Emapalumab

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Emapalumab
Monoclonal antibody
TypeWhole antibody
SourceHuman
TargetIFN-gamma
Clinical data
Pronunciation/ˈɛməpəlˌuməb/ EM-a-PAL-eu-mab[1]
SynonymsNI-0501
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 mass154.4 g/mol g·mol−1

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]

Adverse effects[edit]

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[edit]

Mechanism of action[edit]

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]

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]

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[edit]

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).[citation needed]

References[edit]

  1. ^ a b "STATEMENT ON A NONPROPRIETARY NAME ADOPTED BY THE USAN COUNCIL" (PDF). Retrieved 21 November 2018.
  2. ^ a b c d e "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. ^ a b "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.