CSL Behring announced today it has been granted national marketing authorization in Italy and Luxembourg to market Berinert® for the treatment of acute hereditary angioedema (HAE) attacks in any body location. This latest authorization brings to 28 the number of countries (in Europe, Asia, North America, South America and Australia) in which Berinert is now licensed.
In October 2009, the United States Food and Drug Administration approved Berinert for the treatment of acute abdominal and facial attacks of HAE, a rare and serious genetic disorder, in adolescent and adult patients. CSL Behring completed the European Mutual Recognition Procedure (MRP) for Berinert in 23 countries in December, 2008. Marketing authorization was also granted for Australia in January, 2010, and applications for licensing in Canada and Israel have also been filed with authorities. Berinert has been marketed in Germany for more than 30 years.
The approvals are mainly based on the results of the phase II/III prospective, double-blind placebo-controlled International Multi-center Prospective Angioedema C1-Inhibitor Trial (I.M.P.A.C.T.), the largest single placebo-controlled HAE trial ever, that studied the efficacy of pasteurized C1-esterase inhibitor (C1-INH) concentrate.
“It is very good news that this well-established therapy, which has helped so many patients to live without the fear of life-threatening and debilitating HAE attacks, is now available to even more HAE patients in Europe,” said Dr. Marco Cicardi, professor of Internal Medicine at the University of Milan, Italy. “CSL Behring is to be commended for its dedication to this relatively small patient community for whom treatment options have been limited for so long.”
HAE is a genetic disorder caused by a deficiency of C1-INH. It is inherited in an autosomal dominant manner. Symptoms of HAE include episodes of edema, or swelling, in the hands, feet, the face, the abdomen, and/or the larynx. Patients who have abdominal attacks of HAE can experience episodes of severe pain, diarrhea, nausea, and vomiting caused by swelling of the intestinal wall. HAE attacks that involve the face and larynx can result in airway closure, asphyxiation, and, if untreated, death. Diagnosis of HAE requires a blood test to confirm low or abnormal levels of C1-INH.
The effective date of the marketing authorization in Italy and Luxembourg was 29 March, 2010.
About I.M.P.A.C.T. Studies
I.M.P.A.C.T. was a study of 124 HAE patients with acute, moderate, or severe abdominal or facial attacks. C1-INH concentrate was administered at two different dose levels and compared to placebo. The main study endpoints were: time to onset of symptom relief from HAE attacks, proportion of subjects with worsening clinical HAE symptoms, and safety.
The I.M.P.A.C.T.1 study found that C1-esterase inhibitor concentrate (C1-INH) is highly effective and safe in rapidly treating acute abdominal and facial skin swellings in patients with HAE. The study found that the median time to symptom relief was 30 minutes after receiving C1-INH compared to 1.5 hours with a placebo.
The I.M.P.A.C.T. 2 study results showed the efficacy and safety of multiple open-label treatments with C1-INH concentrate for HAE attacks at any body site. The median time to onset of symptom relief was most rapid for laryngeal attacks (15 minutes), followed by abdominal attacks (20 minutes), facial attacks (28 minutes), and peripheral attacks (31 minutes). The median times to complete resolution of symptoms were less than 8 hours for laryngeal attacks, 10 hours for abdominal and 24 hours for peripheral attacks and 31 hours for facial attacks. Findings of I.M.P.A.C.T. 2 were based on treatment with 20 U/kg bodyweight of C1-INH concentrate in 975 episodes of HAE attacks involving any location on the body, in 57 patients.
Berinert®, a plasma-derived intravenous therapy, treats the fundamental cause of hereditary angioedema (HAE) symptoms by providing C1-INH deficient patients with the missing human protein. Without C1-INH, patients with HAE suffer from recurrent episodes of rapid swelling of areas of the skin and underlying tissues including the face, mouth and throat. In rare circumstances, these episodes can be life-threatening. Berinert is a well-established HAE therapy because of its reliable record of proven efficacy and safety in over 30 years of international clinical use in more than 500,000 treatments in Germany, Austria, Switzerland, and several other countries.
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, and hereditary angioedema. 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.
Sheila A. Burke