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. Historically, the primary treatment for hemophilia is clotting factor 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.
Factor replacement therapies
Historically, factor replacement therapy has been the gold standard for hemophilia treatment. It generally involves supplying a version of the missing clotting factor to the patient. The administered clotting factors may be plasma-derived (obtained from the blood of healthy donors) or created in the lab (recombinant). Recombinant therapies can be further subdivided into standard or extended half-life therapies, depending on the amount of time they remain in circulation in the body.
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. All these therapies are administered intravenously, or directly into the bloodstream.
Advate
Advate (octocog alfa) is a recombinant factor VIII concentrate used to prevent and control bleeding in children and adults with hemophilia A.
Adynovate
Adynovate (rurioctocog alfa pegol) is a recombinant factor VIII concentrate approved to prevent and treat bleeding episodes, including those occurring during surgery, in children and adults with hemophilia A.
Afstyla
Afstyla (lonoctocog alfa) is a recombinant factor VIII concentrate used to prevent and treat bleeding episodes, including those occurring during surgery, in children and adults with hemophilia A.
Alphanate
Alphanate (human antihemophilic factor/von Willebrand factor complex) is a plasma-derived factor replacement therapy to prevent and control bleeds, including those occurring during surgery, in children and adults with hemophilia A.
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.
Alprolix
Alprolix (eftrenonacog alfa) is a recombinant factor IX concentrate used to prevent and treat bleeds, including those occurring during surgery, in adults and children with hemophilia B.
Altuviiio
Altuviiio (efanesoctocog alfa) is a recombinant factor VIII concentrate used to prevent and treat bleeding episodes, including those occurring during surgery, in children and adults with hemophilia A.
BeneFIX
BeneFIX (nonacog alfa) is a recombinant clotting factor therapy approved to prevent and treat bleeding episodes, including those occurring during surgery, in adults and children with hemophilia B.
Eloctate
Eloctate (efmoroctocog alfa) is an extended half-life recombinant therapy approved to prevent and treat bleeds, including those occurring during surgery, in adults and children with hemophilia A.
Esperoct
Esperoct (turoctocog alfa pegol) is a recombinant factor VIII concentrate indicated to prevent and treat bleeding episodes, including those occurring during surgery, in children and adults with hemophilia A.
Idelvion
Idelvion (albutrepenonacog alfa) is a recombinant factor IX concentrate approved to prevent and treat bleeds, including those occurring during surgery, in children and adults with hemophilia B.
Ixinity
Ixinity (trenonacog alfa) is an approved recombinant replacement therapy designed to prevent or treat bleeding episodes in hemophilia B patients of all ages.
Jivi
Jivi (damoctocog alfa pegol) is a recombinant factor VIII concentrate approved to prevent and treat bleeding episodes, including those occurring during surgery, in previously treated adults and children ages 7 and older with hemophilia A.
Kovaltry
Kovaltry (octocog alfa) is a recombinant clotting factor therapy approved to prevent and treat bleeding episodes, including those occurring during surgery, in adults and children with hemophilia A.
Novoeight
Novoeight (turoctocog alfa) is a recombinant factor replacement therapy that’s approved to prevent and treat bleeds, including those occurring during surgery, in people with hemophilia A.
Nuwiq
Nuwiq (simoctocog alfa) is a recombinant clotting factor therapy that is approved to prevent and control bleeding episodes, including those occurring during surgery, in adults and children with hemophilia A.
Obizur
Obizur (susoctocog alfa) is a recombinant porcine (pig-derived) factor VIII concentrate approved for the on-demand treatment of bleeds in adults with acquired hemophilia A.
Rebinyn
Rebinyn (nonacog beta pegol) is a recombinant factor IX concentrate used to prevent and treat bleeds, including those occurring during surgery, in children and adults with hemophilia B.
Rixubis
Rixubis (nonacog gamma) is a recombinant replacement therapy approved to prevent and treat bleeds, including those occurring during surgery, in adults and children with hemophilia B.
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.
Xyntha
Xyntha (moroctocog alfa) is a recombinant clotting factor replacement therapy approved to prevent and treat bleeds, including those occurring during surgery, in people with hemophilia A.
Nonfactor therapies
Nonfactor 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. All these therapies are administered via subcutaneous, or under-the-skin, injections.
Alhemo
Alhemo (concizumab-mtci) is an antibody-based therapy approved to prevent or reduce the frequency of bleeds in patients with hemophilia A or B, ages 12 and older, with or without inhibitors. It is given once daily and can be self-administered.
Hemlibra
Hemlibra (emicizumab-kxwh) is an antibody-based therapy that is approved to prevent or reduce bleeds in children and adults with hemophilia A, with or without factor VIII inhibitors. It is given once weekly for the first month and every one, two, or four weeks thereafter.
Hympavzi
Hympavzi (marstacimab-hncq) is an antibody-based therapy approved to prevent or reduce the frequency of bleeds in people with hemophilia A or B, ages 12 and older, without inhibitors. It is given once weekly and can be self-administered.
Qfitlia
Qfitlia (fitusiran) is an RNA-based therapy that is approved to prevent or reduce the frequency of bleeds in people with hemophilia A or B, ages 12 and older, with or without inhibitors. It is given as infrequently as once every two months and can be self-administered.
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 called inhibitors. In these cases, bypassing agents may be used. Bypassing agents are treatments that bypass, or circumvent, the need for conventional clotting factor replacement 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. Bypassing agents are administered intravenously.
FEIBA
FEIBA (factor eight inhibitor bypassing activity) is a plasma-derived bypassing agent that’s approved to prevent or control bleeding episodes in people with hemophilia A and hemophilia B with inhibitors.
NovoSeven RT
NovoSeven RT (eptacog alfa [activated]) is a recombinant bypassing agent that’s approved to treat bleeding episodes, including those occurring during surgery, in adults and children with hemophilia A or B with inhibitors. It’s also approved for the same indication in adults with acquired hemophilia.
Sevenfact
Sevenfact (eptacog beta) is a recombinant bypassing agent that’s approved to treat bleeding episodes in people with hemophilia A or B with inhibitors. It’s indicated for adults and adolescents, ages 12 and older.
Gene Therapy
The objective of gene therapy for hemophilia is to deliver a healthy copy of the gene encoding the missing or dysfunctional clotting factor to cells in the body — particularly in the liver, 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.
Hemgenix
Hemgenix (etranacogene dezaparvovec-drlb) is a gene therapy that’s approved for the treatment of certain adults with hemophilia B. The one-time therapy, given via 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 intravenous infusion.
Adjunctive and supportive therapies
There are also treatments that may be used as an add-on to other therapies or in specific circumstances to help manage bleeding episodes for shorter periods of time in people with hemophilia.
Aminocaproic acid
Aminocaproic acid is an antifibrinolytic medication that may be used off-label in some situations to help control bleeding in hemophilia patients, including to manage bleeding in the mouth, such as during tooth extractions. It is available as oral tablets or an oral solution.
Cyklokapron
Cyklokapron (tranexamic acid) is an antifibrinolytic agent that is indicated to be used as a short-term treatment to reduce or prevent bleeds and to lower the need for replacement therapy during and after tooth extraction. It is administered as an intravenous injection.
