Not Such A Sure Thing: FDA Knocks Back BioMarin’s Roctavian
Company’s Hemophilia A Gene Therapy Gets A CRL
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.
You may also be interested in...
A patient developed Factor VIII inhibitors, but he had also recently received a COVID-19 vaccine and was genetically predisposed.
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.