CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the company’s Biologics License Application (BLA) for its low-volume subcutaneous (SC) C1-Esterase Inhibitor (C1-INH) Human replacement therapy, CSL830, as prophylaxis to prevent Hereditary Angioedema (HAE) attacks. HAE is a rare genetic disorder caused by a deficiency of C1-INH, one of the proteins that work with the body’s immune system to control inflammation. Symptoms of HAE include episodes of swelling in the face, abdomen, larynx and extremities and can be fatal if untreated.
“The review of this application is another step towards providing advanced prophylactic treatment options to people living with HAE,” said Dr. Andrew Cuthbertson, Chief Scientific Officer and R&D Director, CSL Limited. “Since CSL Behring first reported the possibility of C1-INH replacement therapy for HAE over 40 years ago, we have remained committed to innovative research and providing advanced treatment options to people living with HAE. Subcutaneous prophylaxis is the next important step in helping HAE patients to prevent HAE attacks.”