Chugai Pharmaceutical recently published data of a Phase I clinical study on the antibody emicizumab. The study, “Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A,” was published in The New England Journal of Medicine. The study revealed possible benefits from emicizumab for the prevention of bleeding in…
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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…
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).
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,…
Up to 14 months after a one-time infusion of SB-525, an investigational gene therapy, durable increases in the activity of clotting factor VIII (FVIII) are continuing in all five severe hemophilia A patients treated at the highest dose in the Alta Phase 1/2 trial. None of these…
Jim Christensen Jr., a hemophilia A patient who has supported the National Hemophilia Foundation (NHF) for more than three decades, has received the Lester A. Rosen Humanitarian and Achievement Award, given annually by Ameritas, for which he is an independent financial representative. With the award, the insurance company…