Timothy Craig, DO, Tenured Professor of Medicine, Pediatrics, and Biomedical Sciences at Penn State University, Principal Investigator of the VANGUARD trial, discusses the recent approval of Andembry (garadacimab) for patients ages 12 years and older with hereditary angioedema (HAE).
HAE is a rare genetic condition characterized by recurrent episodes of severe swelling of the skin and mucous membranes. These attacks often affect the skin, gastrointestinal tract, and upper airway. The most common areas of the skin that are affected include the face, hands, arms, legs, genitals, and buttocks. 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. In some cases, the cause is not yet known.
Garadacimab is a monoclonal antibody inhibiting factor XIIa that is administered as a once-monthly subcutaneous prophylactic treatment for HAE attacks. It has recently been approved for patients ages 12 years or older by the U.S. Food and Drug Administration (FDA) based on results from the VANGUARD clinical trial.
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 in 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%.
For more information, visit www.andembry.com
To learn more about HAE and other rare genetic conditions, visit https://checkrare.com/diseases/congenital-and-genetic-conditions/