CSL Behring announced the U.S. Food and Drug Administration (FDA) has granted the company seven years of orphan-drug exclusivity for HAEGARDA (C1 Esterase Inhibitor Subcutaneous [Human]), the first and only subcutaneous treatment option for prevention of hereditary angioedema (HAE) attacks. HAEGARDA was approved by the FDA on June 22, 2017 for routine prophylaxis to prevent HAE attacks in adolescent and adult patients, and marketing exclusivity will continue through June 22, 2024.
"HAEGARDA represents an important advance in the care of HAE, having been shown to reduce the number of HAE attacks by a median of 95 percent relative to placebo with subcutaneous delivery," said Bill Campbell, Senior Vice President and General Manager, North America, CSL Behring. "CSL Behring is dedicated to delivering innovative products for rare diseases, including HAE, and we are pleased the FDA has recognized our commitment to helping positively impact the lives of patients with this debilitating and potentially life-threatening condition."
HAEGARDA is a plasma-derived concentrate of C1-INH that is self-administered twice weekly subcutaneously. Subcutaneous administration of C1-INH builds and maintains a steady-state level of functional C1-INH activity and offers patients ease of use by eliminating the need for venous access, including ports. In addition, HAEGARDA had a reduction in the use of rescue medication by a median of greater than 99 percent relative to placebo.
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 deficient or dysfunctional C1-INH, a protein in the blood that helps to control inflammation. Inadequate amounts of properly functioning C1-INH can lead to the accumulation of fluid in body tissues, causing considerable swelling 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 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 left untreated, death.
HAEGARDA is a self-administered, plasma-derived concentrate of C1-esterase inhibitor and the only subcutaneous therapy approved in the United States for routine prophylaxis to prevent HAE attacks in adolescent and adult patients.
HAEGARDA targets the root cause of HAE by replacing deficient or dysfunctional natural C1-INH, restoring functional C1-INH levels to above 40 percent of normal levels, which is associated with reduced risk for HAE attacks.1 HAEGARDA is dosed individually based on body weight so that each patient can achieve functional C1-INH levels.