The US Food and Drug Administration (FDA) has approved the orphan drug Haegarda (CSL Behring LLC), the first C1 esterase inhibitor (human) for subcutaneous administration for the prevention of hereditary angioedema (HAE) attacks in patients aged 12 to 72 years.
“The subcutaneous route of administration allows for easier at-home self-injection by the patient or caregiver, once proper training is received,” the FDA said in a news release.
HAE is a rare, potentially life-threatening genetic disease caused by insufficient amounts of the plasma protein C1 esterase inhibitor (C1-INH). It is characterized by attacks of swelling of the hands, feet, limbs, face, intestinal tract, or airway. Attacks can occur spontaneously or can be triggered by stress, surgery, or infection.
HAE affects an estimated 6000 to 10,000 people in the United States.
Haegarda is a human plasma-derived, purified, pasteurized, lyophilized (freeze-dried) concentrate. It is indicated for routine prophylaxis to prevent HAE attacks. Haegarda is not indicated for the treatment of acute HAE attacks.
The efficacy of Haegarda was demonstrated in a multicenter controlled clinical trial in 90 patients aged 12 to 72 years with symptomatic HAE. Patients received subcutaneous doses of Haegarda (40 IU/kg or 60 IU/kg); the treatment effect of Haegarda was compared to effects experienced during a period of treatment with placebo.
During the 16-week treatment period, the number of HAE attacks was significantly reduced in both treatment groups, relative to the placebo treatment period.
The most common side effects were injection site reactions, hypersensitivity reactions, nasopharyngitis, and dizziness.
Haegarda should not be used in individuals who have experienced life-threatening hypersensitivity reactions, including anaphylaxis, to a C1-INH preparation or its inactive ingredients.
“The approval of Haegarda provides a new treatment option for adolescents and adults with hereditary angioedema. The subcutaneous formulation allows patients to administer the product at home to help prevent attacks,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in the news release.
The company expects Haegarda to be available in the United States in the third quarter of this year. “The price of Haegarda has not been released at this time,” Jennifer Purdue, the company’s external communications manager, told Medscape Medical News.
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