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ASH Data Bring UniQure Closer To Filing Hemophilia B Gene Therapy

Late-Breaking Data Suggest Activity At Level Needed For Approval

 
• By Alaric DeArment

The company plans to file with 52-week data for AMT-061 and is confident it won't face the same stringent requirements from the FDA that led to a complete response letter for BioMarin's Roctavian.

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UniQure will present topline Phase III data at ASH • Source: Shutterstock

uniQure N.V. believes that 52-week data for its hemophilia B gene therapy will be enough to support US Food and Drug Administration approval, despite the agency’s recent rejection of another firm’s hemophilia A gene therapy without longer-term data, and that the 26-week data being presented at the American Society of Hematology annual meeting indicate efficacy will be in the range necessary for approval.

At ASH’s virtual meeting, the Netherlands-based biotech will present topline data from the Phase III HOPE-B study AMT-061 (etranacogene dezaparvovec)...

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