Preferred Label : Efanesoctocog Alfa;
NCIt definition : A recombinant fusion protein comprising a single chain B-domain deleted (BDD) analog
of human antihemophilic factor (coagulation factor VIII; FVIII) covalently fused to
the Fc domain of human immunoglobulin G1 (IgG1), the FVIII-binding D'D3 domain of
human von Willebrand factor (VWF), and two XTEN polypeptides, that can be used to
treat and control bleeding episodes in hemophilia A (congenital factor VIII deficiency).
Upon administration, efanesoctocog alfa temporarily replaces the missing coagulation
factor VIII, which binds factor IXa. Factor X is then converted to factor Xa. This
facilitates the clotting cascade by converting prothrombin to thrombin, and leads
to the conversion of fibrinogen to fibrin, and thus clot formation. This normalizes
the activated partial thromboplastin time (aPTT) that is needed for effective hemostasis.
Patients with hemophilia A lack factor VIII, a protein needed for normal clotting
of the blood. B-domain has no known biological function. The FVIII-binding D'D3 domain
of human VWF component of the fusion protein prevents FVIII interaction with endogenous
VWF. The Fc region of human IgG1 binds to the neonatal Fc receptor (FcRn). The two
XTEN polypeptides alter the hydrodynamic radius of the fusion protein. Altogether,
they extend the half-life of the fusion protein.;
UNII : WH7BHQ0RB4;
CAS number : 2252477-42-0;
Drug name : Altuviiio;
Molecule name : BIVV001;
NCI Metathesaurus CUI : CL1412429;
Origin ID : C174933;
UMLS CUI : C5421030;
Currated CISMeF NLP mapping
- BIVV001 [MeSH Supplementary Concept]
Semantic type(s)
concept_is_in_subset
https://ansm.sante.fr/tableau-acces-derogatoire/altuvoct
2025
false
false
false
France
French
Efanesoctocog Alfa
drug information
hemophilia A
hemorrhage
hemorrhage
---
https://www.ema.europa.eu/en/medicines/human/EPAR/altuvoct
2025
false
false
false
Netherlands
French
English
syndication feed
summary of product characteristics
package leaflet
drug evaluation
treatment outcome
Efanesoctocog Alfa
Efanesoctocog Alfa
orphan drug production
---
https://www.has-sante.fr/jcms/p_3563529/fr/altuvoct-efanesoctocog-alfa-hemophilie
2024
false
false
false
France
hemorrhage
hemorrhage
treatment outcome
insurance, health, reimbursement
recombinant fusion proteins
Efanesoctocog Alfa
adult
adolescent
child
infant, newborn
infant
injections, intravenous
evaluation of the transparency committee
hemophilia A
---
https://www.has-sante.fr/jcms/p_3499791/fr/altuvoct-efanesoctocog-alfa-hemophilie
2024
false
false
false
France
Efanesoctocog Alfa
hemophilia A
hemorrhage
evaluation of the transparency committee
---