Approved Treatments for Hemophilia

FEIBA (factor eight inhibitor bypassing activity) is a bypassing agent that’s approved to prevent or control bleeding episodes in people with hemophilia A and hemophilia B with inhibitors — neutralizing antibodies against certain clotting factors. It is administered via an intravenous or into-the-vein infusion.

NovoSeven RT (eptacog alfa [activated]) is a bypassing agent that’s approved to treat bleeding episodes, including those occurring during surgery, in adults and children with hemophilia A or B who have developed inhibitors — neutralizing antibodies against certain clotting factors. It’s also approved for adults with acquired hemophilia, for the treatment of bleeding episodes and the prevention of bleeds in surgical interventions or invasive procedures. The therapy is administered via an intravenous or into-the-vein injection.

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 or factor IX. It’s indicated for adults and adolescents, ages 12 and older. It is given as an intravenous or into-the-vein infusion.

Albumin fusion is a molecular technology that has been used to create extended half-life therapies for hemophilia. Generally, it entails fusing a highly stable protein called albumin to clotting factor proteins, in order to slow the rate at which these proteins are degraded in the blood.

Fc fusion is one of the molecular technologies that has been used to create extended half-life replacement therapies for hemophilia. It involves attaching the Fc domain — the tail region of certain antibodies that helps prevent their degradation — to clotting factor proteins, making them more stable and long-lasting in the bloodstream.

PEGylation is one of the molecular technologies that has been used to create extended half-life replacement therapies for hemophilia. It involves attaching a nontoxic compound called polyethylene glycol, or PEG, to clotting factors, making them less likely to be broken down and extending the time they remain in circulation.

Hemgenix (etranacogene dezaparvovec) is a gene therapy that’s approved for the treatment of certain adults with hemophilia B. The one-time therapy, given via an into-the-vein or intravenous infusion, is marketed by CSL Behring.

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 therapy, developed by BioMarin Pharmaceutical, is given via a single into-the-vein, or intravenous, infusion.

Advate (octocog alfa) is a recombinant, or lab-made, clotting factor VIII concentrate used to prevent and control bleeding in children and adults with hemophilia A. It is given by an into-the-vein, or intravenous, injection.

Afstyla (lonoctocog alfa) is a factor replacement therapy used to prevent and treat bleeding episodes, including those occurring during surgery, in people with hemophilia A. It is given by an into-the-vein, or intravenous, injection.

Alphanate (human antihemophilic factor/von Willebrand factor complex) is a factor replacement therapy to prevent and control bleeds, including those occurring during surgery, in children and adults with hemophilia A. It is given via an into-the-vein, or intravenous, injection.

AlphaNine SD (human coagulation factor IX) is a highly purified, plasma-derived clotting factor IX concentrate that’s approved to prevent and control bleeding in people with hemophilia B. The therapy is given via an infusion directly into the bloodstream.

BeneFIX (nonacog alfa) is a recombinant, or man-made, clotting factor therapy approved to prevent and treat bleeding episodes, including those occurring during surgery, in adults and children with hemophilia B. The therapy is administered via an into-the-vein, or intravenous, injection.

Ixinity (trenonacog alfa) is an approved recombinant, or lab-made, replacement therapy designed to prevent or treat bleeding episodes in hemophilia B patients of all ages. It is administered via an infusion into the vein, or intravenously.

Kovaltry (octocog alfa) is a recombinant, or man-made, clotting factor therapy that is approved to prevent and treat bleeding episodes, including those occurring during surgery, in adults and children with hemophilia A. It is administered via an into-the-vein, or intravenous, infusion.

Novoeight (turoctocog alfa) is a recombinant, or man-made, factor replacement therapy that’s approved to prevent and treat bleeds, including those occurring during surgery, in people with hemophilia A. It is administered via an intravenous, or into-the-vein, injection.

Nuwiq (simoctocog alfa) is a recombinant, or lab-made, clotting factor therapy that is approved to prevent and control bleeding episodes, including those occurring during surgery, in adults and children with hemophilia A. It is administered via an into-the-vein, or intravenous, injection.

Obizur (susoctocog alfa) is a recombinant, or man-made, antihemophilic factor therapy approved for the on-demand treatment of bleeds in adults with acquired hemophilia A. The therapy is given via an into-the-vein, or intravenous, injection.

Rixubis (nonacog gamma) is a recombinant, or man-made, replacement therapy that’s approved to prevent and treat bleeds, including those occurring during surgery, in adults and children with hemophilia B. It is administered via an into-the-vein, or intravenous, infusion.

Wilate (human von Willebrand factor/coagulation factor VIII complex) is a plasma-derived replacement therapy that’s approved to prevent and control bleeds in adults and adolescents with hemophilia A. It is given via an into-the-vein, or intravenous, injection.

Xyntha (moroctocog alfa) is a recombinant, or man-made, clotting factor replacement therapy approved to prevent and treat bleeds, including those occurring during surgery, in people with hemophilia A. The therapy is administered via an intravenous, or into-the-vein, injection.

Alhemo (concizumab-mtci) is an antibody-based therapy 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.

Cyklokapron (tranexamic acid) is an antifibrinolytic agent that’s designed to control or prevent bleeding episodes in hemophilia patients, and reduce the need for replacement therapy during and following tooth extraction. It is administered as an intravenous, or into-the-vein, injection.

Hemlibra (emicizumab-KXWH) is an antibody-based therapy that is approved to prevent or reduce bleeds in hemophilia A patients, with or without factor VIII inhibitors. It is given as an under-the-skin, or subcutaneous, injection.

Hympavzi (marstacimab-hncq) is an antibody-based therapy that is approved to prevent or reduce the frequency of bleeds in patients with hemophilia A or B, without inhibitors. It is given as weekly subcutaneous, or under-the-skin, 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.