On 4-17-2023, Novo Nordisk announced Health Canada’s approval of Alhemo®, their trademark for injectable concizumab, the first anti-tissue factor pathway inhibitor [TFPI] designed to prevent bleeds in Hemophilia A and B patients with inhibitors. The current Canadian approval supports relatively few Hemophilia B [Christmas disease] patients with inhibitors but generalization to Hemophilia A patients could follow as further approvals are given in Canada, Europe, and the USA. Thanks to Dean Willett, Precision BioLogic Inc, for the heads-up.
Here is the US National Institutes of Health concizumab research proposal filed in October 2019:
This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B with inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Participants who usually only take medicine to treat bleeds (on-demand) will be placed in one of two groups. In one group participants will get study medicine from the start of the study. In the other group participants will continue with their normal medicine and get study medicine after 6 months. The group will be decided by chance. Participants who usually take medicine to prevent bleeds (prophylaxis treatment) or who are already being treated with concizumab (study medicine) will receive the study medicine from the start of the study. Participants will have to inject themselves with study medicine every day under the skin. This can be done at home. The study doctor will hand out the medicine in the form of a pen-injector. The pen-injector will contain the study medicine. The study will last for about three years. Participants will have to come to the clinic for up to 33 times. The time between visits will be approximately 4 weeks for the first 6 to 12 months depending on the group participants are in and approximately 8 weeks for the rest of the study. At all visits, blood samples will be taken. Participants will be asked to record information into an electronic diary during the study and may also be asked to wear an activity tracker.