Read about Esperoct, an extended half-life infusion treatment approved to prevent or stop bleeding in children and adults with hemophilia A.
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The U.S. Food and Drug Administration (FDA) has approved Expression Therapeutics‘ request to open a Phase 1 trial of ET3, an investigational gene therapy for people with hemophilia A. The decision followed a review of the company’s investigational new drug application (IND) requesting clearance of ET3…
Hemlibra (emicizumab-KXWH) is an antibody-based therapy used to prevent or reduce bleeds in hemophilia A patients, with or without factor VIII (FVIII) inhibitors. It is given as an under-the-skin, or subcutaneous, injection.
Pfizer’s Hympavzi (marstacimab) has been approved in the European Union (EU) to prevent or reduce the frequency of bleeding episodes in adults and children, ages 12 and older, with severe hemophilia A or hemophilia B who weigh at least 35 kg (77 pounds) and haven’t developed neutralizing…
Alhemo (concizumab-mtci) is an antibody-based therapy that is approved to prevent or reduce the frequency of bleeds in patients with hemophilia A or B, with inhibitors. It is given as daily under-the-skin, or subcutaneous, injections that can be self-administered.
Qfitlia (fitusiran) is an RNA-based therapy that is approved to prevent or reduce the frequency of bleeds in patients with hemophilia A or B, with or without inhibitors. It is given via under-the-skin, or subcutaneous, injections that can be self-administered.
Most people with hemophilia A on preventive treatment with Hemlibra (emicizumab) will experience spontaneous or traumatic bleeds at some point, according to a study using real-world data from Israel. Results also indicated the risk of spontaneous bleeds while on Hemlibra is higher for older individuals. “Our findings indicate that…
Sevenfact (eptacog beta) is a bypassing agent that’s approved to treat bleeding episodes in people with hemophilia A or hemophilia B who developed inhibitors, or neutralizing antibodies, against clotting factor VIII (FVIII) or factor IX (FIX).
BioMarin Pharmaceutical  will add an additional Phase 3 study to the clinical development of its investigational gene therapy BMN 270 for hemophilia A. BioMarin will conduct two separate trials testing the effectiveness and safety of two different doses of BMN 270. Both trials are expected to initiate in the…
Swedish Orphan Biovitrum AB recently announced that Eloctate (efmoroctocog alfa; brand name Elocta in Europe), a treatment for hemophilia A, has been approved for reimbursement in the United Kingdom, Italy, and France. These countries join others in the European Union — Germany, Sweden, Denmark, Norway, Switzerland, the Netherlands,…