Press Release: NEJM publishes once-weekly efanesoctocog alfa Phase 3 data demonstrating its potential to
Paris and Stockholm January 25, 2023 Pivotal study data published in continues to highlight the efficacy, safety, and pharmacokinetic profile of efanesoctocog alfa, an investigational treatment for hemophilia A. These data demonstrate that efanesoctocog alfa delivered normal to near-normal factor activity levels (>40%) for the majority of the week with once-weekly dosing. Efanesoctocog alfa is currently under priority review by the United States Food and Drug Administration (FDA) and the target action date for the decision is February 28, 2023.
Hemophilia A is a rare, lifelong condition in which the ability of a person's blood to clot properly is impaired, leading to excessive bleeds that can result in joint damage and chronic pain, and potentially impact their quality of life. The severity of hemophilia is determined by the level of clotting factor activity in a person's blood.
Investigator of the XTEND-1 Clinical Trial and Associate Professor at Michigan Medicine
The data from the pivotal XTEND-1 Phase 3 study published in show that efanesoctocog alfa met primary and key secondary endpoints, demonstrating clinically meaningful prevention of bleeds and superior bleed protection compared to prior factor VIII prophylaxis based on an intra-patient comparison. Treatment with efanesoctocog alfa prophylaxis resulted in significant and clinically meaningful improvements in physical health, pain, and joint health. Key results include:
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