Antibody therapy Mim8 (denecimig) was well tolerated and provided effective bleed control in children with hemophilia A regardless of inhibitor status. That’s according to interim data from the now-complete Phase 3 FRONTIER3 study (NCT05306418), which enrolled children ages 1-11. These findings were consistent with recent data from…
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Patients with hemophilia A who switched to Kovaltry (octocog alfa) — a recombinant, or lab-made, factor VIII (rFVIII) replacement therapy given at home — had significantly fewer bleeds than with their previous standard half-life replacement therapies, a real-world study in Italy reported. The therapy also reduced the frequency…
Three years after receiving the one-time gene therapy Roctavian (valoctocogene roxaparvovec) at trial, the majority of men with severe hemophilia A in the Phase 3 GENEr8-1 study were off standard replacement therapies and did not experience any bleeds requiring treatment. That’s according to new data announced in a…
Hemophilia A is a bleeding disorder caused by a deficiency in the blood-clotting factor VIII (FVIII) protein. Standard treatment usually relies on factor replacement therapy, which delivers a working version of the missing clotting factor to prevent or stop bleeds. However, the immune systems of some patients — about…
Embracing the start of 2024, I reflect on the friendships I’ve made within the hemophilia community over the past year. As a woman navigating the complexities of hemophilia B and von Willebrand disease, these connections have become a source of strength and solidarity for me. Each person…
Preventive treatment with factor VIII (FVIII) — the missing blood clotting protein in hemophilia A — was not fully effective at preventing MRI-detected joint changes in boys with severe disease, according to a Canadian study. Regular evaluation of joints by MRI or ultrasounds, however, may help in identifying those…
In a society where reality television is king, passing judgment is easier than ever. Contestants in competitions such as “Dancing with the Stars” and “American Idol” are eager to hear the judges’ remarks in hopes of advancing to the next round. On shows like “Judge Judy,” litigants demand arbitration to…
Interim results of Phase 3 clinical studies investigating two major challenges in hemophilia — the development of inhibitors (antibodies to the drug used to treat bleeding episodes) and the need for repeated venous injection of blood clotting protein Factor VIII (FVIII) — were recently released. The results were presented by…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to efanesoctocog alfa, an investigational factor VIII (FVIII) replacement therapy for treating hemophilia A, Sanofi, one of the therapy’s developers, announced in a press release. This designation is given to accelerate the development and review of medications…
More than 21 years ago, when my first child was born and diagnosed with severe hemophilia A, he was very fortunate to have first-generation clotting factors available. These were concentrates stabilized with albumin that were very safe. When the factor was reconstituted with sterile water, a seemingly…