CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Berinert® C1-Esterase Inhibitor, Human for the treatment of acute abdominal or facial attacks of hereditary angioedema (HAE), a rare and serious genetic disorder, in adult and adolescent patients. Berinert is the first and only therapy approved for this indication in the U.S. The approval is 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.), which studied the efficacy and safety of C1-inhibitor (C1-INH) concentrate. The safety and efficacy of Berinert for prophylactic therapy have not been established.
"The FDA approval of Berinert marks an important milestone in CSL Behring’s ongoing commitment to satisfying the unmet needs of patients with rare and serious disorders, such as hereditary angioedema," said Robert Lefebvre, Vice President and General Manager of U.S. Commercial Operations at CSL Behring. "As a leader in developing safe, effective and high-quality therapies, we are pleased to add to our rapidly growing portfolio a proven treatment that can make a positive difference in the lives of HAE patients and their families."
HAE is a genetic disorder caused by a deficiency of C1-INH and is inherited in an autosomal dominant manner. Symptoms of HAE include episodes of edema or swelling in the face and the abdomen. 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 can cause painful distortion and painful swelling. 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 U.S.
“For individuals with HAE, episodes of swelling can be extremely painful and frightening, " said Timothy Craig, MD, professor of medicine and pediatrics, The Pennsylvania State University Hershey Medical Center. "With the approval of Berinert, healthcare professionals can now provide HAE patients in the U.S with a safe and effective treatment option that rapidly relieves the symptoms of acute attacks in the face and abdomen."
“Today’s approval provides adult and adolescent HAE patients and their physicians with a proven, safe, and effective therapy for treating debilitating, painful, and life-threatening facial and abdominal HAE attacks once they have begun,” said Anthony J. Castaldo, President of the United States Hereditary Angioedema Association, a nonprofit patient advocacy organization that represents approximately 6,500 HAE patients in the United States.
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 doses and compared with 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. study found that C1-inhibitor concentrate (C1-INH) is effective and safe in rapidly treating acute abdominal and facial skin swellings in adults and adolescents with HAE. The study found that the median time to symptom relief was 30 minutes after receiving C1-INH compared with 1.5 hours with a placebo.
Berinert, a plasma-derived intravenous therapy, treats the fundamental cause of acute facial and abdominal hereditary angioedema (HAE) symptoms by providing C1-INH deficient adult and adolescent 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 abdomen. Berinert is a unique HAE therapy because of its reliable record of proven efficacy and safety in international clinical use in over 400,000 treatments in Germany, Austria, Switzerland, and several other countries where it is manufactured and sold by CSL Behring under the trade name Berinert® P.
Important Safety Information
Berinert is a plasma derived concentrate of C1 Esterase Inhibitor (Human), indicated for the treatment of acute abdominal or facial attacks of hereditary angioedema (HAE) in adult and adolescent patients.
The safety and efficacy of Berinert for prophylactic therapy have not been established. Berinert is contraindicated in individuals who have had an anaphylactic or severe systemic reaction to C1-INH preparations. Monitor patients for early signs of allergic or hypersensitivity reactions (including hives, generalized urticaria, chest tightness, wheezing, hypotension, and anaphylaxis). If hypersensitivity is suspected, immediately discontinue administration and initiate appropriate treatment. Epinephrine should be immediately available for treatment of acute severe hypersensitivity reactions.
Thrombotic events have occurred in patients receiving off-label high doses of Berinert. Monitor patients with known risk factors for thrombotic events.
Berinert is derived from human plasma. The risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent, cannot be completely eliminated.
The most serious adverse reaction reported in subjects in clinical studies who received Berinert is an increase in the severity of pain associated with HAE. The most common adverse reactions observed in more than 4 percent of subjects after Berinert treatment were headache, abdominal pain, nausea, muscle spasms, pain, diarrhea, and vomiting.
Berinert has not been evaluated in pregnant women or nursing mothers; benefits of treatment should be weighed against potential risks in pregnant women, and Berinert should be given to nursing mothers only if clearly needed. Safety and efficacy of Berinert have not been established in children (ages 0 through 12) or in the geriatric population. For more information, including full prescribing information, visit www.berinert.com
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, 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, Director
Communications & Public Relations
908-234-9900, ext 312