CSL announced that its novel monoclonal antibody therapy, Andembry™ (garadacimab-gxii), had been granted approval by the U.S. Food and Drug Administration (FDA) for the prevention of HAE attacks in patients aged 12 and older. Andembry is the first and only approved treatment that targets activated Factor XIIa, a key initiator of the biochemical pathway responsible for HAE flare-ups.
The approval followed compelling results from the pivotal Phase III VANGUARD trial, which showed that Andembry reduced the median monthly attack rate by more than 99%, with 62% of patients remaining completely attack-free over a six-month period. The therapy also cut moderate-to-severe episodes by 93.6%, and reduced reliance on rescue medication by 91.2%.
Administered via a pre-filled autoinjector in under 15 seconds, Andembry is expected to offer greater convenience than current therapies that require frequent dosing or customisation.
“This approval marks a major step forward for people living with HAE,” said Dr Bill Mezzanotte, Executive Vice President and Head of R&D at CSL. “Andembry is the first therapy to emerge entirely from CSL’s own discovery efforts, and it provides patients with the potential for greater control, freedom, and quality of life.”
HAE is a rare and potentially life-threatening condition affecting approximately one in 50,000 individuals, often causing sudden, painful swelling in the face, limbs, abdomen, or throat.
With earlier regulatory approvals already secured in Europe, the UK, Australia, and Japan, CSL confirmed that Andembry will be launched in the United States before the end of June 2025.
