Haemophilia one-time gene therapy effective for three years

Blood in a Petri dish

An investigational one-time gene therapy for the treatment of adults with severe haemophilia A has been proven to be effective for more than three years from treatment. 

BioMarin Pharmaceutical revealed the positive results from its ongoing global Phase III GENEr8-1 study of Roctavian (valoctocogene roxaparvovec).  

This is the largest and longest global Phase III study to date for any gene therapy in haemophilia, with 134 participants. 

BioMarin also analysed annualised bleeding rate (ABR) for all bleeds, regardless of whether those bleeds were treated with exogenous Factor VIII replacement. In year three, the mean/median ABR for all bleeds was 1.4/0.0, and in year four the mean/median ABR for all bleeds was 1.6/1.0. 

At the end of year three, 92% of patients remained off prophylaxis. Those patients who returned to Factor VIII or emicizumab prophylaxis did so safely.    

“We continue to learn more about the durability, safety and efficacy of valoctocogene roxaparvovec,” said Steven Pipe, Professor of Pediatrics and Pathology at the University of Michigan and an investigator in the study. “I am encouraged to see the consistent clinical response and the significant number of study participants who remain off prophylaxis after three years. This shows the potential transformative impact of this single treatment event for people with severe haemophilia A.” 

These data will be shared with the FDA as part of the agency’s ongoing review of the Biologics License Application (BLA) of Roctavian. The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec in March 2021. 

Valoctocogene roxaparvovec has also received orphan drug designation from the European Medicines Agency (EMA) and FDA for the treatment of severe haemophilia A, as well as conditional marketing authorisation from the European Commission (EC). 

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