Treatment with platelets stripped of their natural sugar molecule coating helped to prevent inhibitors, or neutralizing antibodies, from forming against clotting factor VIII (FVIII) replacement therapy in a mouse model of hemophilia A, a study reported. Platelets promote blood clotting, and they are generally thought to boost inflammation…
Adynovate (antihemophilic factor, rurioctocog alfa pegol), sold as Adynovi in Europe, is a recombinant (lab-made) clotting factor VIII (FVIII) concentrate given as an intravenous (into-the-vein) injection to prevent and control bleeding in children and adults with hemophilia A.
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Baudax Bio’s investigational regulatory T-cell therapy TI-168 for the treatment of hemophilia A with inhibitors. Orphan drug status is given to therapies intended to treat rare conditions, defined as those affecting less than 200,000 people in…
Alprolix (eftrenonacog alfa) is an approved therapy that is used to prevent and control bleeds, including those occurring during surgery, in adults and children with hemophilia B.
Altuviiio (efanesoctocog alfa) has been approved in Japan and Taiwan as a treatment to prevent and control bleeding in people with hemophilia A. The Japanese Ministry of Health, Labor, and Welfare (MHLW) granted Altuviiio marketing authorization on Sept. 25, according to Sanofi, the company that markets…
Hemgenix (etranacogene dezaparvovec), formerly known as AMT-061 and EtranaDez, is a gene therapy given as a one-time intravenous (into-the-vein) infusion to treat bleeding episodes in hemophilia B.
Treatment with Idelvion (albutrepenonacog alfa) was effective in a real-world setting at controlling bleeds for five people with hemophilia B at a center in Spain. That’s according to data reported in the study, “Benefits of rIX-FP prophylaxis in patients with Haemophilia B: real-world evidence from…
Nuwiq (simoctocog alfa) is an approved therapy that works to prevent and control bleeding episodes, including those occurring during surgery, in adults and children with hemophilia A.
Roctavian (valoctocogene roxaparvovec-rvox) is an approved one-time gene therapy that’s intended to reduce the risk of bleeds in certain adults with severe hemophilia A.
The U.S. Food and Drug Administration (FDA) is considering updating the prescribing information for Altuviiio (efanesoctocog alfa) to include final data on a clinical trial that tested the now-approved therapy in children with hemophilia A. Sanofi, which markets Altuviiio in the U.S., submitted a supplemental biologics license…
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