Emicizumab

Summary

Emicizumab (CHEMBL3833393) is an approved antibody (ATC B02BX06) targeting F10 and F9; indicated across 2 conditions including hemophilia a.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Antibody
• ATC class: B02BX06
• Targets: 2 (F10, F9)
• Indications: 2 conditions
• Clinical trials: 34

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3833393
Name	Emicizumab
Type	Antibody
Max phase	4
ATC	B02BX06

Also known as: ACE-910, ACE910, Emicizumab, Emicizumab kxwh, Emicizumab-kxwh, Hemlibra, EMICIZUMAB

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
F10	coagulation factor X	Binding	5.73	0%	P00742
F9	coagulation factor IX	Binding	5.8	0%	P00740

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

Aggregated over 2 target gene(s): F10, F9.

Top Reactome pathways

17 total, by targets touching each:

Pathway	Targets	Genes
R-HSA-140834	2	F10, F9
R-HSA-140837	2	F10, F9
Gamma-carboxylation of protein precursors	2	F10, F9
Transport of gamma-carboxylated protein precursors from the endoplasmic reticulum to the Golgi apparatus	2	F10, F9
Removal of aminoterminal propeptides from gamma-carboxylated proteins	2	F10, F9
Defective factor IX causes thrombophilia	2	F10, F9
Defective cofactor function of FVIIIa variant	2	F10, F9
Defective F9 variant does not activate FX	2	F10, F9
Initiation of coagulation cascade	2	F10, F9
Regulation of clotting cascade	2	F10, F9
Amplification and propagation of coagulation cascade	2	F10, F9
R-HSA-140875	1	F10
Protein hydroxylation	1	F9
Defective F9 secretion	1	F9
Defective F9 activation	1	F9
Defective gamma-carboxylation of F9	1	F9
FXIIa, PKa-dependent activation of coagulation pathway	1	F9

Dominant GO biological processes

GO term	Targets
proteolysis	2
blood coagulation	2
hemostasis	2
positive regulation of cell migration	1
positive regulation of TOR signaling	1
zymogen activation	1

Indications & clinical

Indications

2 indications (2 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
hemophilia A	4	MONDO:0010602	MONDO:0010602

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 34.

Phase distribution

Phase	Trials
PHASE3	14
PHASE4	8
Not specified	6
PHASE2/PHASE3	2
PHASE2	2
PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04567511	PHASE4	RECRUITING	Hemlibra in Mild Hemophilia A
NCT05181618	PHASE4	ACTIVE_NOT_RECRUITING	A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
NCT06145373	PHASE4	RECRUITING	A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
NCT07158606	PHASE4	NOT_YET_RECRUITING	Understanding Treatment Outcomes and Immunologic Mechanisms in Altuviiio Immune Tolerance Induction
NCT03361137	PHASE4	TERMINATED	Study of Emicizumab Prophylaxis in Participants With Hemophilia A With or Without Inhibitors Undergoing Minor Surgical Procedures
NCT03921294	PHASE4	TERMINATED	HemLibra Prophylaxis in Patients With Hemophilic Pseudotumor
NCT04621916	PHASE4	UNKNOWN	Preventing Inhibitor Recurrence Indefinitely
NCT04690322	PHASE4	WITHDRAWN	POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies
NCT04431726	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors
NCT04563520	PHASE3	RECRUITING	SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis
NCT06938659	PHASE2/PHASE3	NOT_YET_RECRUITING	Low Dose Emicizumab vs Low Dose Factor VIII in Prophylaxis in Hemophilia A Patients
NCT06998524	PHASE3	RECRUITING	A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
NCT07416604	PHASE3	RECRUITING	A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A
NCT02196207	PHASE3	WITHDRAWN	Hemophilia Inhibitor Clinical Trials (INHIBIT) Platform
NCT02622321	PHASE3	COMPLETED	A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors
NCT02795767	PHASE3	COMPLETED	A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors
NCT02847637	PHASE3	COMPLETED	A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors
NCT03020160	PHASE3	COMPLETED	A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia A
NCT03191799	PHASE3	COMPLETED	A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
NCT03315455	PHASE3	COMPLETED	Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants
NCT04030052	PHASE3	WITHDRAWN	Emicizumab PUPs and Nuwiq ITI Study
NCT04158648	PHASE3	COMPLETED	A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors
NCT04303572	PHASE3	TERMINATED	The Hemophilia Inhibitor Eradication Trial
NCT06155955	PHASE2/PHASE3	UNKNOWN	Comparison of Outcomes Between Low Dose Emicizumab and Factor VIII in Clinically Severe Hemophilia A
NCT05345197	PHASE2	ACTIVE_NOT_RECRUITING	Emicizumab in Patients With Acquired Hemophilia A
NCT04188639	PHASE2	COMPLETED	Emicizumab in Acquired Hemophilia A
NCT05500807	PHASE1	RECRUITING	Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A
NCT03380780	PHASE1	COMPLETED	A Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Emicizumab in Healthy Chinese Volunteers
NCT04023019	Not specified	RECRUITING	Treatment of Hemophilia A Patients With FVIII Inhibitors
NCT05022459	Not specified	RECRUITING	Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis
NCT03154437	Not specified	APPROVED_FOR_MARKETING	An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors
NCT04805801	Not specified	UNKNOWN	The Safety of Emicizumab SC Injection in Korean Hemophilia A Patients With/Without FVIII Inhibitors
NCT05248594	Not specified	COMPLETED	Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A
NCT06104826	Not specified	UNKNOWN	Health-Related Quality of Life Outcomes and Bleeding Rates Among Patients With Severe Hemophilia A on Emicizumab

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

22 molecules share ≥1 primary target. Top 22 by shared-target count:

Molecule	Source	Status	Shared targets
LETAXABAN	ChEMBL	Phase 2	F10, F9
RAZAXABAN	ChEMBL	Phase 2	F10, F9
APIXABAN	ChEMBL + PubChem	Phase 4 (approved)	F10
EDOXABAN	ChEMBL + PubChem	Phase 4 (approved)	F10
ARGATROBAN	ChEMBL	Phase 4 (approved)	F10
BETRIXABAN	ChEMBL	Phase 4 (approved)	F10
FONDAPARINUX	ChEMBL	Phase 4 (approved)	F10
MELAGATRAN	ChEMBL	Phase 4 (approved)	F10
PENTAMIDINE	ChEMBL	Phase 4 (approved)	F10
RIVAROXABAN	ChEMBL	Phase 4 (approved)	F10
DABIGATRAN	ChEMBL	Phase 3	F10
DAREXABAN	ChEMBL	Phase 3	F10
GABEXATE	ChEMBL	Phase 3	F10
NAFAMOSTAT	ChEMBL	Phase 3	F10
OTAMIXABAN	ChEMBL	Phase 3	F10
EFEGATRAN	ChEMBL	Phase 2	F10
ERIBAXABAN	ChEMBL	Phase 2	F10
FIDEXABAN	ChEMBL	Phase 2	F10
GW813893	ChEMBL	Phase 2	F10
LY-517717	ChEMBL	Phase 2	F10
SEGATROXABAN	ChEMBL	Phase 2	F10
TANOGITRAN	ChEMBL	Phase 2	F10