Garadacimab-gxii FDA Approval – HAE Treatment
- The Food adn drug Administration has given the green light to Andembry (garadacimab-gxii) as a prophylactic treatment for hereditary angioedema (HAE) in patients 12 and older.
- HAE is a rare genetic condition characterized by painful swelling episodes affecting various body parts, including the abdomen, larynx, face, and extremities.
- The approval of garadacimab-gxii, a novel treatment for hereditary angioedema (HAE), is based on the phase 3 VANGUARD clinical trial.
The FDA has approved Andembry (garadacimab-gxii), a important growth in hereditary angioedema (HAE) treatment. This novel therapy offers a once-monthly, subcutaneous self-injection, providing a convenient prophylactic option for those aged 12 and older. clinical trials demonstrated ample reductions in HAE attacks for patients using this novel treatment. The approval marks a pivotal shift,offering renewed hope for individuals managing this challenging condition. Find all the details on our reporting on News Directory 3. What further innovations await the HAE community? Discover what’s next …
FDA Approves Andembry for Hereditary Angioedema Treatment
Updated June 17, 2025
The Food adn drug Administration has given the green light to Andembry (garadacimab-gxii) as a prophylactic treatment for hereditary angioedema (HAE) in patients 12 and older. CSL’s Andembry targets factor XIIa and is administered via a once-monthly subcutaneous self-injection. The injection takes 15 seconds or less using a citrate-free autoinjector.
HAE is a rare genetic condition characterized by painful swelling episodes affecting various body parts, including the abdomen, larynx, face, and extremities. Symptoms can manifest in childhood or adolescence and may worsen during puberty. Type 1 HAE is the most common form, accounting for about 85% of cases.
The approval of garadacimab-gxii, a novel treatment for hereditary angioedema (HAE), is based on the phase 3 VANGUARD clinical trial. The study involved patients with HAE type 1 or type 2, aged 12 and older. Participants were given either garadacimab or a placebo. The results showed that 62% of patients on garadacimab remained attack-free during the trial.
Compared to the placebo,garadacimab significantly reduced HAE attacks by a median of over 99% and a mean of 89.2%. The treatment also lead to a median reduction of over 99% in HAE attacks requiring on-demand therapy, as well as a median reduction of over 99% in moderate or severe attacks.
“[Garadacimab] the first monoclonal antibody discovered and developed entirely by CSL, offers people living with this life-threatening condition long-term control over their disease along with a convenient administration method,” Bill Mezzanotte, MD, executive vice president and head of R&D at CSL, said.
Tim Craig, professor of medicine, pediatrics, and biomedical sciences at Penn State University, noted the importance of this new option. He said it allows doctors to inhibit the top of the HAE cascade by targeting factor XIIa.
“We now have a new option to manage this condition through a new target, as it allows us for the first time to inhibit the top of the HAE cascade by targeting factor XIIa,” Craig said.
Anthony J. Castaldo, CEO and Chairman of the Board, US HAE Association and HAE International, welcomed the new treatment option.He said it offers people with HAE another choice for lessening the burden associated with this lifelong condition.
“[Garadacimab] a novel once-monthly subcutaneous treatment that inhibits factor XIIa, is a welcome addition to the HAE treatment landscape,” Castaldo said.”People with HAE now have another choice for lessening the burden associated with this lifelong condition and realizing the community’s shared goal of experiencing life to the fullest.”
What’s next
With the FDA approval, andembry (garadacimab-gxii) is poised to become a significant option in managing hereditary angioedema, offering patients a convenient, once-monthly prophylactic treatment to reduce the frequency and severity of HAE attacks.