CSL Behring Release: Study Finds C1-Inhibitor Concentrate is Safe and Effective in Treating Skin Swelling in Patients With Hereditary Angioedema
HAE is a genetic disorder caused by an inherited deficiency of C1-INH. Symptoms include episodes of edema or swelling in the hands and feet, the face, the abdomen, and/or the larynx.
"Our findings show that replacement therapy with C1-INH concentrate can significantly reduce the duration and extent of symptoms in HAE patients who experience skin swelling during acute attacks of this serious disease," said Prof. Dr. Konrad Bork of the Department of Dermatology, Johannes Gutenberg University, Germany, and a lead investigator of the study. "This study adds to the growing body of research demonstrating the safety and efficacy of C1-INH concentrate in treating acute HAE attacks."
I.M.P.A.C.T. Trial Under Way in North America, Europe
No government-approved pharmaceutical product indicated specifically for treatment of acute HAE attacks, including abdominal attacks, is available in North America. CSL Behring is currently conducting International Multi-centre Prospective Angioedema C1-inhibitor Trials (I.M.P.A.C.T.), one of which is studying patients with acute abdominal or facial HAE attacks. The purpose of this Phase II/III study, which is being conducted at 45 sites in North America and Europe, is to demonstrate in a prospective and double-blind fashion that human pasteurized C1-INH concentrate leads to faster relief of acute symptoms of abdominal and facial attacks compared to placebo. The results of this global clinical trial will be submitted to the U.S. Food & Drug Administration and Health Canada in support of applications to license C1-INH concentrate for use in North America.
CSL Behring has manufactured and sold C1-INH concentrate for over 20 years in Germany, Austria, Switzerland, and several other countries in which it is licensed under the trade name Berinert® P for the treatment of acute HAE attacks.
Today's Presentation: Study Design
Findings were based on a retrospective clinical analysis of 1,828 swelling attacks in 43 HAE patients over a period of almost 30 years who were treated with 500 units (1,445 attacks) or 1,000 units (383 attacks) of C1-inhibitor concentrate. The time to relief, the duration of the swelling, and the severity of symptoms were documented during personal interviews and compared with 6,625 untreated attacks in the same patients. The skin swelling attacks affected the face (440 attacks), hands and arms (721 attacks), feet and legs (516 attacks), genitals (122 attacks), and the trunk (29 attacks).
Key Findings
The mean time to relief of symptoms was 1.3 hours +/- 1.4 hours in all treated attacks versus 60 hours +/- 26.4 hours in all untreated attacks. The mean duration of the attacks was shortened from 88 hours (SD 40.8 hours) (untreated attacks) to 40.8 hours (SD +/- 33.6 hours) (treated attacks). All patients responded to treatment. In 24 attacks of four patients the course of the treated attacks was not shortened but symptoms were milder compared to untreated attacks. C1-inhibitor concentrate was more effective when injected early in the attacks compared to late injections. There were no drug-related side effects.
About Hereditary Angioedema (HAE)
HAE due to C1-INH deficiency is characterized by relapsing, self-limiting episodes of edema at various body sites -- mostly subcutaneous tissue, the wall of the intestine, and the larynx. There are estimates of 6,000 to 10,000 or more people with HAE in the United States. Patients who have abdominal attacks experience episodes of severe pain, diarrhea, nausea, and vomiting caused by swelling of the intestinal wall. Attacks that involve the face and throat can result in airway closure, asphyxiation and, if left untreated, death. HAE is caused by a genetic deficiency of C1-INH, which is inherited in an autosomal dominant manner.
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.
Contact: Sheila A. Burke, Director, Communications & Public Relations Worldwide Commercial Operations CSL Behring 610-878-4209 Sheila.Burke@cslbehring.com
Source: CSL Behring