Emapalumab
| Monoclonal antibody | |
|---|---|
| Type | Whole antibody |
| Source | Human |
| Target | IFN-gamma |
| Clinical data | |
| Pronunciation | /ˈɛməpəlˌuməb/ EM-a-PAL-eu-mab[1] |
| Trade names | Gamifant |
| Other names | NI-0501, emapalumab-lzsg |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a619024 |
| License data |
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| Routes of administration | Intravenous |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| ChemSpider |
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| UNII | |
| KEGG | |
| E number | {{#property:P628}} |
| CompTox Dashboard (EPA) |
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| ECHA InfoCard | {{#property:P2566}}Lua error in Module:EditAtWikidata at line 29: attempt to index field 'wikibase' (a nil value). |
| Chemical and physical data | |
| Formula | C6430H9898N1718O2038S46 |
| Molar mass | 145352.66 g·mol−1 |
Emapalumab, sold under the brand name Gamifant, is an anti-interferon-gamma (IFNγ) antibody medication used for the treatment of hemophagocytic lymphohistiocytosis (HLH),[3][4][5] which has no cure.[6]
The most common side effects include infections, hypertension, infusion-related reactions, and pyrexia.[3]
The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[7]
In June 2025, the U.S. Food and Drug Administration (FDA) approved emapalumab-lzsg for the treatment of macrophage activation syndrome (MAS) in patients with Still’s disease.[8]
Medical uses
[edit | edit source]Emapalumab is used to treat primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy.[3]
Adverse effects
[edit | edit source]In the clinical trials that lead to emapalumab's FDA approval, the most commonly reported adverse effects were infections (56%), high blood pressure (41%), infusion reactions (27%), and fever (24%).[2][9] Serious adverse effects occurred in about half of the subjects studied in the clinical trial that led to its FDA approval.[9]
Pharmacology
[edit | edit source]Mechanism of action
[edit | edit source]In the setting of HLH, over-secretion of IFN-γ is thought to contribute to the pathogenesis of the disease.[2] Emapalumab binds and neutralizes IFN-γ, preventing it from inducing pathological effects.[2]
Pharmacokinetics
[edit | edit source]Like other antibody-based medications, which are made of amino acid chains called polypeptides, emapalumab is broken down into smaller peptides via the body's normal catabolism.[2]
Society and culture
[edit | edit source]Legal status
[edit | edit source]The U.S. Food and Drug Administration (FDA) granted orphan drug designations in 2010 and 2020,[10][11] and breakthrough therapy designation in 2016, on the basis of preliminary data from the phase II trial.[12]
In July 2020, and again in November 2020, the European Medicines Agency (EMA) recommended the refusal of the marketing authorization for emapalumab.[13][14]
In June 2025, the FDA approved emapalumab-lzsg for the treatment of macrophage activation syndrome (MAS) in patients with Still’s disease.[8]
Research
[edit | edit source]The research name of emapalumab was NI-0501.[1] A phase II/III trial began in 2013 and is ongoing as of August 2018[update].[15] The trial targets patients under the age of 18 who have failed to improve on conventional treatments.[16] This study was realised in the context of an EU-funded FP7 project, named FIGHT-HLH (306124).[citation needed]
References
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- ^ a b c Lua error in Module:Citation/CS1/Configuration at line 2172: attempt to index field '?' (a nil value).[dead link] Public Domain This article incorporates text from this source, which is in the public domain.
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- ^ Clinical trial number NCT02069899 for "Long-term Follow-up of HLH Patients Who Received Treatment With NI-0501, an Anti-interferon Gamma Monoclonal Antibody" at ClinicalTrials.gov
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- ^ Clinical trial number NCT01818492 for "A Study to Investigate the Safety and Efficacy of an Anti-IFNγ mAb in Children Affected by Primary Haemophagocytic Lymphohistiocytosis" at ClinicalTrials.gov
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External links
[edit | edit source]- Clinical trial number NCT01818492 for "A Study to Investigate the Safety and Efficacy of an Anti-IFNγ mAb in Children Affected by Primary Haemophagocytic Lymphohistiocytosis" at ClinicalTrials.gov
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