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news.CSL Behring has submitted a biologics license application to the FDA requesting approval to market its C1-esterase inhibitor concentrate in the US for the treatment of hereditary angioedema or HAE, a rare and serious genetic disorder.
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The submission is based on the recently completed Phase II/III prospective, double-blind placebo-controlled International Multi- center Prospective Angioedema C1-Inhibitor Trial (IMPACT), the largest HAE trial ever, that studied the efficacy of pasteurized C1-INH concentrate. 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 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.
Val Romberg, senior vice president of R&D at CSL Behring, said: “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 US.”