Emapalumab

Summary

Emapalumab (CHEMBL3989977) is an approved antibody (ATC L04AG09); indicated across 9 conditions including adult-onset still disease and macrophage activation syndrome.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Antibody
• ATC class: L04AG09
• Indications: 9 conditions
• Clinical trials: 21

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3989977
Name	Emapalumab
Type	Antibody
Max phase	4
ATC	L04AG09

Also known as: Emapalumab, Emapalumab lzsg, Emapalumab-lzsg, Gamifant, NI-0501, EMAPALUMAB

Targets

Targets

No target linkage available.

Bioactivity

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

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

9 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
adult-onset Still disease	3	MONDO:0019355	EFO:0007135
macrophage activation syndrome	3	MONDO:0015545	EFO:1001806
systemic lupus erythematosus	3	MONDO:0007915	MONDO:0007915
immune system disorder	2	MONDO:0005046	EFO:0000540
graft versus host disease	2	MONDO:0013730	EFO:0004599
severe acute respiratory syndrome	2	MONDO:0005091	MONDO:0100096
chronic granulomatous disease	1	MONDO:0018305	MONDO:0018305

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

Clinical trials

Total trials: 21.

Phase distribution

Phase	Trials
PHASE2	10
PHASE2/PHASE3	4
PHASE1	3
PHASE3	2
PHASE4	1
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05744063	PHASE4	COMPLETED	A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients
NCT01818492	PHASE2/PHASE3	COMPLETED	Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis
NCT02069899	PHASE2/PHASE3	COMPLETED	A Study for Long-term Follow-up of Hemophagocytic Lymphohistiocytosis (HLH) Participants Who Received Treatment With Emapalumab (NI-0501), an Anti-interferon Gamma Monoclonal Antibody
NCT03312751	PHASE3	COMPLETED	Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
NCT03985423	PHASE2/PHASE3	TERMINATED	A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Emapalumab in Adult Patients With HLH
NCT04324021	PHASE2/PHASE3	TERMINATED	Efficacy and Safety of Emapalumab and Anakinra in Reducing Hyperinflammation and Respiratory Distress in Patients With COVID-19 Infection.
NCT05001737	PHASE3	COMPLETED	Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still’s or SLE
NCT05787574	PHASE2	RECRUITING	A Study of Treatment of Inflammation Before Stem Cell Transplant in People With a Primary Immune Regulatory Disorder (PIRD) and/or an Autoinflammatory Condition
NCT06285825	PHASE2	RECRUITING	A Pilot Study of Emapalumab for the Treatment of CAR T-Cell Therapy-Associated Prolonged Cytopenia
NCT06430788	PHASE2	RECRUITING	A Study of Emapalumab for Pediatric Aplastic Anemia
NCT06550141	PHASE2	RECRUITING	Emapalumab Prevention of CAR-T Cell Associated Toxicities
NCT07202598	PHASE2	RECRUITING	Randomized Stepped Wedge Study of Emapalumab in APECED Enteritis
NCT07486869	PHASE2	NOT_YET_RECRUITING	Emapalumab MDA5 Rapidly Progressive Interstitial Lung Disease (RP-ILD) Study
NCT07538336	PHASE2	NOT_YET_RECRUITING	Targeting Interferon Gamma With Emapalumab to Lung Transplant Recipients With Interferon Gamma-high Acute Lung Allograft Dysfunction
NCT07567014	PHASE2	NOT_YET_RECRUITING	Emapalumab Prophylaxis of Bispecific T-Cell Engagers (BiTEs) Associated CRS and ICANS
NCT03311854	PHASE2	COMPLETED	A Study to Investigate the Safety and Efficacy of Emapalumab, an Anti-IFN-gamma mAb in Patients With Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still’s Disease (AOSD) Developing Macrophage Activation Syndrome/Secondary HLH (MAS/sHLH)
NCT04731298	PHASE2	TERMINATED	Study to Investigate the Pharmacokinetics, Pharmacodynamics and Assess the Efficacy and Safety to Support Dose Selection of Emapalumab in Pre-empting Graft Failure in Patients at High Risk After HSCT.
NCT06996119	PHASE1	RECRUITING	Emapalumab With Post-Transplant Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Graft-versus-Host Disease After Donor Reduced-Intensity Hematopoietic Cell Transplant
NCT01459562	PHASE1	COMPLETED	First in Human Study of an Anti-IFN Gamma Monoclonal Antibody in Healthy Volunteers
NCT04765553	PHASE1	COMPLETED	A Study to Evaluate Emapalumab in Japanese Healthy Volunteers.
NCT07244419	EARLY_PHASE1	RECRUITING	Prevention of Graft Rejection in Hematopoietic Stem Cell Transplant (HSCT) Recipients

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

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).