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, 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.