In a CRL issued for valoctocogene roxaparvovec, the FDA told BioMarin that differences between its Phase I/II and Phase III studies limited the ability to rely on the earlier trial to support durability of effect, which an analyst said likely referred to a product comparability issue.
The biotech company said the FDA had given priority review to valoctocogene roxaparvovec for hemophilia A in adults, with the PDUFA date set at Aug. 21.
By reducing administrative burden and redesigning workflows around human needs, it creates space for what matters most: connection between clinicians and patients.
An analyst wrote that while the Phase III data - released alongside Phase I/II data - for valrox could be enough for accelerated FDA approval and appeared to allay concerns about declining Factor VIII levels, it could face commercial challenges.