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 | 145352.66 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]
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 called polypeptides, emapalumab is broken down into smaller peptides via the body's normal catabolism.[2]
Society and culture
[edit]Legal status
[edit]The U.S. Food and Drug Administration (FDA) granted orphan drug designations in 2010 and 2020,[9][10] and breakthrough therapy designation in 2016, on the basis of preliminary data from the phase II trial.[11]
In July 2020, and again in November 2020, the European 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]- ^ a b "STATEMENT ON A NONPROPRIETARY NAME ADOPTED BY THE USAN COUNCIL" (PDF). Retrieved 21 November 2018.
- ^ a b c d e "Gamifant (- emapalumab-lzsg injection". DailyMed. 30 June 2020. Retrieved 7 October 2020.
- ^ a b c This article incorporates text from this source, which is in the public domain.
- ^ "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.
- ^ 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.
- ^ 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
- ^ New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Retrieved 16 September 2020.
- ^ a b "Emapalumab Approved for Rare Primary Hemophagocytic Lymphohistiocytosis". Rare Disease Report. Archived from the original on 22 November 2018. Retrieved 22 November 2018.
- ^ "Emapalumab Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 25 September 2020. Retrieved 19 March 2021.
- ^ "Emapalumab Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 26 March 2010. Retrieved 19 March 2021.
- ^ "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. Retrieved 21 September 2020.
- ^ "Gamifant EPAR". European Medicines Agency (EMA). 21 July 2020. Retrieved 19 March 2021.
- ^ 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
- ^ "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 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
