Global biotherapeutics leader CSL Behring announced that the U.S. Food and Drug Administration (FDA) has approved HAEGARDA® (C1 Esterase Inhibitor Subcutaneous [Human]), the first and only subcutaneous therapy indicated for routine prophylaxis to prevent hereditary angioedema (HAE) attacks in adolescent and adult patients. HAE is a rare, genetic, and potentially life-threatening condition that causes painful, debilitating, and unpredictable episodes of swelling on the body, including the abdomen, face, larynx, and extremities.
The safety and efficacy of HAEGARDA were established in the Phase III COMPACT (C Studies for Optimal Management in Preventing Angioedema with low-volume subcutaneous C1-inhibitor replacement Therapy) trial, which showed that at the approved dose of 60 IU/kg, HAEGARDA reduced the median number of HAE attacks by 95 percent relative to placebo. Use of rescue medication was reduced by greater than 99 percent versus placebo. HAEGARDA is a self-administered, plasma-derived concentrate of C1 esterase inhibitor (C1-INH) injected twice weekly subcutaneously (just under the skin).
“The FDA approval of HAEGARDA is a transformational milestone for the HAE community because it addresses the primary need of patients: to effectively prevent debilitating HAE attacks,” said Dr. Andrew Cuthbertson, Chief Scientific Officer and R&D Director, CSL Limited. “CSL Behring has a long heritage of delivering on its promise to the HAE community. Thanks to our clinical trial participants, we’re proud to lead the community into the next era of treatment by offering the first and only subcutaneous preventive treatment option.”
HAE is caused by deficient or dysfunctional C1-INH, a protein in the blood that helps to control inflammation. Depending on the severity of the disease, patients could experience several debilitating attacks each month. Laryngeal attacks, which are experienced by more than half of all HAE patients during their lifetime, block the airway and can be fatal – with mortality rates as high as 33 percent in untreated patients.
HAEGARDA targets the root cause of HAE by replacing deficient or dysfunctional C1-INH protein, restoring C1-INH levels above 40 percent of normal levels, which is associated with reduced risk of HAE attacks.1 Subcutaneous administration of C1-INH builds and maintains steady-state functional C1-INH levels within three to four doses of HAEGARDA.
“With the unpredictability of HAE, HAEGARDA offers patients a 95 percent reduction in HAE attacks and more than a 99 percent reduction in the use of rescue medications, along with the ability to self-administer subcutaneously,” said Timothy Craig, D.O., Professor of Medicine and Pediatrics, Pennsylvania State University Medical School, Hershey, Pennsylvania. “This provides a new and exciting option for patients and physicians.”