BioMarin initially withdrew the EU marketing application for the gene therapy – also known as Roctavian – last year, after stating that it was unable to provide the data needed to resolve a ‘major objection’ raised by the EMA’s Committee for Advanced Therapies.
The first EMA filing contained interim results from 32 adults with severe haemophilia A in a Phase III trial.
According to BioMarin, the EMA had requested the full 52-week results from all 134 patients enrolled in the study.
