Catalyst Biosciences recently announced updates on two of its lead compounds — Factor VIIa (FVIIa) marzeptacog alfa (activated) (MarzAA) and Factor IX (FIX) dalcinonacog alfa (DalcA) — for treatment of hemophilia A or B with inhibitors, and for hemophilia B. DalcA is a potent recombinant (artificial) Factor IX prophylactic therapy designed to prevent acute bleeding…
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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…
The safety and efficacy of emicizumab in patients with severe hemophilia A (HA) were recently evaluated in a study, “Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A,” published in The New England Journal Of Medicine. Hemophilia A is caused by a lack of blood clotting factor…
The U.S. Food and Drug Administration (FDA) has approved Novo Nordisk’s concizumab, which will be sold under the brand name Alhemo, as a daily treatment to prevent or reduce the frequency of bleeding episodes in certain adults and adolescents with hemophilia. Eligible patients are those with hemophilia…
In children with severe hemophilia A who are undergoing treatment to remove inhibitors, adding Hemlibra (emicizumab-KXWH) was associated with faster immune tolerance and fewer treated bleeds. Adding Hemlibra also did not increase direct treatment costs during immune tolerance induction, study data showed. The work was partially funded by…
Health Canada, the agency that oversees health decisions and regulations in Canada, has approved Afstyla (lonoctocog alfa) for the treatment and prevention of bleeding in adults and children with hemophilia A. In May, the U.S. Food and Drug Administration (FDA) approved Afstyla for the same indications. Developed by CSL Behring, Afstyla is…
Brazilian hemophilia A patients who are 19 or older had significantly higher median care costs per patient per year than younger patients, a study reported. The study also revealed an increase in median costs for patients with more severe disease — that is, those who have less than 1%…
Investigational gene therapy SB-525 can safely induce durable clotting factor VIII (FVIII) activity in patients with severe hemophilia A, preliminary data from the Phase 1/2 Alta clinical trial show. The trial’s most recent results were discussed at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress in…
Koāte (human antihemophilic factor) is a plasma-derived factor replacement therapy approved to prevent and control bleeding episodes or help manage bleeds during surgery in people with hemophilia A.
The first patient has been dosed in China in a registrational clinical trial of BBM-H803, a one-time investigational gene therapy that Belief BioMed is developing to prevent bleeding in hemophilia A. The Phase 1/2/3 clinical trial (CTR20233400) is testing how safe and well tolerated BBM-H803 is as a…