FDA Approves HAEGARDA® (C1 Esterase Inhibitor Subcutaneous [Human]), First and Only Subcutaneous Preventive Treatment for Hereditary Angioedema

- Use of rescue medication was reduced by greater than 99 percent1

- HAEGARDA has been shown to reduce HAE attacks by 95 percent (median) in patients with a life-threatening, rare genetic disorder

- Subcutaneous administration builds and maintains steady-state C1-INH levels

News provided by

CSL Behring

Jun 23, 2017, 14:16 ET

KING OF PRUSSIA, Pa., June 23, 2017 /PRNewswire/ -- Global biotherapeutics leader CSL Behring today announced that the U.S. Food and Drug Administration (FDA) has approved HAEGARDA^® (C1 Esterase Inhibitor Subcutaneous [Human]), the first and only subcutaneous therapy indicated for routine prophylaxis to prevent hereditary angioedema (HAE) attacks in adolescent and adult patients. HAE is a rare, genetic, and potentially life-threatening condition that causes painful, debilitating, and unpredictable episodes of swelling of the abdomen, larynx, face, and extremities, among other areas of the body.

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The safety and efficacy of HAEGARDA were established in the Phase III COMPACT (Clinical Studies for Optimal Management in Preventing Angioedema with low-volume subcutaneous C1-inhibitor replacement Therapy) trial, which showed that at the approved dose of 60 IU/kg, HAEGARDA reduced the median number of HAE attacks by 95 percent relative to placebo. Use of rescue medication was reduced by greater than 99 percent versus placebo. HAEGARDA is a self-administered, plasma-derived concentrate of C1 esterase inhibitor (C1-INH) injected twice weekly subcutaneously (just under the skin).

"The FDA approval of HAEGARDA is a transformational milestone for the HAE community because it addresses the primary need of patients: to effectively prevent debilitating HAE attacks," said Dr. Andrew Cuthbertson, Chief Scientific Officer and R&D Director, CSL Limited. "CSL Behring has a long heritage of delivering on its promise to the HAE community. Thanks to our clinical trial participants, we're proud to lead the community into the next era of treatment by offering the first and only subcutaneous preventive treatment option."

HAE is caused by deficient or dysfunctional C1-INH, a protein in the blood that helps to control inflammation. Depending on the severity of the disease, patients could experience several debilitating attacks each month. Laryngeal attacks, which are experienced by more than half of all HAE patients during their lifetime, block the airway and can be fatal – with mortality rates as high as 33 percent in untreated patients.^2,3

"HAE patients face painful, debilitating, and life-disrupting swelling episodes that are also life-threatening when the airway is involved," said Anthony J. Castaldo, President of the U.S. Hereditary Angioedema Association. "Patient care in the United States takes an important step forward with the approval of this subcutaneous treatment for preventing HAE attacks."

HAEGARDA targets the root cause of HAE by replacing deficient or dysfunctional C1-INH protein, restoring C1-INH levels above 40 percent of normal levels, which is associated with reduced risk of HAE attacks.¹ Subcutaneous administration of C1-INH builds and maintains steady-state functional C1-INH levels within three to four doses of HAEGARDA.

"With the unpredictability of HAE, HAEGARDA offers patients a 95 percent reduction in HAE attacks and more than a 99 percent reduction in the use of rescue medications, along with the ability to self-administer subcutaneously," said Timothy Craig, D.O., Professor of Medicine and Pediatrics, Pennsylvania State University Medical School, Hershey, Pennsylvania. "This provides a new and exciting option for patients and physicians."

HAEGARDA will be available to U.S. patients with HAE in the near future. CSL Behring is committed to helping patients get access to HAEGARDA regardless of their financial situation. 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 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.

¹ Longhurst H, et al. Prevention of hereditary angioedema attacks with a subcutaneous C1 inhibitor. N Eng J Med. 2017;376:1131-1140.
² Bork K, et al. Clinical studies of sudden upper airway obstruction in patients with hereditary angioedema due to C1 esterase inhibitor deficiency. Ach Intern Med. 2003;163:1229-35.
³ Bork K, et al. Efficacy of different medical therapies for the treatment of acute laryngeal attacks of hereditary angioedema due to C1-esterase inhibitor deficiency. J Emerg Med. 2016;50(4):567-80.