Efanesoctocog alfa met primary and key secondary endpoints in pivotal study in haemophilia A, showing superiority to prior factor prophylaxis treatment- SOBI + Sanofi
Swedish Orphan Biovitrum AB (publ) (Sobi) and Sanofi announced positive top-line results from the pivotal XTEND-1 phase III study evaluating the safety, efficacy and pharmacokinetics of efanesoctocog alfa (BIVV001) in previously treated patients greater than 12 years of age with severe haemophilia A.
The study met the primary endpoint, showing a clinically meaningful prevention of bleeds in people with severe haemophilia A receiving weekly prophylaxis with efanesoctocog alfa over a period of 52 weeks. The median annualised bleeding rate (ABR) was 0 with a mean ABR of 0.71. The key secondary endpoint was also met, demonstrating once-weekly efanesoctocog alfa was superior to prior prophylactic factor VIII replacement therapy, showing a statistically significant reduction in ABR based on intra-patient comparison. Efanesoctocog alfa was well-tolerated, and inhibitor development to factor VIII was not detected. The most common treatment-emergent adverse events ( greater than 5 per cent of participants overall) were headache, arthralgia, fall, and back pain.
The data will be the basis for submission to regulatory authorities around the globe beginning this year. Submission in the EU will follow availability of data from the ongoing XTEND-Kids paediatric study, expected in 2023. Efanesoctocog alfa was granted Orphan Drug Designation by the FDA in August 2017 and the European Commission in June 2019. The FDA granted Fast Track Designation in February 2021. Efanesoctocog alfa is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
Related news and insights
Palisade Bio, Inc. announced the first patient has been dosed in its Phase III study evaluating LB 1148 to accelerate the return of bowel function in adult patients undergoing gastrointestinal surgery.
bluebird bio, Inc. announced the FDA has approved Zynteglo (betibeglogene autotemcel), also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying genetic cause of beta-thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.
Edwards Lifesciences Corporation announced the company's PASCAL Precision transcatheter valve repair system received CE Mark for the treatment of mitral and tricuspid regurgitation (MR and TR).