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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.
It priced the one-time therapy, Roctavian, at $2.9 million. Pivotal trial results showed that Roctavian reduced bleeding events, but its durability is not known, and the company said it would ...
Giroctocogene fitelparvovec (PF-07055480) is an experimental gene therapy for hemophilia A via a recombinant adeno-associated virus serotype 6-based vector. [1]
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 ...
ASC618 (also known as AAV2/8 HCB-ET3-LCO BDD FVIII) is an experimental gene therapy for Hemophilia A. It was developed by Applied StemCell Therapeutics and is delivered via a hybrid of adeno-associated virus types 2 and 8. The gene therapy is hoped to be more effective than earlier gene therapies for hemophilia A. [1] [2] [3]
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]
(Reuters) -High cost, logistical issues and the prospect of potential treatment advances are holding back adoption of the first gene therapies for hemophilia, experts said this week during the ...
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 ...
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