Hemophilia A

Prophylactic concizumab conferred significant reductions in treated spontaneous and traumatic bleeds among patients with hemophilia A or B without inhibitors, a primary analysis of the randomized phase 3 explorer8 study showed.

Once-weekly prophylaxis with a high-sustained factor VIII replacement therapy exhibited effective bleed protection for children with severe hemophilia A, according to the agent’s manufacturer.

Marstacimab reduced annualized bleeding rate compared with prophylaxis and on-demand IV regimens for patients with hemophilia A or B, according to the agent’s manufacturer.

NEW ORLEANS — Emicizumab-kxwh prophylaxis appeared effective and safe for infants with severe hemophilia A without factor VIII inhibitors, according to study results presented at ASH Annual Meeting and Exposition.

The FDA granted priority review to efanesoctocog alfa as prophylactic treatment for patients with hemophilia A.

A once-weekly dose of prophylactic efanesoctocog alfa conferred a significant reduction in annualized bleed rate compared with the use of prophylactic factor VIII agents, results of the pivotal phase 3 XTEND-1 study showed.

The FDA granted breakthrough therapy designation to efanesoctocog alfa for treatment of hemophilia A.

Valoctocogene roxaparvovec induced endogenous factor VIII production and significantly reduced bleeding and factor VIII concentrate use relative to factor VIII prophylaxis in patients with severe hemophilia A, according to study results.

In this video, Allyson Pishko, MD, MSCE, highlighted two studies from ASH Annual Meeting and Exposition that investigated fitusiran (Sanofi) as treatment for both hemophilia A and B.

A once-monthly subcutaneous prophylaxis dose of fitusiran appeared safe and effective for people with hemophilia A or B with inhibitors, according to results of a randomized phase 3 study presented at ASH Annual Meeting and Exposition.