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 | |
| Routes of administration | Intravenous |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| ChemSpider |
|
| UNII | |
| KEGG | |
| Chemical and physical data | |
| Formula | C6430H9898N1718O2038S46 |
| Molar mass | 145352.66g·mol−1 |
Emapalumab,sold under the brand nameGamifant,is an anti-interferon-gamma (IFNγ)antibody medication used for the treatment ofhemophagocytic 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 afirst-in-class medication.[7]
Medical uses
[edit]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]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][8]Serious adverse effects occurred in about half of the subjects studied in the clinical trial that led to its FDA approval.[8]
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 calledpolypeptides,emapalumab is broken down into smaller peptides via the body's normalcatabolism.[2]
Society and culture
[edit]Legal status
[edit]The U.S.Food and Drug Administration(FDA) grantedorphan drugdesignations in 2010 and 2020,[9][10]andbreakthrough therapydesignation in 2016, on the basis of preliminary data from the phase II trial.[11]
In July 2020, and again in November 2020, theEuropean Medicines Agency(EMA) recommended the refusal of the marketing authorization for emapalumab.[12][13]
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[update].[14]The trial targets patients under the age of 18 who have failed to improve on conventional treatments.[15]This study was realised in the context of an EU-funded FP7 project, named FIGHT-HLH (306124).[citation needed]
References
[edit]- ^ab"STATEMENT ON A NONPROPRIETARY NAME ADOPTED BY THE USAN COUNCIL"(PDF).Retrieved21 November2018.
- ^abcde"Gamifant (- emapalumab-lzsg injection".DailyMed.30 June 2020.Retrieved7 October2020.
- ^abc"FDA approves emapalumab for hemophagocytic lymphohistiocytosis".U.S.Food and Drug Administration(FDA).20 November 2018.Retrieved19 March2021.
This article incorporates text from this source, which is in thepublic domain.
- ^"FDA Approves Gamifant (emapalumab-lzsg), the First and Only Treatment Indicated for Primary Hemophagocytic Lymphohistiocytosis (HLH)"(Press release). Sobi. 20 November 2018.Retrieved21 November2018– via Business Wire.
- ^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.PMID32648854.S2CID220473902.
- ^Clinical trial numberNCT02069899for "Long-term Follow-up of HLH Patients Who Received Treatment With NI-0501, an Anti-interferon Gamma Monoclonal Antibody" atClinicalTrials.gov
- ^New Drug Therapy Approvals 2018(PDF).U.S.Food and Drug Administration(FDA)(Report). January 2019.Retrieved16 September2020.
- ^ab"Emapalumab Approved for Rare Primary Hemophagocytic Lymphohistiocytosis".Rare Disease Report.Archived fromthe originalon 22 November 2018.Retrieved22 November2018.
- ^"Emapalumab Orphan Drug Designations and Approvals".U.S.Food and Drug Administration(FDA).25 September 2020.Retrieved19 March2021.
- ^"Emapalumab Orphan Drug Designations and Approvals".U.S.Food and Drug Administration(FDA).26 March 2010.Retrieved19 March2021.
- ^"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.
- ^"Gamifant: Pending EC decision".European Medicines Agency.24 July 2020.Retrieved21 September2020.
- ^"Gamifant EPAR".European Medicines Agency(EMA).21 July 2020.Retrieved19 March2021.
- ^Clinical trial numberNCT01818492for "A Study to Investigate the Safety and Efficacy of an Anti-IFNγ mAb in Children Affected by Primary Haemophagocytic Lymphohistiocytosis" atClinicalTrials.gov
- ^"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.
External links
[edit]- Clinical trial numberNCT01818492for "A Study to Investigate the Safety and Efficacy of an Anti-IFNγ mAb in Children Affected by Primary Haemophagocytic Lymphohistiocytosis" atClinicalTrials.gov
