring immunoglobulin (Ig) franchise. This comprehensive Ig product portfolio also includes the first U.S. FDA-approved subcutaneous immunoglobulin and the first proline-stabilized intravenous immunoglobulin. CSL Behring manufactures Hizentra at its state-of-the-art facility in Bern, Switzerland, where advanced technologies are applied to further ensure product safety and ample supply. This facility represents the long-term commitment of CSL Behring to global Ig markets.
Study Design
The European Commission approval of Hizentra was based on results from a prospective, open-label, multicenter, single-arm, clinical study conducted in Europe, evaluating the efficacy, tolerability, and safety of Hizentra in adult and pediatric subjects with PI. In the study, 51 PI patients (3 to 60 years old), previously treated with IVIg or SCIg therapies, received weekly subcutaneous infusions of Hizentra at doses equivalent to those used in previous treatment. The primary endpoint to sustain IgG trough levels comparable to the previous treatment was achieved (mean trough level of 8.10 g/L for Hizentra). While the mean IgG trough level increased by 17.7 percent in patients who had been with IVIG (6.78 g/L pre-study level ), it remained similar in patients who had been previously treated with subcutaneous Ig (pre-study level of 8.43 g/L). No serious bacterial infections were reported during the efficacy period in patients receiving Hizentra during the clinical study. Almost all adverse events (98.7 percent) were mild to moderate in intensity.
About Primary Immunodeficiencies
Primary immunodeficiency (PI) is a group of more than 150 diseases that affect the cells, tissues and proteins of the immune system. In people with PI, the immune system is either absent or functioning inadequately, leaving them more susceptible to infection. For individuals with PI – 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 or be hospitalized for treatment. Repeated infections can lead to organ damage, which, over time, can become life-threatening.
Collectively, PIs affect an estimated 10 million people worldwide, and the incidence is estimated to be 1 in 10,000. For more information on PI, please visit www.cslbehring.com. For patients with primary immunodeficiencies, 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 PI, but infusions of replacement antibodies (immunoglobulins) can help supplement the immune system to prevent infection in nearly three-quarters of PI cases that are due to antibody deficiencies.
Immunoglobulin is a blood component that has become standard immune replacement therapy for most people living with PI, and nearly 70 percent of PI patients receive Ig replacement therapy. Since the 1980s, the first-line therapy for most PI patients has been intravenous immunoglobulin (IVIg), in 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 veins. Hizentra allows patients to use a small, portable pump to self-administer their infusions by injection under the skin (subcutaneous administration).
For more information, including full Summary of Product Characteristics, visit http://ec.europa.eu/health/documents/community-register/index_en.htm
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 indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema 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.
###
Contacts:
Sheila Burke
CSL Behring
610-878-4209
Sheila.Burke@cslbehring.com
Etanjalie Ayala
Weber Shandwick
212-445-8225
eayala@webershandwick.com
2.National Institute of Child Health & Human Development. Primary Immunodeficiency: What is primary immunodeficiency? http://www.nichd.nih.gov/health/topics/Primary_Immunodeficiency.cfm. Accessed February 2011.
3.Jeffrey Modell Foundation. Primary Immunodeficiency Resource Center. About PI. http://www.info4pi.org/aboutPI/index.cfm?section=aboutPI&CFID=36419223&CFTOKEN=3244. Accessed February 2011.