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ICER evaluates Roctavian and EtranaDez gene therapy for hemophilia A and B

 Last Update: 2022-08-12
 Source: Internet
 Author: User

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Hemophilia is mostly a hereditary disease that prevents the body from clotting and forming a blood clot to stop bleedi.

The Institute for Clinical and Economic Review (ICER) announced that it will evaluate the clinical efficacy and value of EtranaDez (etranacogene dezaparvovec) in the treatment of hemophili.

EtranaDez is an AAV5-mediated gene therapy that delivers the Padua variant of the factor IX gene to liver cells, resulting in an active variant of factor .

The results showed that the annualized bleeding rate (ABR) assessed 18 months after infusion decreased to 51, compared with 19 in the run-in period, meeting the primary noninferiority endpoint as well as secondary endpoin.

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