Sanofi recently announced newly published clinical trial data for efanesoctocog alfa, the company’s investigational recombinant factor VIII therapy (rFVIII) for the prevention of bleeding episodes in hemophilia A patients via once weekly prophylactic intravenous infusions. Efanesoctocog alfa is manufactured through proprietary Fc fusion technologies to extend the half-life of the rFVIII, resulting in more sustained protection from bleeds per infusion. In contrast to other FVIII replacement therapies, it is independent of von Willebrand factor.
The data, which were published January 27th online in The New England Journal of Medicine (NEJM), is based on the XTEND-1 clinical trial program. It was designed to evaluate the safety, efficacy, and pharmacokinetics of efanesoctocog alfa in previously treated adults and adolescents with hemophilia A, who are 12 years of age and older.
According to a new company press release, the phase 3 trial demonstrated “clinically meaningful prevention of bleeds and superior bleed protection” compared to prior factor VIII prophylaxis based on an intra-patient comparison. The median and mean annualized bleeding rates (ABR) were 0.00 and 0.71, respectively. The results also showed a statistically significant and clinically meaningful reduction in ABR (77%) versus prior factor VIII prophylaxis.
“We are excited about the potential for efanesoctocog alfa to address unmet needs by allowing people living with hemophilia to enjoy an active lifestyle. Currently, they often need to make trade-offs between bleed protection and dosing frequency. Based on the XTEND-1 study results assessing efanesoctocog alfa, we have the opportunity to provide near normal factor activity levels for an extended period of time (the majority of a week) with a single dose, which is a first for hemophilia A,” stated Angela Weyand, MD. Weyand is an investigator in the XTEND-1 trial, and Associate Professor at Michigan Medicine. “The data show that efanesoctocog alfa can offer patients increased bleed protection, leading to improved outcomes, such as reduced pain and improved physical functioning, that may impact daily life with a reduced treatment burden.”
Efanesoctocog alfa is currently under priority review by the United States Food and Drug Administration (FDA) and the target action date for the decision is February 28, 2023.
Source: Sanofi press release dated January 25, 2023