JW Jungwoo Pharmaceutical "Hemlibra Effective for Mild to Moderate Hemophilia A"

Publication of Phase 3 Clinical Trial Results for Non-Antibody Patients in an International Journal

JW Pharmaceutical announced on the 6th that the Phase 3 clinical trial results proving the efficacy and safety of the hemophilia A treatment 'Hemlibra (active ingredient emicizumab)' for patients with mild to moderate severity were published in the online edition of the international journal 'The Lancet Hematology 2023.'

Hemlibra, a treatment for Hemophilia A.
Photo by JW Choongwae Pharmaceutical

Hemlibra is a treatment for hemophilia A caused by a deficiency of coagulation factor VIII. It simultaneously binds to coagulation factors IX and X, mimicking the mechanism of factor VIII. It is the only hemophilia A treatment that can be used for both antibody-positive patients who have developed resistance to existing treatments and antibody-negative patients.

Hemophilia A is classified based on the activity level of coagulation factor VIII into mild (factor VIII activity over 5% and less than 40%), moderate (1% or more to 5% or less), and severe (less than 1%). Hemlibra is currently approved in South Korea as a prophylactic treatment for severe hemophilia A.

Professor Claude Negrier of Claude Bernard Lyon 1 University in France, along with 18 other faculty members, conducted a clinical trial over approximately 55 weeks involving 72 antibody-negative patients with mild to moderate hemophilia A at 22 institutions across Europe, North America, and South Africa. The researchers administered Hemlibra once weekly for the first 4 weeks, then allowed patients to choose dosing intervals of once weekly, once every two weeks, or once every four weeks, evaluating bleeding frequency and thrombotic adverse reactions.

The clinical results showed that the annualized bleeding rate (ABR), which was 10.1 episodes before treatment, decreased to 0.9 episodes after Hemlibra administration. Among these, the ABR for joint bleeding requiring treatment and spontaneous bleeding were both 0.2 episodes. Individually, the ABR for 21 mild patients decreased from 20.2 episodes before Hemlibra to 2.4 episodes after treatment, and for moderate patients, it decreased from 6.0 to 2.2 episodes. In the group of patients who had previously received coagulation factor therapy as prophylaxis, the ABR improved from 12.2 episodes before the trial to 2.2 episodes after, and in those treated during bleeding episodes, from 8.0 to 2.4 episodes. Eight patients experienced no bleeding during the trial. Regarding safety, mild injection site reactions occurred in 15 patients, but there were no cases of death or thrombotic microangiopathy.

Based on these clinical results, Hemlibra was approved earlier this month by the European Union (EU) as a prophylactic treatment for antibody-negative patients with moderate hemophilia A. In South Korea, it is prescribed for antibody-positive severe hemophilia A patients, and on the 9th of last month, the Health Insurance Review & Assessment Service recognized its health insurance reimbursement appropriateness for antibody-negative severe hemophilia A patients.

A JW Pharmaceutical official stated, "This clinical trial shows that Hemlibra is effective in preventing bleeding not only in severe but also in mild and moderate hemophilia A patients," adding, "We will strive to ensure that more hemophilia A patients can benefit from this treatment."

Hemlibra, developed by Chugai Pharmaceutical, a subsidiary of the global pharmaceutical company Roche, was domestically developed and licensed by JW Pharmaceutical in 2017 and launched as a treatment for severe hemophilia A in 2020.