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Relief For UniQure Hemophilia Program Sends Ball To FTC’s Court

Good News For Gene Therapy Continues As FDA Also Removes Hold On Voyager’s Huntington’s Trial

 
• By Alaric DeArment

While anticipated, the removal of the hold on uniQure’s hemophilia B program removes significant overhang, as the focus shifts to an anticipated FTC antitrust clearance of its CSL Behring deal.

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FDA lifts hold on uniQure's hemophilia B program • Source: Shutterstock

uniQure N.V. is back in the hemophilia B gene therapy game as the US Food and Drug Administration has lifted a hold on the clinical development program for AMT-061 (etranacogene dezaparvovec) following a determination that an incident of liver cancer was unrelated to the therapy. Although anticipated, the hold had caused some nervousness among investors, both for the company and for gene therapy broadly. The focus now shifts to the Federal Trade Commission ruling on a licensing deal for the therapy between the company and CSL Limited.

Amsterdam-based uniQure said 26 April that the FDA had removed the hold after an investigation satisfactorily addressed concerns about a patient in its Phase III HOPE-B clinical trial of the gene therapy,

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