Sporting a $3.5M price tag, CSL and uniQure's hemophilia B gene therapy crosses FDA finish line
Fierce Pharma
NOVEMBER 23, 2022
price tag, CSL and uniQure's hemophilia B gene therapy crosses FDA finish line. Sporting a $3.5M Wed, 11/23/2022 - 10:10.
tag uniqure 
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First gene therapy for haemophilia B gets go ahead in Europe
Drug Discovery World
DECEMBER 20, 2022
The clinical development of etranacogene dezaparvovec was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialise the treatment. . Thank you to all for the role you have played in helping us reach this regulatory milestone in Europe.” . World’s most expensive drug.
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Zynteglo halt re-ignites viral vector safety concerns; analysts
pharmaphorum
FEBRUARY 22, 2021
The case comes just weeks after UniQure halted a trial of its haemophilia B gene therapy AMT-061 after an unexpected case of liver cancer. News of the pause came as UK cost-effectiveness watchdog NICE rejected Zynteglo for regular NHS use in first draft guidance, despite a confidential discount to its price tag of around €1.57
No sign of stopping: The rise of cell and gene therapy
Drug Discovery World
MAY 22, 2023
The cost 2022 has not been shy of approvals – Uniqure and CSL’s Haemophilia B drug even nabbed the title of “Most Expensive Drug” at $3.5 But are these price tags a barrier to access? million per dose. Simply, yes, as we saw when the European Commission rejected the price point for bluebird bio’s Zynteglo.
CSL doses first patient with haemophilia B gene therapy Hemgenix
Pharmaceutical Technology
JUNE 21, 2023
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.
Hemgenix Approved as First Gene Therapy for Hemophilia B
XTalks
NOVEMBER 24, 2022
The Hemgenix approval was granted to CSL Behring , a rare disease biotech company based in Pennsylvania, which developed Hemgenix jointly with gene therapy biotech uniQure. CSL Behring is set to market the drug after having secured global commercialization rights from uniQure for $450 million in 2020.
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