Severe Hemophilia

Efanesoctocog alfa prophylaxis exhibited high sustained factor VIII activity among younger children with severe hemophilia, according to findings published in The New England Journal of Medicine.

Elderly individuals with moderate or severe hemophilia had poorer joint or functional status and greater joint pain sensitivity than those with mild hemophilia or healthy controls, according to study results.

FDA approved valoctocogene roxaparvovec-rvox for the treatment of certain adults with severe hemophilia A, according to the agent’s manufacturer.

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.

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 accepted a biologics license application resubmission for valoctocogene roxaparvovec as treatment for adults with severe hemophilia A, according to a press release from the agent’s manufacturer.

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.

Once-monthly fitusiran prophylaxis improved outcomes compared with on-demand treatment for patients with severe hemophilia A or B without inhibitors, according to study results presented at ASH Annual Meeting and Exposition.

An IV injection of BIVV001 induced high sustained factor VIII activity levels among men with severe hemophilia A, according to results of a phase 1/phase 2 open-label trial published in The New England Journal of Medicine.