FDA Accepts CSL Behring’s Biologics License Application for First Subcutaneous Prophylactic Therapy to Prevent Hereditary Angioedema Attacks
KING OF PRUSSIA, Pa. — 30 August 2016
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.”
About Hereditary Angioedema
HAE is a rare and potentially life-threatening genetic condition that occurs in about 1 in 10,000 to 1 in 50,000 people. HAE is caused by lack of or malfunctioning C1-INH. As a result, there are inadequate amounts of properly functioning C1-INH, leading fluid to build up in body tissues, causing considerable swelling episodes referred to as angioedema. HAE attacks can affect many parts of the body and can spread to multiple sites, including the face, abdomen, larynx and extremities. Patients who have abdominal attacks of HAE can experience episodes of extreme pain, diarrhea, nausea and vomiting caused by swelling of the intestinal wall. HAE attacks that involve the face or throat can result in airway closure, asphyxiation and, if untreated, death.
About Subcutaneous C1 Esterase Inhibitor (C1-INH)
C1 Esterase Inhibitor (Human) is an investigational, low-volume, subcutaneous prophylactic therapy. C1-INH replaces the missing or malfunctioning C1-INH protein in patients with a C1-INH deficiency, thereby preventing HAE attacks.