The FDA approved CSL Behring’s subcutaneously administered C1 esterase inhibitor (human), Haegarda, for routine use in preventing hereditary angioedema (HAE) attacks in adolescent and adult patients. The approval marks the first for a subcutaneously C1 esterase inhibitor which gives patients the option to self-inject at home.
“The approval of Haegarda provides a new treatment option for adolescents and adults with hereditary angioedema,” said Peter Marks, M.D., Ph.D., director of the FDA's Center for Biologics Evaluation and Research. “The subcutaneous formulation allows patients to administer the product at home to help prevent attacks.”
“The FDA approval of Haegarda is an important milestone for the HAE community because it addresses the primary need of patients: to effectively prevent debilitating HAE attacks,” added Andrew Cuthbertson, M.D., Ph.D., CSO and R&D director, CSL.
FDA clearance of Haegarda was based on data from a clinical trial of 90 symptomatic HAE patients aged from 12 years to 72 years. The study data confirmed that twice-weekly subcutaneous doses of Haegard for 16-weeks led to a significantly reduced number of HAE attacks, when compared with placebo. CSL also has an FDA approved C1 esterase inhibitor (human) therapy, Berinert, for treating acute HAE attacks. In mid-2016, the FDA expanded the drug's approved use to patients under 12 years of age, making it first HAE treatment cleared for use in all age groups.
Last month, rival Shire reported positive topline data from the Phase III HELP™ study evaluating its subcutaneously administered plasma kallikrein-targeting monoclonal antibody lanadelumab, for preventing angioedema attacks in HAE patients aged 12 years and older. Shire’s marketed HAE drug portfolio includes the intravenously administered C1 esterase inhibitor (human) Cinryze® for routine HAE prophylaxis, and Firazyr® (icatibant injection), a subcutaneous injection for treating HAE attacks. In March, the EC approved a label extension for Cinryze that clears the drug for use in children aged from just 2 years.
In April, Shire slapped a lawsuit on CSL, claiming infringement of Shire’s newly granted U.S. patent covering the subcutaneous administration of C1 esterase inhibitor for treating HAE. CSL responded by saying it was “highly confident” that its own product “does not infringe any valid claim” of the Shire patent.