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Marstacimab, sold under the brand name Hympavzi, is a monoclonal antibody medication used for the treatment of hemophilia A and hemophilia B. [1] [4] [2] It is a tissue factor pathway inhibitor (TFPI) antagonist. [4] It was developed by Pfizer. [5]
Factor VIII is used in haemophilia A and factor IX in haemophilia B. Factor replacement can be either isolated from human plasma, recombinant, or a combination of the two. Some people develop antibodies (inhibitors) against the replacement factors given to them, so the amount of the factor has to be increased or non-human replacement products ...
Coagulation factor VIII (Factor VIII, FVIII, also known as anti-hemophilic factor (AHF)) is an essential blood clotting protein. In humans, it is encoded by F8 gene . [ 5 ] [ 6 ] Defects in this gene result in hemophilia A , an X-linked bleeding disorder .
Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (FVIII in hemophilia A, FIX in hemophilia B), impacting more than 800,000 people globally.
Factors VII, IX, and X all play key roles in blood coagulation and also share a common domain architecture. [10] The factor IX protein is composed of four protein domains: the Gla domain, two tandem copies of the EGF domain and a C-terminal trypsin-like peptidase domain which carries out the catalytic cleavage.
The cost of Factor VIII and similar clotting factors has been described as "highly expensive". [20] The cost of the clotting factors is 80% of all medical costs for people with hemophilia. [22] They are so expensive that gene therapy for haemophilia might be less expensive, especially for people with severe hemophilia. [22]
Haemophilia B, also spelled hemophilia B, is a blood clotting disorder causing easy bruising and bleeding due to an inherited mutation of the gene for factor IX, and resulting in a deficiency of factor IX.
As of 2012, recombinant factor VIIa is not supported by the evidence for treating most cases of major bleeding. [11] There is a significant risk of arterial thrombosis with its use and thus, other than in those with factor VII deficiency or acquired hemophilia, it should only be given in clinical trials. [11]
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