FDA Approves Hereditary Angioedema Attack Treatment
The US Food and Drug Administration (FDA) approved the first subcutaneous C1 esterase inhibitor for the prevention Hereditary Angioedema (HAE) attacks in adolescent and adult patients.
The subcutaneous treatment, Haegarda, allows for easier at-home self-injection by the patient or caregiver. It is licensed by biopharmaceutical company CSL Behring, LLC.
HAE affects approximately 6,000 to 10,000 people in the country, and is characterized by rapid swelling of the hands, feet, limbs, face, intestinal tract, or airway. According to the FDA, these attacks of swelling can occur spontaneously, or can be triggered by stress, surgery or infection.
The condition itself is caused by having insufficient amounts of a plasma protein called C1-esterase inhibitor (or C1-INH). Peter Marks, MD, PhD, director of the FDA Center for Biologics Evaluation and Research, advocated for Haegarda's utility as an at-home treatment to prevent HAE's attacks.
Haegarda demonstrated efficacy in a multi-center, controlled clinical trial, involving 90 symptomatic HAE patients aged between 12-72 years old.
Subjects were randomized to receive twice per week subcutaneous doses of either 40 IU/kg or 60 IU/kg, according to the FDA, and the treatment effect was compared to a placebo treatment period. During the 16 week treatment period, patients in both treatment groups experienced a significantly reduced number of HAE attacks versus placebo treatment.
Patients experienced clinical side effects such as injection site reactions, allergic reactions, swelling of the nasal passages and throat, 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.
A press release regarding the approval was made available.
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