Emapalumab

Emapalumab, marketed under the trade name Gamifant, is an anti-interferon-gamma (IFNγ) antibody used for the treatment of hemophagocytic lymphohistiocytosis (HLH),[3] which currently has no cure.[4]

Emapalumab
Monoclonal antibody
TypeWhole antibody
SourceHuman
TargetIFN-gamma
Clinical data
Pronunciation/ˈɛməpəlˌuməb/ EM-a-PAL-eu-mab[1]
Other namesNI-0501, emapalumab-lzsg
AHFS/Drugs.comMonograph
MedlinePlusa619024
License data
Routes of
administration		IV, parenteral
ATC code
Legal status
Legal status
  • US: Rx-only[2]
Identifiers
CAS Number
DrugBank
ChemSpider
  • none
UNII
KEGG
Chemical and physical data
FormulaC6430H9898N1718O2038S46
Molar mass145352.66 g·mol−1

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][5] Serious adverse effects occurred in about half of the subjects studied in the clinical trial that led to its FDA approval.[5]

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 FDA awarded orphan drug status in 2012, and breakthrough therapy designation in 2016 on the basis of preliminary data from the phase II trial.[6][7]

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.[8] The trial targets patients under the age of 18 who have failed to improve on conventional treatments.[9] This study was realised in the context of an EU-funded FP7 project, named FIGHT-HLH (306124).

References
  1. "STATEMENT ON A NONPROPRIETARY NAME ADOPTED BY THE USAN COUNCIL" (PDF). Retrieved 21 November 2018.
  2. "Gamifant injection, for intravenous use. Package Insert" (PDF). gamifant.com. Swedish Orphan Biovitrum. Retrieved 21 November 2018.
  3. "FDA Approves Gamifant® (emapalumab-lzsg), the First and Only Treatment Indicated for Primary Hemophagocytic Lymphohistiocytosis (HLH)". Business Wire. Business Wire, Inc. Retrieved 21 November 2018.
  4. "Long-term Follow-up of HLH Patients Who Received Treatment With NI-0501, an Anti-interferon Gamma Monoclonal Antibody - Full Text View - ClinicalTrials.gov". clinicaltrials.gov.
  5. "Emapalumab Approved for Rare Primary Hemophagocytic Lymphohistiocytosis". Rare Disease Report. Retrieved 22 November 2018.
  6. "Novimmune's NI-0501 Granted Breakthrough Therapy Designation by US FDA for Treatment of Patients With Primary Hemophagocytic Lymphohistiocytosis (HLH) - FierceBiotech". www.fiercebiotech.com.
  7. "Emapalumab - NovImmune - AdisInsight". adisinsight.springer.com.
  8. "A Study to Investigate the Safety and Efficacy of an Anti-IFNγ mAb in Children Affected by Primary Haemophagocytic Lymphohistiocytosis - Full Text View - ClinicalTrials.gov". clinicaltrials.gov.
  9. "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.
  • "Emapalumab". Drug Information Portal. U.S. National Library of Medicine.
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