KING OF PRUSSIA, Pa. – March 15, 2018 – Imagine not knowing when you might experience unpredictable, severe and painful swelling without warning in different parts of your body, including the abdomen, face and larynx. This is what it’s like for patients with hereditary angioedema (HAE) who often go undiagnosed well into their adult years, ”imprisoned” by their rare disorder.
Life Sciences Pennsylvania presented its 2018 Patient Impact Award to CSL Behring for developing HAEGARDA®, C1 Esterase Inhibitor Subcutaneous (Human), which has been shown to reduce HAE attacks by 95% versus placebo (median), and is administered subcutaneously rather than intravenously.
Most patients can learn to administer their own injections subcutaneously at a time and place that’s convenient for them. The therapy can improve the lives of patients who often describe the impact of HAE on their lives as being like a “roller coaster” because of its unpredictability.
In accepting the award, CSL Behring’s Executive Vice President and Chief Commercial Officer, Bill Campbell said the company and its employees are proud to receive the 2018 Patient Impact Award as another affirmation that the 20,000 employees of CSL, “always put patients first.”
“HAEGARDA,” Campbell continued, “is the most recent example of CSL Behring’s commitment to delivering on its promise to bring innovative new medicines to patients with rare and serious diseases. We begin by listening to patients and healthcare providers to better understand the types of medicines they need and want.”
HAE is caused by deficient or dysfunctional C1 Esterase Inhibitor (C1-INH), a protein in the blood that helps to control inflammation in the body. Swelling of the larynx is particularly dangerous for people with HAE as it can cause asphyxiation if not immediately treated. Abdominal swelling can cause excruciating, debilitating pain, and facial swelling can be so pronounced that it causes severe changes in a person’s appearance--to the extent he or she may be unrecognizable even to themselves.
HAEGARDA was granted Orphan Drug Exclusivity for seven years by the U.S. Food & Drug Administration.
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 C1-INH, restoring functional C1-INH levels to above 40 percent, which is associated with reduced risk for HAE attacks. HAEGARDA is dosed based on body weight so that each patient receives an individualized amount of HAEGARDA.
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, which is 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 more than 16,000 people with operations in more than 30 countries. For more information visit www.cslbehring.com and follow us on www.Twitter.com/CSLBehring.
 Longhurst H., et al. Prevention of hereditary angioedema attacks with a subcutaneous C1 inhibitor. N Eng J Med. 2017376: 1131-1140.