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Sporting a $3.5M price tag, CSL and uniQure's hemophilia B gene therapy crosses FDA finish line

Fierce Pharma

price tag, CSL and uniQure's hemophilia B gene therapy crosses FDA finish line. Sporting a $3.5M Wed, 11/23/2022 - 10:10.

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CSL doses first patient with haemophilia B gene therapy Hemgenix

Pharmaceutical Technology

The high price tag may be hard to defend , but with lifetime treatment costs for haemophilia B reaching as high as $23m for some, the single-dose treatment could prove far more cost-effective. Already a big player in haemophilia treatments, CSL licensed exclusive global rights to Hemgenix from gene therapy developers uniQure back in 2021.