Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A.
Annette von DrygalskiPratima ChowdaryRoshni KulkarniSophie SusenBarbara A KonkleJohannes OldenburgDavide MatinoRobert KlamrothAngela Christine WeyandVictor Jimenez-YusteKeiji NogamiStacey PoloskeyBent WindingAnnemieke WillemzeKarin Knobenull nullPublished in: The New England journal of medicine (2023)
In patients with severe hemophilia A, once-weekly efanesoctocog alfa provided superior bleeding prevention to prestudy prophylaxis, normal to near-normal factor VIII activity, and improvements in physical health, pain, and joint health. (Funded by Sanofi and Sobi; XTEND-1 ClinicalTrials.gov number, NCT04161495.).