Takhzyro Approved in Japan as Prophylactic for HAE Swelling Attacks
Takhzyro (lanadelumab) has been approved in Japan to prevent acute swelling attacks in people, 12 and older, with hereditary angioedema (HAE).
An estimated 2,000 to 3,000 people with HAE live in Japan, but fewer than 500 have been diagnosed due to a lack of awareness, according to Takeda, which markets the therapy.
“In addition to the burden of debilitating and potentially life-threatening HAE attacks, the unpredictable nature of this disease presents significant challenges to patients and their support networks,” Naoyoshi Hirota, general manager at Takeda Development Center in Japan, said in a press release. “We hope Takhzyro, a new treatment option for patients in Japan living with HAE … will contribute to HAE treatment.”
Takhzyro has been approved to prevent HAE swelling attacks in patients 12 and older in the U.S. and in the European Union since 2018. According to Takeda, Takzyro is currently available in more than 50 countries worldwide, with additional regulatory submissions underway.
In HAE, excessive levels of a signaling molecule called bradykinin trigger bouts of swelling. Takhzyro is an antibody-based medication that stops these swelling attacks by blocking the activity of an enzyme called plasma kallikrein, which produces bradykinin. The medication is administered via subcutaneous (under-the-skin) injection, at a dosage of 300 mg every two weeks.
Takeda’s application for Takhzyro in Japan was supported by data from the Phase 3 HELP trial (NCT02586805) and its open-label extension study (NCT02741596).
Results from the initial 26-week trial showed that Takhzyro reduced the average number of monthly HAE attacks by 87% compared with a placebo. Further data from the extension study indicated the reduction in swelling attacks persisted for up to 2.5 years of treatment, and that Takhzyro markedly improved health-related life quality.
The submission in Japan was also supported by data from another Phase 3 trial (NCT04180163) that evaluated the safety and efficacy of Takhzyro in 12 Japanese HAE patients, 12 and older. Over the 26-week efficacy evaluation period, 41.7% of participants in this study were free of swelling attacks, according to Takeda.
The most common side effects associated with Takhzyro treatment in clinical trials were injection site reactions, which may include pain, redness, and/or bruising at the injection site. Other common side effects include upper respiratory infections, headache, rash, muscle pain, dizziness, and diarrhea.