UniQure Needs Longer-Term Data For Hemophilia B Gene Therapy EtranaDez

Company Presents HOPE-B Data At R&D Day

 
• By Alaric DeArment
  
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The FDA may want longer follow-up data for companies developing treatments for hemophilia A as well as hemophilia B.

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uniQure presented an update to data from its Phase III HOPE-B study of etranacogene dezaparvovec in hemophilia B • Source: Shutterstock

The delay in uniQure N.V.’s filing plans for its hemophilia B gene therapy etranacogene dezaparvovec reflects changing requests from the US Food and Drug Administration, which could affect players across the hemophilia A and B space – but does not reflect any negatives for uniQure’s product itself. The latest Phase III data show sustained increases in Factor IX (FIX) activity a year after administration of the therapy.

At its R&D meeting on 22 June, the Amsterdam-headquartered company announced an update from the Phase III HOPE-B trial and...

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