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If approved, Pfizer's hemophilia A gene therapy will compete with BioMarin Pharmaceutical's one-time treatment Roctavian, which was approved in the U.S. last year and is priced at $2.9 million.
The companies expect the gene therapy to serve as a one-time shot to cut the rate of annual bleeding for hemophilia B patients, who represent about 15% of all patients with hemophilia.
Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. [ 5 ] [ 6 ] [ 7 ] Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX . [ 7 ]
The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...
Monoclonal antibody emicizumab has been approved by the FDA in 2017 for therapy of hemophilia A. [28] In July 2024, a recent study published in the New England Journal of Medicine demonstrated that efanesoctocog alfa , a bioengineered human factor VIII recombinant protein, prophylaxis for children with severe hemophilia A could have therapeutic ...
Protein kinase RNA-activated also known as protein kinase R (PKR), interferon-induced, double-stranded RNA-activated protein kinase, or eukaryotic translation initiation factor 2-alpha kinase 2 (EIF2AK2) is an enzyme that in humans is encoded by the EIF2AK2 gene on chromosome 2. [5] [6] PKR is a serine/tyrosine kinase that is 551 amino acids ...
The price is less than the $3.5 million announced last year for a similar gene therapy for hemophilia B, a less common form of the disease. Like most medicines in the U.S., the new treatment will ...
In human, the F9 gene is located on the X chromosome at position q27.1. Because the gene for factor IX is located on the X chromosome (Xq27.1-q27.2), loss-of-function mutations thereof are X-linked recessive : males experience the disease phenotype much more frequently than females.
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