You can also add to your health journal encourading news about advances in the hemophilia management. Here are some of them:
Qfitlia - the first universal injection for hemophilia
Sanofi in 2025 received FDA approval for its hemophilia drug Qfitlia, making it the first universal treatment for the condition. It is approved for adults and adolescents with hemophilia A or B, with or without factor VIII or IX inhibitors. Qfitlia does not replace the missing clotting factor, but works by reducing the amount of a protein called antithrombin, which leads to an increase in thrombin, an enzyme critical for blood clotting. Qfitlia uses small interfering RNA technology, allowing for low-frequency treatment (about every two months), subcutaneous administration and small volumes. In clinical trials it reduced the number of bleeding episodes by about 70% more than standard treatment.
Rocatvian gene therapy for hemophilia A approved in the US
BioMarin in 2023 received FDA approval for its drug Rocatvian for the treatment of hemophilia A. It is a viral vector (delivering a gene into a cell) containing a gene that increases the blood clotting factor VIII - a protein deficiency that leads to hemophilia A, in which the main problem is internal and external bleeding. And many doctors are already excited about this drug. However, the bad news is that, like the hemophilia B drug Hemgenix, approved last year, Rocatvian costs millions. Specifically, $2.9 million per course of treatment. However, in the US, such treatments are often covered by insurance and BioMarin is obliged to refund the money if the treatment does not achieve the promised results.
New hemophilia drug Hemgenix has become the most expensive drug in history.
The US FDA has approved Hemgenix (produced by CSL Behring), a drug designed to treat hemophilia B. This is not only the first gene therapy for hemophilia, but also the most expensive drug in history. One dose of Hemgenix costs $3.5 million (although the treatment course consists of only one dose). Currently, patients with hemophilia in the US are treated with expensive intravenous injections of a special protein. It promotes blood clotting and prevents bleeding. CSL Behring representatives claim that the drug will ultimately reduce healthcare costs. The main argument is that patients will suffer from bleeding less often and will require fewer injections to normalize blood clotting.
Novo Nordisk aims to steal Roche's title of leader in hemophilia treatment
The reign of Roche's hemophilia drug Hemlibra in 2022 came to an end when Danish pharmaceutical giant Novo Nordisk released data from a phase 3 clinical trial of its antibody Concizumab for the treatment of people with both hemophilia A and hemophilia B. The results showed an 86% reduction in bleeds, exceeding the "wildest expectations" of Novo Nordisk's chief medical officer for rare diseases, Stephanie Seremetis. "This is a remarkable result," she said. "As a continuing active member of the hemophilia community, I believe this is a truly important development for hemophilia patients." The FDA is expected to approve the drug next year.
BioMarin has developed an effective drug against hemophilia A
BioMarin Pharmaceutical in 2021 published positive results from a phase 3 clinical trial of Valoctocogene Roxaparvovec, a gene therapy for the treatment of adults with severe hemophilia A. The company claimed that this was the largest gene therapy clinical trial ever, involving 134 patients. All study participants received a single dose of valoctocogene roxaparvovec and completed a year or more of follow-up. BioMarin said all goals were achieved, including an 84% reduction in bleeding (hemophilia is a condition that impairs blood clotting).