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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.
D12908. Fidanacogene elaparvovec, sold under the brand name Beqvez among others, is a gene therapy delivered via adeno-associated virus used for the treatment of hemophilia B (congenital Factor IX deficiency). [ 1 ][ 5 ][ 8 ] Fidanacogene elaparvovec was approved for medical use in Canada in December 2023, [ 1 ] in the United States in April ...
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 ...
Haemophilia (British English), or hemophilia (American English) [6] (from Ancient Greek αἷμα (haîma) 'blood' and φιλία (philía) 'love of'), [7] is a mostly inherited genetic disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding. [2][3] This results in people bleeding for a longer time after ...
681K1JDI8M. KEGG. D12434. Valoctocogene roxaparvovec, sold under the brand name Roctavian, is a gene therapy used for the treatment of hemophilia A. [1][3] It was developed by BioMarin Pharmaceutical. [4][5][6] Valoctocogene roxaparvovec is made of a virus (AAV5) that has been modified to contain the gene for factor VIII, which is lacking in ...
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