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 C1-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(R) 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 cslbehring.
CSL Behring
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