A few years ago, I went to a tattoo convention. Before I could enter, I was required to sign a liability release. The form said that people with bleeding disorders (and a long list of other conditions) should not get tattoos. Needless to say, I am not always a…

A single dose of the experimental gene therapy SPK-8016 showed promising safety and efficacy in lowering the frequency of bleeds and the need for infusions in men with severe hemophilia A. These preliminary findings from an ongoing Phase 1/2 trial (NCT03734588) were presented by Spencer Sullivan, MD,…

The investigational medication marzeptacog alfa activated (MarzAA) showed promise at stopping bleeding in animal models of hemophilia. The findings were presented in two posters at the Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) by researchers from Catalyst Biosciences, which is developing MarzAA. MarzAA is…

Kovaltry (octocog alfa) is effective and safe as a preventive treatment for children with hemophilia A, a European real-world study reports. The study, “Real-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a…

Severe hemophilia A patients using Esperoct (turoctocog alfa pegol) as a preventive treatment do well, with fewer bleeds or breakthrough bleeds, lower costs, and better life quality compared with other extended half-life (EHL) therapies, a modeling analysis suggested. The…

When given as a preventive treatment for nearly six years, Hemlibra (emicizumab) safely and effectively lowered the number of bleeding episodes in patients with severe hemophilia A, according to data from a Phase 1 clinical trial and its long-term extension study. Importantly, reductions in the number of bleeds…

Note: This story was updated Jan. 8, 2021, to clarify that the second set of experiments in mice and non-human primates used a research construct, not the gene therapy. Generation Bio has announced new data showing that its investigational non-viral gene therapy platform can increase levels of the clotting…

Prime Therapeutics and Takeda will work together to establish the value of Advate, relative to its costs and benefits, as a treatment for hemophilia A. According to Prime, work comparing factors like total health care costs and emergency hospital visits allow the company to better evaluate and distinguish…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to marzeptacog alfa activated (MarzAA), a lab-made version of clotting factor VIIa for the subcutaneous (under-the-skin) treatment of episodic bleeds in hemophilia A and B patients with inhibitors. MarzAA, developed by Catalyst Biosciences, will…

Using a combination of genetic and functional approaches, scientists have identified new mutations in parts of the F8 gene that do not code for factor VIII (FVIII) in a small group of French and Canadian patients with hemophilia A. These results were described in the study, “The…