Accession Number

Emapalumab, also known as NI-0501, is a fully human monoclonal antibody that targets interferon gamma. Emapalumab development was sponsored by NovImmune SA, further developed by Sobi and FDA approved on November 20, 2018.1,5 The approval of emapalumab was followed by the designation of orphan drug, priority review and breakthrough therapy.5 As well, emapalumab was given the status of PRIME by the EMA.7

Approved, Investigational
Biologic Classification
Protein Based Therapies
Monoclonal antibody (mAb)
Protein Chemical Formula
Protein Average Weight
154400.0 Da
Not Available
  • emapalumab-lzsg
External IDs
  • NI-0501



Emapalumab is indicated for the treatment of pediatric and adult patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance to conventional HLH therapy.5

The HLH condition is a hyperinflammatory status characterized by the overwhelming activation of normal T lymphocytes and macrophages which can lead to disturbances in the hematology profile and even death. As part of the condition profile, there have been reports proving a massive overexpression of interferon-gamma which is thought to drive the immune hyperactivation leading to organ failure.1 This condition is usually developed and present the symptomatic profile within the first months or years of life. These symptoms consist of fever, enlarged liver or spleen and a lower number of blood cells.5

Associated Conditions
Contraindications & Blackbox Warnings
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In phase 2/3 clinical trials, emapalumab administered concomitantly with dexamethasone reported an overall response in 63% of the patients. The overall response was defined as achievement of a complete or partial response or HLH improvement.6 In this trial and as a proof of interferon-gamma neutralization, there was registered a sharp decrease in serum CXCL9 and to avoid QT prolongation in the presence of low doses of emapalumab.8

Mechanism of action

Emapalumab acts by binding and neutralizing interferon-gamma.1 The specific interaction between emapalumab and interferon-gamma produces an inhibition in the interaction between interferon-gamma and its cognate receptor on T-cells which produces the neutralizing activity.7 It is important to consider that emapalumab inhibits both free and IFNGR1-bound interferon-gamma as well as the interaction with IFNGR1 and IFNGR2 at the cell surface.3

HLH is an immune dysregulation syndrome in which several cytokines are involved but it has been reported that interferon-gamma plays a pivotal role in the development of this disease as studies have shown a vast increase in the interferon-gamma levels in HLH patients.2

AInterferon gamma

In clinical pharmacokinetic studies, a dose of 1 mg/kg of emapalumab was administered which generated a peak concentration at steady state of 44 mcg/ml and a median steady-state concentration of 25 mcg/ml. The serum concentration of emapalumab increases proportionally between a dose of 1-3 mg/kg and the steady-state is attained by the 7th infusion.Label

Volume of distribution

The central and peripheral volume of distribution of emapalumab are 4.2 and 5.6 L, respectively.Label

Protein binding

Monoclonal antibodies are usually not required to have protein binding studies.Label


Monoclonal antibodies are thought to be internalized in endothelial cells bound to Fc receptor and rescued from metabolism by recycling. Later, they are degraded in the reticuloendothelial system to small peptides and amino acids which can be used for de-novo protein synthesis.4

Route of elimination

Emapalumab presents a target-mediated clearance that is dependent on interferon-gamma production.Label


Emapalumab elimination half-life is of approximately 22 days in healthy subjects and it ranges between 2.5-18.9 in HLH patients.Label


Emapalumab clearance is reported to be 0.007 L/h in healthy subjects. This clearance rate can vary in HLH patients depending on the production of interferon-gamma.Label

Adverse Effects
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There are no reported effects in male or female reproductive organs after an 8- or 13-week repeat-dose toxicity study in animals.Label

Affected organisms
  • Humans
Not Available
Pharmacogenomic Effects/ADRs
Not Available


Drug Interactions
This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
AbataceptThe risk or severity of adverse effects can be increased when Abatacept is combined with Emapalumab.
AbciximabThe risk or severity of adverse effects can be increased when Abciximab is combined with Emapalumab.
AbirateroneThe metabolism of Abiraterone can be increased when combined with Emapalumab.
AcalabrutinibThe metabolism of Acalabrutinib can be increased when combined with Emapalumab.
AcebutololThe metabolism of Acebutolol can be increased when combined with Emapalumab.
AcenocoumarolThe metabolism of Acenocoumarol can be increased when combined with Emapalumab.
AcetaminophenThe metabolism of Acetaminophen can be increased when combined with Emapalumab.
AcetohexamideThe metabolism of Acetohexamide can be increased when combined with Emapalumab.
Acetylsalicylic acidThe metabolism of Acetylsalicylic acid can be increased when combined with Emapalumab.
AcyclovirThe metabolism of Acyclovir can be increased when combined with Emapalumab.
Additional Data Available
  • Extended Description
    Extended Description

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  • Severity

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  • Evidence Level
    Evidence Level

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  • Action

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Food Interactions
Not Available


