CSL Behring Submits BLA Requesting Approval of C1-Esterase Inhibitor for the Treatment of Hereditary Angioedema
Pivotal phase II/III study demonstrates C1-INH is effective treatment for acute HAE attacks
King Prussia, PA — 06 March 2008
CSL Behring has submitted a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) requesting approval to market its C1-esterase inhibitor concentrate in the United States for the treatment of hereditary angioedema (HAE), a rare and serious genetic disorder. The submission is based on the recently completed phase II/III prospective, double-blind placebo-controlled International Multi-center Prospective Angioedema C1-Inhibitor Trial (I.M.P.A.C.T.), the largest HAE trial ever, that studied the efficacy of pasteurized C1-INH concentrate.
The proposed indication is for the treatment of acute attacks in patients with HAE. Currently, there are no specifically-approved therapies for HAE in North America.
"Submission of the BLA for C1-INH brings CSL Behring one step closer to providing therapy for hereditary angioedema," said Val Romberg, Senior Vice President of Research and Development at CSL Behring. "We are confident that our expertise in developing and manufacturing plasma-derived protein therapies will be advantageous in our pursuit of regulatory approval in the United States."
HAE is a genetic disorder caused by a deficiency of C-INH which is inherited in an autosomal dominant manner. Symptoms include episodes of edema or swelling in the hands and feet, the face, the abdomen, and/or the larynx. Patients who have abdominal attacks can experience episodes of severe pain, diarrhea, nausea, and vomiting caused by swelling of the intestinal wall. 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. There are estimates of 6,000 to 10,000 or more people with HAE in the United States.
The submission is based on a study of 124 HAE patients with acute, moderate, or severe abdominal or facial attacks. C1-INH concentrate was administered at two different doses 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.
CSL Behring manufactures and sells C1-INH concentrate in Germany, Austria, Switzerland, and several other countries under the trade name Berinert® P.
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 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, 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.
Sheila A. Burke, Director, Communications & Public Relations
Worldwide Commercial Operations