ASH Data Bring UniQure Closer To Filing Hemophilia B Gene Therapy
Late-Breaking Data Suggest Activity At Level Needed For Approval
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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BioMarin’s confidence that its first-ever hemophilia gene therapy would be cleared by the US FDA was shaken by a complete response letter that the company claims changed the requirements for approval.
Observers have been expecting uniQure to be sold off as a whole but the Netherlands-based firm has chosen to sell just one asset - EtranaDez - and focus on advancing its Huntington’s disease gene therapy.
The company has come a long way since gene therapy flop Glybera, and claims best-in-class status for viral vector.