JW Pharmaceutical said Monday the first study to demonstrate the efficacy and safety of Hemlibra (emicizumab) for Korean pediatric patients with severe non-inhibitor hemophilia A was featured in the journal Blood Research.

While international studies have already shown Hemlibra’s effectiveness in reducing bleeding episodes in pediatric patients, a JW Pharmaceutical representative noted the study’s significance as the “first to confirm Hemlibra’s efficacy and safety using actual prescription data for pediatric patients in Korea.”

Hemlibra is a treatment designed to mimic the activity of clotting factor VIII (antihemophilic factor), which is deficient in hemophilia A patients. The drug utilizes bispecific antibody technology, allowing it to bind to clotting factors IX (Christmas factor) and X (Stuart-Prower factor) simultaneously. Hemlibra is the only treatment for hemophilia A available for both inhibitor and non-inhibitor patients, unlike conventional factor VIII therapies, and offers sustained prophylactic effects through a once-monthly subcutaneous injection.

The study, led by Professor Kim Sung-eun from the department of pediatrics at Kyungpook National University Hospital, followed 21 pediatric patients aged 0-11 with severe non-inhibitor hemophilia A, from June 2020 through March 2024. Patients received Hemlibra for over six months, with researchers tracking their annual bleed rate (ABR) and annual joint bleed rate (AJBR).

Results showed a significant decrease in ABR, from 7.04 episodes to 0.41 episodes after Hemlibra treatment, while AJBR dropped from 2.28 episodes to 0.21 episodes. 18 of the 21 patients experienced no bleeding episodes within six months of starting treatment.

In terms of safety, no serious or life-threatening side effects, such as thromboembolism or systemic hypersensitivity reactions, were observed.

The full study detailing these findings was published in the Korean journal Blood Research.