Efanesoctocog Alfa Prophylaxis Benefits Children With Hemophilia A

For children suffering from severe hemophilia A, a once-weekly preventive treatment with efanesoctocog alfa has shown promising results in maintaining high levels of factor VIII activity and effectively preventing bleeding. This conclusion comes from a study featured in the July 18 issue of the New England Journal of Medicine.

The research, spearheaded by Dr. Lynn Malec from the Versiti Blood Research Institute in Milwaukee, Wisconsin, detailed a phase 3, open-label study. The study included 74 male patients under the age of 12, all previously treated for severe hemophilia A. These young patients underwent a regimen of once-weekly prophylaxis with efanesoctocog alfa (50 IU/kg) over a 52-week period.

The findings from the research were promising. Notably, there were no instances of factor VIII inhibitors developing among the patients. Most of the adverse events reported were minor, and no serious adverse events were attributed to the treatment. Out of the 74 participants, 73 adhered to the study protocol. These patients exhibited median and model-based mean annualized bleeding rates of 0.00 and 0.61, respectively. Furthermore, a significant percentage of patients experienced no bleeding episodes: 64% had no treated bleeding episodes, 88% had no spontaneous bleeding episodes, and 82% had no bleeding into joints.

The efficacy of efanesoctocog alfa in resolving bleeding episodes was notable. Out of 43 bleeding episodes recorded, 95% were resolved with just one injection of the treatment. The study also measured factor VIII activity at steady state, finding it to be greater than 40 IU/dL for three days and over 10 IU/dL for nearly seven days post-administration. Additionally, the research noted a geometric mean terminal half-life of 40 hours.

Dr. Malec and her team emphasized the significance of these findings. "Once-weekly efanesoctocog alfa (50 IU per kilogram) provided high sustained factor VIII activity and highly efficacious protection against bleeding episodes in children with severe hemophilia A, a population in which this goal has been difficult to achieve without burdensome treatment regimens," they stated.

The study received funding from Sanofi and Sobi, the development partners for efanesoctocog alfa.

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