Emapalumab

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Wikipedia

Emapalumab
Monoclonal antibody
TypeWhole antibody
SourceHuman
TargetIFN-gamma
Clinical data
Pronunciation/ˈɛməpəlˌuməb/ EM-a-PAL-eu-mab[1]
Trade namesGamifant
Other namesNI-0501, emapalumab-lzsg
AHFS/Drugs.comMonograph
MedlinePlusa619024
License data
Routes of
administration		Intravenous
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
ChemSpider
  • none
UNII
KEGG
Chemical and physical data
FormulaC6430H9898N1718O2038S46
Molar mass145352.66 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

Emapalumab is used to treat primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy.[3]

Adverse effects

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

Mechanism of action

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

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

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

The research name of emapalumab was NI-0501.[1] A phase II/III trial began in 2013 and is ongoing as of August 2018.[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

  1. 1 2 "STATEMENT ON A NONPROPRIETARY NAME ADOPTED BY THE USAN COUNCIL" (PDF). Retrieved 21 November 2018.
  2. 1 2 3 4 5 "Gamifant (- emapalumab-lzsg injection". DailyMed. 30 June 2020. Retrieved 7 October 2020.
  3. 1 2 3 "FDA approves emapalumab for hemophagocytic lymphohistiocytosis". U.S. Food and Drug Administration (FDA). 20 November 2018. Retrieved 19 March 2021.[dead link] Public Domain This article incorporates text from this source, which is in the public domain.
  4. ↑ "FDA Approves Gamifant (emapalumab-lzsg), the First and Only Treatment Indicated for Primary Hemophagocytic Lymphohistiocytosis (HLH)" (Press release). Sobi. 20 November 2018. Retrieved 21 November 2018 – via Business Wire.
  5. ↑ Cheloff AZ, Al-Samkari H (July 2020). "Emapalumab for the treatment of hemophagocytic lymphohistiocytosis". Drugs of Today. 56 (7): 439–446. doi:10.1358/dot.2020.56.7.3145359. PMID 32648854. S2CID 220473902.
  6. ↑ 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
  7. ↑ New Drug Therapy Approvals 2018. U.S. Food and Drug Administration (FDA) (Report). January 2019. Archived from the original (PDF) on 26 August 2019. Retrieved 16 September 2020.
  8. 1 2 "FDA approves Sobi's Gamifant to treat severe inflammatory arthritis complication - PMLiVE". pmlive.com. 30 June 2025. Retrieved 30 June 2025.
  9. 1 2 "Emapalumab Approved for Rare Primary Hemophagocytic Lymphohistiocytosis". Rare Disease Report. Archived from the original on 22 November 2018. Retrieved 22 November 2018.
  10. ↑ "Emapalumab Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 25 September 2020. Archived from the original on 15 December 2023. Retrieved 19 March 2021.
  11. ↑ "Emapalumab Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 26 March 2010. Archived from the original on 11 December 2023. Retrieved 19 March 2021.
  12. ↑ "Novimmune's NI-0501 Granted Breakthrough Therapy Designation by US FDA for Treatment of Patients With Primary Hemophagocytic Lymphohistiocytosis (HLH) - FierceBiotech". www.fiercebiotech.com. 16 March 2016.
  13. ↑ "Gamifant: Pending EC decision". European Medicines Agency. 24 July 2020. Archived from the original on 23 September 2020. Retrieved 21 September 2020.
  14. ↑ "Gamifant EPAR". European Medicines Agency (EMA). 21 July 2020. Retrieved 19 March 2021.
  15. ↑ 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
  16. ↑ "NI-0501: A Study to Investigate the Safety and Efficacy of an Anti-IFN? mAb in Children Affected by Primary Hemophagocytic Lymphohistiocytosis". www.cincinnatichildrens.org.
  • 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