ICER Final Evidence Report on gene therapies for hemophilia A and B

22 December 2022

US health technology assessor the Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative clinical effectiveness and value of two gene therapies intended for the treatment of hemophilia A and B.

One of these was for CSL Behring, part of Australia's CSL Limited (ASX: CSL), for its one-time hemophilia B treatment Hemgenix (etranacogene dezaparvovec-drlb). The US Food and Drug Administration (FDA) granted approval for the treatment of certain adults with hemophilia B last month, and it will be most expensive medicine in the world when it hits the US market, with an anticipated list price of around $3.5 million.

The other is for California, USA-based BioMarin Pharmaceutical’s (Nasdaq: BMRN) Roctavian (valoctocogene roxaparvovec AAV gene therapy) for adults with severe hemophilia A, which has yet to gain FDA approval, although this was granted approval in Europe in August. BioMarin revealed that the price of Roctavian per patient will be 1.5 million euros ($1.5 million) net of all discounts and reserves in the European Union.

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