Signs Of Long-Term Efficacy Boost UniQure’s Hem B Gene Therapy
Will Await Main Study’s One-Year Milestone Before Submission
Unveiling its latest data, UniQure is tipped to avoid the knockback which affected BioMarin’s hemophilia A gene therapy, with a filing expected in the second half of 2021.
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The FDA may want longer follow-up data for companies developing treatments for hemophilia A as well as hemophilia B.
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.
The gene therapy EtranaDez for hemophilia B placed on clinical hold after a patient was diagnosed with liver cancer, but UniQure points to that patient’s extensive history of liver disease.