JW Pharmaceutical reported on Wednesday that 91% of patients in Japan experienced no adverse drug reactions from its hemophilia treatment, Hemlibra.
A research team led by Professor Midori Shima of Nara Medical University conducted a study to evaluate the long-term safety and efficacy of Hemlibra in Japan. The study involved 134 patients with congenital hemophilia A who had developed antibodies, spanning from May 2018 to January 2023.
The team monitored two groups over a period of up to three years: the safety analysis group comprising all 134 patients, and the ‘efficacy analysis group’ consisting of 101 new patients.
The study included 116 severe cases (86.6% of participants), with ages ranging from infants to 78-year-olds.
Safety assessments revealed that 122 out of 134 patients (91%) in the safety analysis group experienced no adverse drug reactions (ADRs). Notably, 30 patients underwent surgeries during the study period, including complex procedures such as joint replacements.
Even when hemostatic agents like bypassing agents (BPA) were used for bleeding control, no thromboembolic events (TE) or thrombotic microangiopathy (TMA) were observed.
The efficacy analysis group showed a median annual bleeding rate (ABR) of 1.3, indicating stable outcomes.
Remarkably, 53 patients (39.6%) maintained a zero bleed status throughout the treatment period, experiencing no bleeding episodes.
The findings were published in the international journal Haemophilia.
A JW Pharmaceutical spokesperson emphasized that these results demonstrate Hemlibra’s long-term safety in high-risk situations, such as surgeries, for Asian populations with genetic predispositions similar to Koreans.