Regulators in Japan Asked to Extend Hemlibra to Acquired Hemophilia A

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Chugai Pharmaceutical is asking regulatory authorities in Japan to extend the use of Hemlibra (emicizumab) in preventing or reducing the frequency of bleeding episodes to people with acquired hemophilia A.

Hemophilia A is caused by genetic mutations that make the body unable to produce a fully functional version of the factor VIII (FVIII) clotting protein. In acquired hemophilia A, low levels of FVIII result from the immune system erroneously attacking and destroying the protein, rather than a genetic mutation.

Hemlibra is a bispecific antibody — an antibody that binds to two targets simultaneously — that is designed to mimic the function FVIII naturally has in the body. The medication, which is administered by a subcutaneous (under-the-skin) injection, is widely approved to manage symptoms of hemophilia A, including in Japan.

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However, it is not approved in Japan to treat people with acquired hemophilia A.

“Acquired hemophilia A is a disease with high unmet medical needs,” Osamu Okuda, president and CEO of Chugai, said in a press release. Okuda also noted that acquired hemophilia A in Japan is “designated as an intractable disease,” whose definition includes disorders that lack clearly established treatments.

“More treatment options are needed for this disease, and I am glad that we have filed application for Hemlibra for this indication,” Okuda added.

Chugai’s application to Japan’s Ministry of Health, Labour and Welfare (MHLW) is supported by data from an ongoing Phase 3 clinical trial in Japan, called AGEHA (JapicCTI-205151), which aims to enroll 11 adults with acquired hemophilia A. Its main goal is to evaluate the safety of Hemlibra in these patients. Treatment efficacy and pharmacological data will also be assessed.

Notably, several case reports in recent years have suggested that Hemlibra can be safely used to manage bleeds in people with acquired hemophilia A.

In early October, the MHLW designated Hemlibra an orphan drug as a potential treatment for acquired hemophilia A. In Japan, this designation is reserved for therapies aiming to treat serious conditions affecting fewer than 50,000 people for which there is a high unmet need. Through this designation, Chugai’s application requesting the therapy’s approval for this patient group has been given priority review.

“Chugai will continue working toward obtaining approval to contribute to the treatment of acquired hemophilia A,” Okuda said.