Andembry now available in Japan to prevent HAE attacks
Pre-filled pen 'a significant advancement' in treatment
Andembry (garadacimab), a first-in-class treatment to prevent swelling attacks in people with hereditary angioedema (HAE), ages 12 and older, is now available in Japan.
The treatment was approved by Japan’s Ministry of Health, Labour and Welfare in February, Andembry is the first prophylactic (preventive) therapy in Japan that comes in a pre-filled pen to be administered as a subcutaneous, or under-the-skin, injection. Treatment begins with a 400 mg loading dose, followed by regular 200 mg monthly doses, according to its developer, CSL Behring.
“We are extremely proud to offer ANDEMBRY to patients in Japan as a new treatment option for HAE,” Izumi Yoshida, president and representative director of CSL Behring K.K., said in a company press release. “Featuring a first-in-class mechanism of action and the convenience of a once-monthly pre-filled pen, ANDEMBRY represents a significant advancement in HAE care.”
HAE is a genetic disease in which the overproduction of a signaling molecule called bradykinin triggers swelling attacks.
Andembry is an antibody-based therapy designed to block activated factor XII, a protein involved in the cascade leading to the production of bradykinin. Through this mechanism, it’s expected that Andembry will reduce bradykinin levels and lower the risk of swelling attacks.
Phase 3 trial showed treatment reduced swelling attack rate
“We are confident that ANDEMBRY will provide effective long-term prophylaxis of recurrent attacks, reducing the disease burden on patients and enhancing their quality of life,” Yoshida said.
Andembry’s approval was backed by data from a Phase 3 clinical trial called VANGUARD (NCT04656418). The study enrolled 64 HAE patients, ages 12 and older, who were randomly assigned to receive monthly injections of Andembry or a placebo for about six months.
Data showed that Andembry reduced the rate of swelling attacks by more than 85% compared with a placebo, with about two-thirds of Andembry-treated patients remaining attack-free throughout the trial.
Most VANGUARD participants enrolled in an open-label extension study (NCT04739059), in which all are being treated with Andembry to monitor the treatment’s long-term safety and efficacy. Early data from the ongoing extension study continued to demonstrate Andembry’s ability to reduce the frequency of swelling attacks.
The company is also testing the therapy in a Phase 3 trial (NCT05819775) in children with HAE ages 2-11, which is expected to wrap up in late 2026.
Andembry has been approved for HAE in the U.K. and Australia, as well as Switzerland and the European Union, and it is under review for approval in the U.S.
“This milestone is the culmination of CSL Behring’s 40 years of dedication to delivering innovative therapies in the HAE field,” Yoshida said.
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