Most children with hemophilia A given Altuviiio (efanesoctocog alfa) once a week were bleed-free throughout about a year of treatment. That’s according to top-line data from the Phase 3 XTEND-Kids trial (NCT04759131) announced by Sobi and Sanofi, the therapy’s co-developers, less than a month after…
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Sangamo Therapeutics and Pfizer have entered a global collaboration and license agreement to develop and market gene therapy programs for hemophilia A, including SB-525. SB-525 was cleared by the U.S. Food and Drug Administration (FDA) in January as an investigational new drug (IND) for the…
The ratio between the blood clotting protein factor VIII (FVIII) and the von Willebrand factor (VWF) may be a reliable biomarker of recovery and relapse in patients with acquired hemophilia A (AHA), a study suggests. The research, “The factor VIII:C/VWF:Ag ratio as a useful tool to…
Mim8 (denecimig) is an antibody therapy being developed to prevent or reduce the frequency of bleeding episodes in people with hemophilia A, with or without neutralizing antibodies (inhibitors).
The U.S. Food and Drug Administration (FDA) has approved BioMarin Pharmaceutical‘s Investigational New Drug (IND) application for its gene therapy candidate to treat severe hemophilia A. FDA’s decision on BMN 270, also known for its generic name valoctocogene roxaparvovec,…
A young woman who sought emergency care for severe pain and swelling of her left hand five weeks after giving birth was ultimately diagnosed with acquired hemophilia A — caught early enough that she did not require surgery. That’s according to a new report detailing the woman’s case, which,…
Catalyst Biosciences has begun enrolling patients in a Phase 2/3 clinical trial of marzeptacog alfa (activated) as a preventive treatment for hemophilia A or B. Some patients develop immune responses — or inhibitors — to clotting factor treatments, preventing the therapies from being able to stop bleeding. Catalyst’s therapy, also known…
Since hemophilia was discovered, it has been accepted as a male disease. No one questions that a man is struggling with hemophilia. A man with a bleeding disorder living in a developed country is rarely denied access to healthcare. When he receives a diagnosis, he is told that…
Interim results from the Phase 3 ASPIRE study indicate that patients with hemophilia A showed continuous improvement in joint health for nearly three years while receiving prophylactic dosing of Bioverativ and Sobi’s Eloctate. The interim results were reported in a study titled, “Improved joint health in subjects with severe haemophilia A…
Once-monthly injection of the investigational RNA therapy Fitusiran reduced the levels of the protein antithrombin, a natural inhibitor of several components of the clotting system, and restored the balance of clotting elements production in adult patients with hemophilia A or B. These findings were featured in the article “Targeting…