New Hope in Immunodeficiency Treatment for Improved Tolerability and Convenience in the use of Immune Globulin Intravenous (Human) 10% Liquid (Privigen®)
Bern, Switzerland — 09 November 2007
CSL Behring today announced data on the use of L-proline as a new single stabilizer for Immune Globulin Intravenous (Human) 10% Liquid (Privigenâ). Researchers found that the use of L-proline, a naturally occurring amino-acid, inhibited dimer formation more efficiently when compared with other additives like glycine, used as a stabilizer in conventional immunoglobulin products. In addition, the formulation with L-proline was found to prevent aggregation. The data were presented today at the American Congress of Allergy, Asthma and Immunology (ACAAI) in Dallas, Texas.
“Liquid intravenous immunoglobulin (IVIg) products have limited stability mainly due to the potential for the immunoglobulin to form excessive dimers, and to become degraded and aggregated, causing tolerability problems for patients,” commented Reinhard Bolli, PhD, lead researcher for the formulation development, from CSL Behring. “A product that ensures a lower formation of dimers and concomitantly assures a high stability would offer an advantage to patients and convenience and flexibility to clinicians.”
In a separate study, researchers found that Privigen, which uses only L-proline as a stabilizer, can be stored for at least three years at room temperature without loss of functionality and impairment of safety and tolerability.
“Room temperature stability eliminates the need for special refrigerated storage facilities and, importantly, also allows immediate infusion without the need to warm up the solution to room temperature,” commented Andrea Sebald, PhD, lead researcher of the Privigen stability study, CSL Behring. She continued: “The formulation with L-proline has resulted in a ready-to-go medication for immediate use – where patients do not have to wait, where healthcare professionals are freed up to focus on the medically important issues and where the warmed-up product is not uselessly discarded in the event the patient is unable to attend their treatment. Privigen therefore can save time, manpower and waste, making it simpler and more convenient than other products.”
Privigen is used for treating patients diagnosed with primary immunodeficiency (PI) which occurs when part of a person’s immune system is missing or does not work correctly. People with primary immunodeficiency have weakened immune systems and can not fight infections as well as they might otherwise. Privigen is also used to treat chronic immune thrombocytopenic purpura (ITP) to rapidly raise platelet counts to prevent bleeding.
For many people with antibody deficiencies, antibody replacement therapy is the only treatment option. The patient receives regular infusions or injections of immunoglobulins, which have been isolated and purified from blood donations of healthy donors.
In July 2007, the U.S. Food and Drug Administration (FDA) granted marketing approval to CSL Behring for Privigen, an intravenous immunoglobulin (IVIg), for treating patients diagnosed with primary immunodeficiency (PI) and immune thrombocytopenic purpura (ITP). CSL Behring plans to launch Privigen in the first quarter of 2008. The application is currently under review by European and Swiss Regulatory Authorities.
Important Safety Information
In clinical studies, Privigen has been shown to be safe. As with any medication, side effects may accompany treatment. The frequency of side effects was based on a review of 1,038 injections given during the clinical trial in the United States and Europe. Because Privigen is made from plasma, as are all commercial human polyvalent immunoglobulins, the risk of transmitting infectious agents, including viruses, and theoretically, the Creutzfeldt-Jakob disease (CJD) agent, cannot be completely eliminated.
Privigen is contraindicated in patients with known anaphylactic or severe hypersensitivity responses to Immune Globulin (Human). Patients with severe selective IgA deficiency (IgA < 0.05 g/L) may develop anti-IgA antibodies that can result in a severe anaphylactic reaction. Such patients should only receive intravenous immune globulin with utmost caution and in a setting where supportive care is available for treating life-threatening reactions. As a class, Immune Globulin Intravenous Human (IVIg) products have been associated with renal dysfunction, acute renal failure, osmotic nephrosis, and death. While reports of renal dysfunction and acute renal failure have been associated with the use of many of the licensed IVIg products, those containing sucrose as a stabilizer accounted for a disproportionate share of the total number. Privigen does not contain sucrose.
About Primary Immune Deficiencies
These are a group of predominantly genetic disorders that cause a malfunction in part or all of the immune system, keeping the patient from fighting off infections caused by everyday germs. 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. In some severe cases of PI, infections may result in a patient being hospitalized repeatedly. Some infections, such as meningitis, may even result in death. Nearly 100 types of PIs exist. Most are inherited, but in some cases the cause is unknown.
No single treatment works for all of the different types of PI. Infusions of replacement antibodies (immunoglobulins or Ig) can help supplement the immune system to prevent infection in nearly three-quarters of those people living with PI whose disease is tied to an antibody deficiency.
Immune Thrombocytopenic Purpura, or ITP, is an autoimmune disease in which the immune system attacks and destroys the body's own platelets, the cells that prevent bleeding in blood vessels and facilitate clotting. There are two forms of ITP: acute ITP, which resolves within six months, and chronic ITP, which most often occurs in adults and by definition lasts six months or longer. The annual incidence of ITP is 100 to 115 in every one million people. In the U.S., approximately 200,000 people have the disorder.
ITP is characterized by a low number of platelets (<30 x 109/L), usually caused by the body’s production of substances (antibodies) that coat the platelets and signal their elimination from the blood. Diagnosis of ITP is often made by excluding other possible causes of the low platelet count and bleeding. People with the disorder often have purple bruises on the skin called purpura, a sign that bleeding has occurred in small blood vessels under the skin. They can also have petechiae, small red splotches on the skin that resemble a rash.
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, hemophilia, von Willebrand disease, other bleeding disorders and inherited emphysema. Other products are used for the prevention of hemolytic disease in newborns, in cardiac surgery, organ transplantation and in the treatment of burns. The company also operates one of the world's largest plasma collection networks, ZLB Plasma. CSL Behring is a subsidiary of CSL Limited, a biopharmaceutical company with headquarters in Melbourne, Australia. For more information, visit www.CSLBehring.com.
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