Efanesoctocog alfa (Altuviiio)

Diseases for which it is used

• Congenital hemophilia A

Preliminary data

1. XTEND-1: von Drygalski A, Chowdary P, Kulkarni R, Susen S, Konkle BA, Oldenburg J, Matino D, Klamroth R, Weyand AC, Jimenez-Yuste V, Nogami K, Poloskey S, Winding B, Willemze A, Knobe K; XTEND-1 Trial Group. Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A. N Engl J Med. 2023 Jan 26;388(4):310-318. link to original article PubMed NCT04161495
2. XTEND-Kids: Malec L, Peyvandi F, Chan AKC, Königs C, Zulfikar B, Yuan H, Simpson M, Álvarez Román MT, Carcao M, Staber JM, Dunn AL, Chou SC, d'Oiron R, Albisetti M, Demissie M, Santagostino E, Yarramaneni A, Wong N, Abad-Franch L, Gunawardena S, Fijnvandraat K; XTEND-Kids Trial Group. Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A. N Engl J Med. 2024 Jul 18;391(3):235-246. link to original article PubMed NCT04759131

History of changes in FDA indication

• 2023-02-22: Initial approval for use in adults and children with Hemophilia A (congenital Factor VIII deficiency) for: (1) Routine prophylaxis to reduce the frequency of bleeding episodes; (2) On-demand treatment and control of bleeding episodes; and (3) Perioperative management of bleeding. (Based on XTEND-1)
• 2024-05-10: Approval updated to include additional evidence. (Based on XTEND-Kids)

Also known as

• Code name: BIVV-001
• Brand name: Altuviiio