Data demonstrates that efanesoctocog alfa provides superior bleed protection
Sanofi and Swedish Orphan Biovitrum AB (Sobi) have presented positive results from the XTEND-1 pivotal phase 3 study evaluating the safety, efficacy and pharmacokinetics of efanesoctocog alfa – also known as BIVV001.
The treatment is an investigational factor 8 replacement therapy which is used on previously treated adults and adolescents older than 12 years with severe haemophilia A.
The study met the primary efficacy endpoint, with once-weekly efanesoctocog alfa prophylaxis providing clinically meaningful bleed protection for people with severe haemophilia. It also met the key secondary endpoint, demonstrating superior bleed protection over prior factor 8 prophylaxis.
Meanwhile, data demonstrated that adults and adolescents treated with once-weekly efanesoctocog alfa experienced statistically significant and clinically meaningful improvements in physical health, pain intensity and joint health when comparing week 52 and baseline measurements.
Furthermore, efanesoctocog alfa was effective at treating bleeds – including in target joints – with 96.7% were resolved with a single 50 IU/kg dose. The therapy was generally well tolerated with the most common adverse events being headaches, arthralgia and back pain.
Dietmar Berger, global head of Development and chief medical officer at Sanofi, was optimistic about the results: “We are committed to advancing innovative medicines that disrupt the status-quo and address the unmet needs that persist for people with rare conditions like haemophilia.”
“This robust data illustrates the promise of efanesoctocog alfa’s efficacy with once-weekly dosing and underscore its potential as a therapy with best-in-disease efficacy,” he added.
Anders Ullman, Head of R&D and Chief Medical Officer at Sobi, concluded: “We believe transforming the treatment paradigm for haemophilia A can only be achieved through elevating standards of care towards normal haemostasis. This data demonstrates the profile of efanesoctocog alfa in significant clinical terms and further strengthen its potential to ultimately improve the lives of many living with this condition.”
