The US Food and Drug Administration (FDA) has approved Haegarda, which is the first C1 Esterase Inhibitor (human) for subcutaneous (under the skin) administration in the prevention of hereditary angioedema (HAE) attacks in adolescent and adult patients.
FDA approval
Haegarda is a human plasma-derived, purified, pasteurised, lyophilised concentrate prepared from large pools of human plasma from US donors. It has been indicated for routine prophylaxis to prevent HAE attacks but is not indicated for the treatment of acute HAE attacks.
“The approval of Haegarda provides a new treatment option for adolescents and adults with hereditary angioedema,” said Dr Peter Marks, PhD, 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.”
HAE is caused by having insufficient amounts of a plasma protein called C1-esterase inhibitor. When suffering an attack, people can develop rapid swelling of the hands, feet, limbs, face, intestinal tract or airway. The attacks can occur spontaneously or can be triggered by stress, surgery or infection.
Efficacy of Haegarda in the prevention of HAE attacks was demonstrated in a multicentre controlled clinical trial, comprising 90 subjects between the ages of 17–72 years with symptomatic HAE. In the trial patients were randomised to receive subcutaneous doses of either 40 IU/kg or 60 IU/kg twice a week and a comparison was made with a placebo treatment period. During the study period patients in both treatment groups experienced a significantly reduced number of HAE attacks compared to when they were receiving placebo treatment.
“With the unpredictability of HAE, HAEGARDA offers patients a 95% reduction in HAE attacks and more than a 99% reduction in the use of rescue medications, along with the ability to self-administer subcutaneously,” stated Timothy Craig, DO, Professor of Medicine and Pediatrics, Pennsylvania State University Medical School, Hershey, Pennsylvania. “This provides a new and exciting option for patients and physicians.”
Approval has been granted to CSL Behring, which develops, manufacturers and markets a range of products including coagulation and immunology therapies.