The U.S. Food and Drug Administration (FDA) has approved Andembry (garadacimab) for the treatment of hereditary angioedema (HAE) in patients ages 12 years and older.
Hereditary Angioedema
HAE is a rare condition characterized by recurrent episodes of severe swelling. These attacks often affect the skin, gastrointestinal tract, and upper airway. While skin swelling can cause pain, dysfunction, and disfigurement, it is not considered dangerous. When the gastrointestinal tract is involved, this may cause symptoms such as nausea, vomiting, diarrhea, and abdominal pain. The upper airway is less commonly affected, but can cause upper airway obstruction, which can be dangerous. Attacks may involve one area or a combination of areas of the body and typically go away on their own within 2 to 5 days. While people with HAE have reported various triggers of attacks, common triggers for attacks include emotional stress, physical stress, and dental procedures. HAE may be caused by genetic changes in the SERPING1 gene (also called the C1NH gene) or in the F12 gene.
Garadacimab and the VANGUARD Clinical Trial
Garadacimab is a monoclonal antibody inhibiting factor XIIa, the first protein activated in the pathophysiology of HAE attacks. This treatment is the first factor XIIa inhibitor to be approved by the FDA. The approval follows positive data from the VANGUARD clinical trial.
The VANGUARD clinical trial was a phase 3, multicenter, randomized, double-blind, parallel-group study evaluating the safety and efficacy of garadacimab as prophylactic treatment for patients with HAE. Treatment with garadacimab was observed to reduce HAE attacks by a median of more than 99% and a least squares mean of 89.2% compared to placebo. The treatment also achieved more than 99% median reduction and 88% mean reduction in HAE attacks requiring on-demand therapy and more than 99% median reduction and 90% mean reduction in moderate to severe attacks, compared to placebo. Additionally, 62% of patients treated with garadacimab remained attack-free throughout the treatment period.
The most common adverse events were nasopharyngitis and abdominal pain, with an incidence rate greater than or equal to 7%.
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To learn more about HAE and other rare skin conditions, visit https://checkrare.com/diseases/skin-conditions/