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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 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 ...
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 ...
Giroctocogene fitelparvovec (PF-07055480) is an experimental gene therapy for hemophilia A via a recombinant adeno-associated virus serotype 6-based vector. [1]
Gene therapy is a medical technology that aims to produce a therapeutic effect through ... hemophilia A: August 2022 ... The success of the gene insertion was later ...
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 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.
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 ...
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