Hemlibra Can Now Treat People in Japan With Acquired Hemophilia A

Japan’s Ministry of Health, Labor and Welfare has extended Hemlibra (emicizumab)’s approval to include routine prophylaxis to prevent or lower the frequency of bleeds in people with acquired hemophilia A.

Its decision comes seven months after Hemlibra’s maker, Chugai Pharmaceutical, applied for extended use. Hemlibra was previously approved in Japan to treat congenital hemophilia A, which is caused by genetic mutations.

“We are very pleased to offer Hemlibra as a treatment for acquired hemophilia A, following congenital hemophilia A,” Osamu Okuda, Chugai’s president and CEO, said in a press release.

“The approval today allows an additional measure for patients to effectively control bleeding at an earlier stage after diagnosis. We believe this will provide flexibility in the treatment strategy for acquired hemophilia A and may lead to the realization of better treatment according to each patient’s condition,” Okuda added.

Hemophilia A is caused by insufficient activity of the clotting protein called factor VIII (FVIII), most commonly because of a mutation in the gene that provides instructions for making this protein. Whereas mutations are responsible for congenital hemophilia A, the disease’s acquired form is caused by the immune system mistakenly attacking and destroying FVIII.

Hemlibra is a bispecific antibody designed to simultaneously bind to two targets, factor IX and factor X, to which FVIII normally binds to initiate blood clotting. By binding these proteins, Hemlibra can functionally replace the missing FVIII in patients.

The medication, which is administered as a weekly subcutaneous (under-the-skin) injection, is widely approved as a preventive treatment for congenital hemophilia A.

“Hemlibra was created by applying Chugai’s proprietary bispecific antibody technology,” Okuda said. “Chugai’s mission is to dedicate ourselves to adding value for patients through innovative medicines. We will continue working with all our efforts to realize innovation for patients awaiting new treatments.”

The treatment’s approval for acquired hemophilia A in Japan was supported by data from a Phase 3 trial called AGEHA (JapicCTI-205151), which tested Hemlibra in adults in that country with the condition.

According to Chugai, primary analyses from the trial will be presented next month at the 30th International Society on Thrombosis and Hemostasis, being held in London and online.