Efanesoctocog alfa 1,000unit powder and solvent for solution for injection vials
Requires a prescription from a doctor or prescriber
Antifibrinolytic drugs and haemostatics
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Suspected adverse reactions reported for Efanesoctocog alfa
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1 branded products available
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Altuvoct 1,000unit powder and solvent for solution for injection vials
Therapeutically similar medicines
Similarity based on WHO Anatomical Therapeutic Chemical (ATC) classification and NHS BNF section grouping. Source data: NHS dm+d via TRUD (OGL v3.0), WHO ATC/DDD Index.
NHS prescribing volume and spending trends
Clinical guidelines and formulary information
British National Formulary
Efanesoctocog alfa
Source: British National Formulary, NICE. Joint Formulary Committee. Contains public sector information licensed under the Open Government Licence v3.0.
NICE clinical guidance(1)
Source: National Institute for Health and Care Excellence (NICE). Contains public sector information licensed under the Open Government Licence v3.0.
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SNOMED CT and dm+d codes from NHS TRUD (Technology Reference data Update Distribution), licensed under the Open Government Licence v3.0. BNF codes from NHS Business Services Authority (NHSBSA). ATC codes from the WHO Collaborating Centre for Drug Statistics Methodology (whocc.no).
Active and completed clinical studies from ClinicalTrials.gov
Source: ClinicalTrials.gov, a database of the U.S. National Library of Medicine (NLM), National Institutes of Health (NIH). Data accessed via ClinicalTrials.gov API v2. Trial information is provided for research purposes and does not constitute medical advice.
Pharmacology and chemical data from DrugBank
Key facts
Drug status
Approved
Major interactions
None known
Half-life
39.9 h
Mechanism
Efanesoctocog alfa is a recombinant factor VIII (FVIII) analogue fusion protein…
Food interactions
None known
Human targets
4 targets
Data: DrugBank · CC BY-NC 4.0
Pharmacokinetics at a glance
Absorption
6 years
Half-life
39.9 h
Volume of distribution
38.0 mL
Metabolism
Clearance
0.74 mL
Pharmacokinetic data: DrugBank · CC BY-NC 4.0
In February 2023, efanesoctocog alfa was approved by the FDA as a new class of factor VIII therapy for hemophilia A.[L45379][L45414]
[L45379]
[L45379]
Symptomatic and supportive measures are recommended. The carcinogenicity, mutagenicity or effects on fertility of efanesoctocog alfa have not been evaluated.
[L45379]
In plasma, most FVIII circulates in a complex with VWF, which protects FVIII from degradation and extends its half-life. However, it also sets a half-life ceiling of approximately 15 to 19 h.[A257469] Linking the D'D3 domain of VWF to the recombinant FVIII-Fc fusion protein provides protection and stability to FVIII and prevents FVIII interaction with endogenous VWF. The lysosomal degradation of efanesoctocog alfa is delayed thanks to the Fc region of human immunoglobulin G1 (IgG1) that binds to the neonatal Fc receptor (FcRn), and the XTEN polypeptides alter the hydrodynamic radius of the fusion protein and reduce clearance and degradation rates.[L45379] Altogether, these modifications lead to a 3- to 4-fold increase in FVIII half-life.[A257469][L45379]
Hemophilia A is a genetic disorder caused by missing or defective FVIII. The use of efanesoctocog alfa in patients with hemophilia A increases FVIII plasma levels, temporarily correcting coagulation deficiency.[L45379]
Efanesoctocog alfa has an acceptable side-effect profile and is usually well tolerated.[A257484][A257489] However, patients treated with efanesoctocog alfa may present allergic-type hypersensitivity reactions, including anaphylaxis. Also, the formation of FVIII neutralizing antibodies is possible following the administration of efanesoctocog alfa.[L45379]
How the body processes this drug — absorption, distribution, metabolism, and elimination
Sex, race (White, Asian), VWF antigen activity, hematocrit level, blood type, HCV status, or HIV status did not have a clinically significant effect on efanesoctocog alfa either. At steady state (week 26), the pharmacokinetic profile of efanesoctocog alfa was comparable to the one obtained after the first dose.
[L45379]
[L45379]
[L45379]
[L45379]
Proteins and enzymes this drug interacts with in the body
PMID:22409427
Factor Xa activates pro-inflammatory signaling pathways in a protease-activated receptor (PAR)-dependent manner .
PMID:24041930 PMID:30568593 PMID:34831181 PMID:18202198
Up-regulates expression of protease-activated receptors (PARs) F2R, F2RL1 and F2RL2 in dermal microvascular endothelial cells .
PMID:35738824
Triggers the production of pro-inflammatory cytokines, such as MCP-1/CCL2 and IL6, in cardiac fibroblasts and umbilical vein endothelial cells in PAR-1/F2R-dependent manner .
PMID:30568593 PMID:34831181
Triggers the production of pro-inflammatory cytokines, such as MCP-1/CCL2, IL6, TNF-alpha/TNF, IL-1beta/IL1B, IL8/CXCL8 and IL18, in endothelial cells and atrial tissues .
PMID:24041930 PMID:35738824 PMID:9780208
Induces expression of adhesion molecules, such as ICAM1, VCAM1 and SELE, in endothelial cells and atrial tissues .
