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UniQure/CSL Set To Launch First Hemophilia B Gene Therapy Next Year At Record Price

With $3.5m Price Tag

 
• By Ayisha Sharma

The firms’ gene therapy Hemgenix has won a US thumbs up for the treatment of hemophilia B and plans are underway for a US launch with a big price tag that experts agree should pay off in the long-term.  

Hands woman hold drop of blood on blue background.
Hemophilia B Is A Rare Clotting Disorder That Mostly Affects Males • Source: Shutterstock

uniQure N.V./CSL Limited’s Hemgenix (tranacogene dezaparvovec-drlb) has been approved for the treatment of adults with moderately severe to severe hemophilia B by the US Food and Drug Administration, making it the first gene therapy for the rare bleeding disorder and the most expensive drug to date.

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