FDA Approves First Subcutaneous Therapy to Treat HAE

The U.S. Food and Drug Administration (FDA) has approved CSL Behring’s 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. Approval is based on a Phase III COMPACT (Clinical 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, and 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 injected twice subcutaneously. “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 research and development director at CSL Limited. “CSL Behring has a long heritage of delivering on its promises 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.”