BioMarin has resubmitted a marketing authorisation application (MAA) for its haemophilia A gene therapy valoctocogene roxaparvovec to the European Medicines Agency (EMA), the company announced yesterday.
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.
Now, BioMarin has that data in hand – included in the new MAA is safety and efficacy data from the 134 patients enrolled in the study, all of whom have been followed for at least one year post-treatment with valoctocogene roxaparvovec.
“This is an important step to deliver the potential first gene therapy for people with haemophilia A. The data package for this submission includes the largest Phase III study in any gene therapy for haemophilia A supported by up to four years of observation in a Phase I/II study,” said Hank Fuchs, president, worldwide research and development at BioMarin.
“We look forward to the EMA reviewing the robust data set. We believe that valoctocogene roxaparvovec represents a scientific innovation that may become an important treatment choice for those people who have unmet medical needs,” he added.
BioMarin also announced that it is planning to submit two-year follow-up safety and efficacy data on all participants from the Phase III trial to support the benefit/risk profile of the gene therapy, which was previously requested by the US Food and Drug Administration (FDA).
Last August, BioMarin received a complete response letter from the FDA for valoctocogene roxaparvovec.
At the time, the FDA recommended that BioMarin complete the Phase III study and submit two-year follow-up safety and efficacy data on all study participants.