Sanofi and Swedish Orphan Biovitrum’s (Sobi) Biologics Licence Application (BLA) for efanesoctocog alfa (BIVV001) for the treatment of haemophilia A has been accepted by the US Food and Drug Administration (FDA) for priority review.
The application is supported by data from the pivotal XTEND-1 phase 3 study, which showed adults and adolescents 12 years and older treated once-weekly with efanesoctocog alfa experienced clinically meaningful prevention of bleeds and superiority to prior factor prophylaxis based on an intra-patient comparison.
Moreover, efanesoctocog alfa was well-tolerated, and inhibitor development to factor VIII was not detected.
People living with haemophilia A lack sufficient functioning factor VIII protein to help their blood clot. The condition occurs in around one in 5,000 male births annually, and more rarely in females. People with haemophilia can experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening haemorrhages.
Efanesoctocog alfa is a novel and investigational recombinant factor VIII therapy designed to extend protection from bleeds with once-weekly dosing.
The treatment was granted Breakthrough Therapy Designation by the FDA in May 2022, having received Fast Track designation in February 2021 and Orphan Drug designation in August 2017. The European Commission (EC) also granted efanesoctocog alfa Orphan drug designation in June 2019.
Sanofi outlined that regulatory submission in the EU will follow the availability of data from the ongoing XTEND-Kids paediatric study, with both events expected in 2023.
Commenting on the FDA’s decision, Steve Pipe, professor and director of Pediatric Hemophilia and Coagulation Disorders Program, University of Michigan, said: “Factor therapy remains a cornerstone of haemophilia treatment, but innovation has been needed in this area to address challenges related to bleed protection and cumbersome treatment regimens.
If approved, efanesoctocog alfa can deliver close to normal factor activity levels for the majority of the week, potentially offering a new tier of protection. Such therapeutic benefits would represent important advances in unmet medical needs for people with haemophilia A and may transform the prophylactic treatment landscape.”
Earlier this month, the EC granted BioMarin Pharmaceutical’s Roctavian (valoctocogene roxaparvovec) gene therapy a conditional marketing authorisation for the treatment of severe haemophilia A. The authorisation is specifically for the treatment of adult patients without a history of factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).