Brand Name Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing EndRegionImage
GamifantInjection10 mg/2mLIntravenousSWEDISH ORPHAN BIOVITRUM AB (PUBL)2019-05-17Not applicableUS flag
GamifantInjection10 mg/2mLIntravenousNovImmune SA2018-11-202021-11-30US flag
GamifantInjection50 mg/10mLIntravenousSWEDISH ORPHAN BIOVITRUM AB (PUBL)2019-05-17Not applicableUS flag
GamifantInjection50 mg/10mLIntravenousNovImmune SA2018-11-202021-11-30US flag
GamifantInjection100 mg/20mLIntravenousSWEDISH ORPHAN BIOVITRUM AB (PUBL)2020-06-26Not applicableUS flag
Additional Data Available
  • Application Number
    Application Number

    A unique ID assigned by the FDA when a product is submitted for approval by the labeller.

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  • Product Code
    Product Code

    A governmentally-recognized ID which uniquely identifies the product within its regulatory market.

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ATC Codes
L04AA39 — Emapalumab
Drug Categories
Chemical TaxonomyProvided by Classyfire
Not Available
Organic Compounds
Super Class
Organic Acids
Carboxylic Acids and Derivatives
Sub Class
Amino Acids, Peptides, and Analogues
Direct Parent
Alternative Parents
Not Available
Not Available
Molecular Framework
Not Available
External Descriptors
Not Available

Chemical Identifiers

CAS number


General References
  1. Kaplon H, Reichert JM: Antibodies to watch in 2018. MAbs. 2018 Feb/Mar;10(2):183-203. doi: 10.1080/19420862.2018.1415671. Epub 2018 Jan 16. [PubMed:29300693]
  2. Avau A, Matthys P: Therapeutic Potential of Interferon-gamma and Its Antagonists in Autoinflammation: Lessons from Murine Models of Systemic Juvenile Idiopathic Arthritis and Macrophage Activation Syndrome. Pharmaceuticals (Basel). 2015 Nov 25;8(4):793-815. doi: 10.3390/ph8040793. [PubMed:26610523]
  3. Zuber B, Rudstrom K, Ehrnfelt C, Ahlborg N: Epitope Mapping of Neutralizing Monoclonal Antibodies to Human Interferon-gamma Using Human-Bovine Interferon-gamma Chimeras. J Interferon Cytokine Res. 2016 Sep;36(9):542-51. doi: 10.1089/jir.2016.0017. Epub 2016 Jun 23. [PubMed:27336613]
  4. Tabrizi MA, Tseng CM, Roskos LK: Elimination mechanisms of therapeutic monoclonal antibodies. Drug Discov Today. 2006 Jan;11(1-2):81-8. doi: 10.1016/S1359-6446(05)03638-X. [PubMed:16478695]
  5. FDA news [Link]
  6. Clinical trials [Link]
  7. NHS [Link]
  8. American Society of Hematology [Link]
FDA label
Download (521 KB)

Clinical Trials

Clinical Trials
3RecruitingTreatmentPrimary Hemophagocytic Hymphohistiocytosis1
2Active Not RecruitingTreatmentJuvenile Idiopathic Arthritis (JIA) / Lymphohistiocytosis, Hemophagocytic / Macrophage Activation Syndrome1
2, 3CompletedTreatmentPrimary Haemophagocytic Lymphohistiocytosis1
2, 3RecruitingTreatmentHemophagocytic Lymphohistiocytoses1
2, 3RecruitingTreatmentNovel Coronavirus Infectious Disease (COVID-19)1
1CompletedNot AvailableHealthy Volunteers1
Not AvailableRecruitingOtherLymphohistiocytosis, Hemophagocytic1


Not Available
Not Available
Dosage Forms
InjectionIntravenous10 mg/2mL
InjectionIntravenous100 mg/20mL
InjectionIntravenous50 mg/10mL
Not Available
Not Available


Experimental Properties
melting point (°C)78 ºCJi-Hee, et al. Front Immunol, 7:394 (2016)
boiling point (°C)Fab and Fc domains denaturates at 60 and 70 ºC respectivelyArnoldus W. et al. (2000). Biophysical Journal. Vol 78. 394-404
water solubility50 mg/mlHuman IgG purified. Product Information
isoelectric point6.6 - 7.2Jin, et al. Electrophoresis. Sep;23(19):3385-91. (2002).


Pharmacological action
General Function
Interferon-gamma receptor binding
Specific Function
Produced by lymphocytes activated by specific antigens or mitogens. IFN-gamma, in addition to having antiviral activity, has important immunoregulatory functions. It is a potent activator of macrop...
Gene Name
Uniprot ID
Uniprot Name
Interferon gamma
Molecular Weight
19348.165 Da
  1. Kaplon H, Reichert JM: Antibodies to watch in 2018. MAbs. 2018 Feb/Mar;10(2):183-203. doi: 10.1080/19420862.2018.1415671. Epub 2018 Jan 16. [PubMed:29300693]

Drug created on November 29, 2018 10:57 / Updated on June 12, 2020 11:42

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