Steve Bryson, PhD, science writer —

A delayed diagnosis is common for people with acquired hemophilia A (AHA), according to a new study that found that it took more than a month to get a diagnosis after the first bleeding episode for about 1 in 3 patients. Despite these delays, all AHA patients in the…

Prophylactic replacement therapy to prevent bleeds was more cost-effective than on-demand treatment of active bleeds for children and adolescents in China with moderate or severe hemophilia A without inhibitors, a study reported. The superior cost-effectiveness of prophylaxis over on-demand treatment was mainly driven by the on-demand costs of treating…

Note: This story was updated July 30, 2024, to clarify patients participating in AFFINE had moderately severe to severe disease, and that a secondary study goal involved the mean treated annualized bleeding rate. Giroctocogene fitelparvovec, an investigational gene therapy for adults with hemophilia A, is generally well…

TU7710, an experimental treatment for managing bleeds in people with hemophilia who have inhibitors, demonstrated an average half-life that ranged from 10.4 to 16.6 hours across different doses in a Phase 1a study with healthy volunteers. This extended half-life, that is, the time it takes for a medication’s levels…

Efanesoctocog alfa, a long-lasting factor VIII (FVIII) replacement therapy, has now been approved in the European Union for hemophilia A. It will be marketed under the brand name Altuvoct. Altuvoct can be used as an on-demand treatment for bleeding episodes, a once-weekly prophylactic (preventive) therapy to reduce the…

In real-world settings, Hemlibra (emicizumab-KXWH) can safely control bleeds in children with hemophilia A better than previous treatment regimens, a study reports. Hemlibra’s improved efficacy was more pronounced among patients with inhibitors, or neutralizing antibodies targeting factor VIII (FVIII), which is the clotting protein missing in people with…

The U.S. Food and Drug Administration (FDA) has approved an update to the prescribing information for Altuviiio (efanesoctocog alfa), a long-lasting factor VIII (FVIII) replacement therapy for hemophilia A, to include the full results from a clinical trial, the XTEND-Kids study. As requested by Sanofi, which markets…

Nearly all medical directors at hemophilia treatment centers in the U.S. said they would consider starting preventive Hemlibra (emicizumab-KXWH) in untreated infants with hemophilia A, ages 12 months and younger, a survey study reports. Most respondents also said they’d transition from FVIII replacement therapies, used on-demand to treat…

Hemlibra (emicizumab-KXWH) safely controlled bleeds among hemophilia A patients without inhibitors over nearly five years of preventive treatment, according to long-term data pooled from two Phase 3 clinical trials. “Five years of follow-up data showed a favorable benefit-risk profile of [Hemlibra] prophylaxis,” the researchers wrote in “…

Over seven years of follow-up, a single dose of Roctavian (valoctocogene roxaparvovec-rvox) sustainably reduced the yearly bleeding rate in adults with severe hemophilia A by up to 96%. That’s according to an update on a Phase 1/2 clinical trial (NCT02576795), launched in the U.K. in 2015, that’s…