Approved Treatments for Hemophilia
Hemophilia is a blood disorder that is characterized by a shortage of specific clotting factors that help to form blood clots and stop bleeding. The primary treatment for hemophilia is replacement therapy that supplies the clotting factors to hemophilia patients who lack them. Other approved treatments include bypassing agents, gene therapies, and nonfactor replacement therapies.
Bypassing Agents
In some cases, patients develop resistance to treatments — usually by developing antibodies against the clotting factors that are used to treat the disease. In these cases, bypassing agents may be used. Bypassing agents are treatments that “bypass,” or circumvent, the need for clotting factor treatment. They also can be used to treat acquired hemophilia, a rare type of hemophilia in which the body erroneously develops antibodies against its own clotting factors.
FEIBA
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
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
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.
Extended Half-Life Products
Currently available factor replacement therapies are limited by their relatively short half-lives and require intravenous injections several times a week to maintain clotting factor levels high enough to prevent bleeds. Extended half-life products contain versions of clotting factors that have been modified to last longer in the body, allowing these therapies to be administered less frequently.
Albumin Fusion
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
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
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.
Gene Therapy
The objective of gene therapy for hemophilia is to deliver a healthy copy of the mutated gene to cells in the body — particularly in the liver, which is where most clotting factors are produced — to restore the body’s ability to produce its own functional clotting factor. To deliver their genetic cargo, gene therapies commonly use viral vectors, most commonly adeno-associated viruses.
Beqvez
Beqvez (fidanacogene elaparvovec-dzkt) is a gene therapy approved in the U.S. for the treatment of certain adults with moderate to severe hemophilia B. The one-time therapy, given via an into-the-vein or intravenous infusion, is marketed by Pfizer.
Hemgenix
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
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.
Factor replacement therapies
Replacement therapy is the gold standard treatment for hemophilia. It generally involves supplying a version of the missing clotting factor to the patient. The administered clotting factors may be obtained from the blood of healthy donors or created in the lab. Replacement therapies can be used on a prophylactic basis to prevent or reduce the frequency of bleeds, or on an on-demand basis to treat active bleeds.
Advate
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
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
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
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
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
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
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
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
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
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.
Recombinate
Recombinate (antihemophilic factor recombinant) is a recombinant, or lab-made, clotting factor VIII concentrate that is approved to prevent and control bleeds, including those occurring during surgery, in children and adults with hemophilia A. It is administered via an intravenous, or into-the-vein, injection.
Rixubis
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
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
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.
Nonfactor replacement therapies
Nonfactor replacement therapies are medications that promote blood clotting through mechanisms that do not involve replacing the missing clotting factors and may be used to help manage bleeding in hemophilia.
Cyklokapron
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
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
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.
