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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 is expressed in the liver to produce ...
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 ...
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 ...
Earlier this year, FDA approved Pfizer’s Beqvez (fidanacogene elaparvovec-dzkt) for moderate to severe hemophilia B in adult patients who currently use factor IX (FIX) prophylaxis therapy or ...
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 ...
In November 2022, the first gene therapy treatment for Hemophilia B was approved by the U.S. Food and Drug Administration, called Hemgenix. It is a single-dose treatment that gives the patient the genetic information required to produce Factor IX. [90] In June 2023, the FDA approved the first gene therapy treatment for Hemophilia A, called ...
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