PMID:24041930 PMID:35738824 PMID:9780208
Increases expression of phosphorylated ERK1/2 in dermal microvascular endothelial cells and atrial tissues .
PMID:24041930 PMID:35738824
Triggers activation of the transcription factor NF-kappa-B in dermal microvascular endothelial cells and atrial tissues .
PMID:24041930 PMID:35738824
Activates pro-inflammatory and pro-fibrotic responses in dermal fibroblasts and enhances wound healing probably via PAR-2/F2RL1-dependent mechanism .
PMID:18202198
Activates barrier protective signaling responses in endothelial cells in PAR-2/F2RL1-dependent manner; the activity depends on the cleavage of PAR-2/F2RL1 by factor Xa .
PMID:22409427
Up-regulates expression of plasminogen activator inhibitor 1 (SERPINE1) in atrial tissues PMID:24041930
In the kidney, mediates the tubular uptake and clearance of leptin (By similarity). Also mediates transport of leptin across the blood-brain barrier through endocytosis at the choroid plexus epithelium (By similarity). Endocytosis of leptin in neuronal cells is required for hypothalamic leptin signaling and leptin-mediated regulation of feeding and body weight (By similarity).
Mediates endocytosis and subsequent lysosomal degradation of CST3 in kidney proximal tubule cells (By similarity). Mediates renal uptake of 25-hydroxyvitamin D3 in complex with the vitamin D3 transporter GC/DBP (By similarity). Mediates renal uptake of metallothionein-bound heavy metals .
PMID:15126248
Together with CUBN, mediates renal reabsorption of myoglobin (By similarity).
Mediates renal uptake and subsequent lysosomal degradation of APOM (By similarity). Plays a role in kidney selenium homeostasis by mediating renal endocytosis of selenoprotein SEPP1 (By similarity). Mediates renal uptake of the antiapoptotic protein BIRC5/survivin which may be important for functional integrity of the kidney .
PMID:23825075
Mediates renal uptake of matrix metalloproteinase MMP2 in complex with metalloproteinase inhibitor TIMP1 (By similarity).
Mediates endocytosis of Sonic hedgehog protein N-product (ShhN), the active product of SHH (By similarity). Also mediates ShhN transcytosis (By similarity). In the embryonic neuroepithelium, mediates endocytic uptake and degradation of BMP4, is required for correct SHH localization in the ventral neural tube and plays a role in patterning of the ventral telencephalon (By similarity).
Required at the onset of neurulation to sequester SHH on the apical surface of neuroepithelial cells of the rostral diencephalon ventral midline and to control PTCH1-dependent uptake and intracellular trafficking of SHH (By similarity). During neurulation, required in neuroepithelial cells for uptake of folate bound to the folate receptor FOLR1 which is necessary for neural tube closure (By similarity). In the adult brain, negatively regulates BMP signaling in the subependymal zone which enables neurogenesis to proceed (By similarity).
In astrocytes, mediates endocytosis of ALB which is required for the synthesis of the neurotrophic factor oleic acid (By similarity). Involved in neurite branching (By similarity). During optic nerve development, required for SHH-mediated migration and proliferation of oligodendrocyte precursor cells (By similarity).
Mediates endocytic uptake and clearance of SHH in the retinal margin which protects retinal progenitor cells from mitogenic stimuli and keeps them quiescent (By similarity). Plays a role in reproductive organ development by mediating uptake in reproductive tissues of androgen and estrogen bound to the sex hormone binding protein SHBG (By similarity). Mediates endocytosis of angiotensin-2 (By similarity).
Also mediates endocytosis of angiotensis 1-7 (By similarity). Binds to the complex composed of beta-amyloid protein 40 and CLU/APOJ and mediates its endocytosis and lysosomal degradation (By similarity). Required for embryonic heart development (By similarity).
Required for normal hearing, possibly through interaction with estrogen in the inner ear (By similarity)
Plays essential roles in vascularization. Critical for normal heart development and for regulating the vascular response to injury. Also required for avascular cartilage development
Chemical identifiers
CAS, UNII, InChI Key and database cross-references
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Chemical identifiers
CAS, UNII, InChI Key and database cross-references
Linked compound data from DrugBank Open Data (CC BY-NC 4.0)
Efanesoctocog alfa
Additional database identifiers
HUGO Gene Nomenclature Committee (HGNC)
HGNC:3551
GenAtlas
F9
GeneCards
F9
GenBank Gene Database
K02402
GenBank Protein Database
182609
Guide to Pharmacology
2364
UniProt Accession
FA9_HUMAN
HUGO Gene Nomenclature Committee (HGNC)
HGNC:3528
GenAtlas
F10
GeneCards
F10
GenBank Gene Database
K03194
GenBank Protein Database
182841
Guide to Pharmacology
2359
UniProt Accession
FA10_HUMAN
HUGO Gene Nomenclature Committee (HGNC)
HGNC:6694
GenAtlas
LRP2
GeneCards
LRP2
GenBank Gene Database
U33837
GenBank Protein Database
1809240
UniProt Accession
LRP2_HUMAN
HUGO Gene Nomenclature Committee (HGNC)
HGNC:5273
GenAtlas
HSPG2
GeneCards
HSPG2
GenBank Gene Database
X62515
GenBank Protein Database
29470
UniProt Accession
PGBM_HUMAN
DrugBank citations
If you use DrugBank data in your research, please cite the following publications: