FDA approves Berinert for treating HAE in adults and adolescents

Accovion supports CSL Behring in successfully obtaining marketing authorization for Berinert®

Berinert®, a human C1 esterase inhibitor (C1-INH) for treating acute episodes of facial or abdominal hereditary angioedema (HAE) in adults and adolescents has obtained approval from the US Food and Drug Administration (FDA). HAE is a rare and serious autosomal dominant genetic disease caused by C1-INH deficiency. Symptoms include sudden onset of edema (swelling) of the face, abdomen, extremities, or the larynx (which can be life threatening). Berinert® is a unique new treatment option for HAE, being the first C1-INH to be licensed in the USA for treatment of acute attacks of HAE.

Approval was based on the results of the I.M.P.A.C.T. (International Multi-center Prospective Angioedema C1-Inhibitor Trial) study, a double-blind, placebo-controlled, phase II/III study of 124 patients with HAE and acute episodes of moderate to severe facial or abdominal angioedema. This study successfully demonstrated the efficacy and safety of Berinert®. In addition, efficacy and safety data have been collected from over 400,000 treatments in Germany, Austria, Switzerland, and several other countries, where the drug is marketed with the trade name Berinert® P.

Accovion GmbH, a Contract Research Organization based in Eschborn, Germany, supported Marburg-based pharmaceutical company CSL Behring in the clinical development of Berinert®, from preparation and conduct of the I.M.P.A.C.T. study through to marketing authorization. During the design, conduct, and evaluation of the study, Accovion took key responsibility for biostatistics, statistical programming, data management, and the Data Safety Monitoring Board.

Rainer Maria Schulz, Global Head of Clinical R&D at CSL Behring Marburg said: “We are very grateful to the Accovion team for their dedication and a successful long-term partnership that has led to the approval of Berinert®. Together we have reached another important milestone.” Robert Lefebvre, Vice President and General Manager for US Commercial Operations at CSL Behring added: “US approval for Berinert® is an important step in our determination to fill the gaps in care for patients with rare and serious diseases, including HAE. We are delighted at the addition of a demonstrably effective product that improves quality of life for patients with HAE to our fast-growing product portfolio”.