Andembry is the first monthly Factor XIIa inhibitor for the prevention of HAE attacks and will be available to patients “before the end of June,” CSL announced.
The FDA on Tuesday signed off on CSL’s Factor XIIa blocker garadacimab to prevent attacks in patients with hereditary angioedema. The biologic will be marketed under the brand name Andembry.
Andembry’s label, which covers patients aged 12 years and up, marks the first ever regulatory approval in the U.S. for an anti-Factor XIIa prophylactic agent for hereditary angioedema (HAE), according to Tuesday’s release. CSL will launch Andembry “immediately,” working to make the once-monthly drug available to patients “before the end of June.”
Designed to be delivered via an injection under the skin, Andembry is a monoclonal antibody that targets and blocks activated Factor XII. Through this mechanism of action, Andembry “inhibits the top of the HAE cascade by targeting factor XIIa and provides sustained protection from attacks,” CSL said on Tuesday.
Andembry’s approval was backed by data from the pivotal Phase III VANGUARD study. Data, released back in February 2023, showed that 61.5% of patients remained free from HAE attacks after receiving the Factor XIIa blocker. Compared with placebo, Andembry cut the HAE attack rate by 86.5% on average.
Additionally, interim open-label extension data published in October 2024 in the journal Allergy support Andembry’s long-term efficacy and safety. Over nearly 14 months of treatment, 93% of treated patients graded their response to the therapy as “good” or “excellent.” Treatment-related toxicities were documented in 13% of patients, most of which were injection-site reactions.
Andembry, which is the first monoclonal antibody that was discovered and developed entirely by CSL, “offers people with this life-threatening condition long-term control over their disease along with a convenient administration method,” Bill Mezzanotte, head of R&D at CSL, said in the company’s statement.
Tuesday’s approval for Andembry comes just days after the FDA had to delay its target action date for KalVista’s own HAE hopeful sebetralstat. According to the biotech, the agency had to push its decision back by approximately four weeks due to “heavy workload and limited resources.” The FDA’s original target data was June 17.
Also playing in the HAE space is Intellia Therapeutics, which on Monday revealed three-year follow-up findings from an ongoing Phase I/II study of its investigational gene editor lonvoguran ziclumeran. Data presented at the European Academy of Allergy and Clinical Immunology showed a 98% mean decrease in monthly HAE attacks in all 10 dosed patients, who remained attack-free for a median of 23 months.
