Endothelial cells — those that line the inside of blood vessels — derived from stem cells of people with hemophilia A and modified to produce functional factor VIII were able to ease blood loss in a mouse model of the disease, a study reported. These findings support the potential of induced…

Dosing has begun in a Phase 2b clinical trial evaluating the safety and efficacy of SerpinPC, Centessa Pharmaceuticals’ investigational therapy for hemophilia. The open-label study, called PRESent-2 (NCT05789524), is part of the company’s registrational program for hemophilia B, which includes multiple clinical trials aiming to…

The relapse rate is higher in older patients with acquired hemophilia A (AHA) treated with immunosuppressive therapies, but it doesn’t affect their overall survival compared with those who don’t relapse, a study suggests. The study, “Relapse pattern and long‐term outcomes in subjects with acquired haemophilia A,” was published in…

The U.K.’s National Institute for Health and Care Excellence (NICE) has recommended Altuvoct (efanesoctocog alfa) be available through the National Health Service (NHS), England’s public healthcare system, as a treatment option to manage and prevent bleeds in people with severe hemophilia A, ages 2 and older. This decision…

Health Canada has approved Hemlibra (emicizumab) as a treatment to prevent or reduce bleeding in Hemophilia A patients in Canada who have factor VIII inhibitors. Hemlibra is an antibody therapy designed to combine factors IX and X of the blood clotting series and promote clotting. Restoring the…

Pfizer said the U.S. Food and Drug Administration (FDA) will review the company’s request to expand approval of its preventive treatment Hympavzi (marstacimab) to cover all people with hemophilia A or B ages 6 and older. Hympavzi is FDA-approved to treat hemophilia A and B in patients ages…

A gene therapy approach using a so-called antibody-drug conjugate (ADC) conditioning regimen led to safe and sustained production of factor VIII (FVIII) in platelets, and prevented joint bleeding in a mouse model of hemophilia A, according to new research. The study, “Nongenotoxic antibody-drug conjugate conditioning…

Catalyst Biosciences announced positive interim results from a Phase 2/3 clinical trial (NCT03407651) of the Factor VIIa variant marzeptacog alfa (activated) (MarzAA) in the preventative treatment of patients with hemophilia A or B with inhibitors. The study, aiming to include up to 12 patients, is recruiting in Russia…

A new study from Finland demonstrated that neutralizing antibodies (inhibitors) can develop in people with untreated hemophilia, and that early intensive prevention might prevent bleeding and inhibitor development. The research report, titled “Inhibitor development in previously untreated patients with severe haemophilia A: a nationwide multicentre study in Finland,” was published in…

Healthcare costs, particularly related to disease-specific medications, are especially high for people with hemophilia A or hemophilia B on preventive treatments and/or for those with inhibitors. That’s according to a study in France, which also found that patients have a significant clinical burden, with a higher risk of…