CSL Behring Receives Orphan-Drug Exclusivity for HAEGARDA® (C1 Esterase Inhibitor Subcutaneous [Human])

KING OF PRUSSIA, Pa., July 21, 2017 /PRNewswire/ -- Global biotherapeutics leader CSL Behring announced today that 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.

To support access to HAEGARDA, CSL Behring offers HAEGARDA Connect^SM, a comprehensive support program providing a range of resources and programs to assist with HAE and HAEGARDA education and information, financial assistance programs, nursing services, and reimbursement support.

For more information about HAEGARDA, including the U.S. Prescribing Information, visit www.HAEGARDA.com.

About Hereditary Angioedema (HAE)

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.

About HAEGARDA^® (C1 Esterase Inhibitor Subcutaneous [Human])

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.

Indications and IMPORTANT SAFETY INFORMATION About HAEGARDA

Indications

HAEGARDA^®, C1 Esterase Inhibitor Subcutaneous (Human), is a plasma-derived concentrate of C1 Esterase Inhibitor (C1-INH) indicated for routine prophylaxis to prevent Hereditary Angioedema (HAE) attacks in adolescent and adult patients. HAEGARDA is for subcutaneous use after reconstitution only.

IMPORTANT SAFETY INFORMATION

HAEGARDA^®, C1 Esterase Inhibitor Subcutaneous (Human), is a plasma-derived concentrate of C1 Esterase Inhibitor (C1-INH) indicated for routine prophylaxis to prevent Hereditary Angioedema (HAE) attacks in adolescent and adult patients. HAEGARDA is for subcutaneous use after reconstitution only.

HAEGARDA is contraindicated in patients with a history of life-threatening hypersensitivity reactions, including anaphylaxis, to C1-INH preparations or their excipients.

Severe hypersensitivity reactions to HAEGARDA could occur. In such cases, discontinue administration and institute appropriate treatment. Epinephrine should be immediately available to treat hypersensitivity reactions.

At the recommended subcutaneous dose of HAEGARDA, no causal relationship to thromboembolic events (TEs) has been established. However, TEs have been reported with intravenous administration of C1-INH products, usually at high doses.

In clinical trials, adverse reactions observed in more than 4% of subjects treated with HAEGARDA were injection-site reactions, hypersensitivity, nasopharyngitis, and dizziness.

HAEGARDA is derived from human plasma. The risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent and its variant (vCJD), cannot be completely eliminated.

Full HAEGARDA Prescribing Information can be found at www.HAEGARDA.com.

About CSL Behring

CSL Behring is a global biotherapeutics leader driven by its promise to save lives. Focused on serving patients' needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, inherited respiratory disease, and neurological disorders. The company's products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn. 

CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. The parent company, CSL Limited (ASX: CSL), headquartered in Melbourne, Australia, employs nearly 20,000 people, delivering its life-saving therapies to people in more than 60 countries. For more information visit www.cslbehring.com and follow us on www.Twitter.com/CSLBehring.

SOURCE CSL Behring