Emapalumab
| Monoclonal antibody | |
|---|---|
| Type | Whole antibody |
| Source | Human |
| Target | IFN-gamma |
| Clinical data | |
| Pronunciation | /ˈɛməpəlˌuməb/ EM-a-PAL-eu-mab[1] |
| Other names | NI-0501, emapalumab-lzsg |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a619024 |
| License data |
|
| Routes of administration | IV, parenteral |
| 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, 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
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[update].[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
- ^ a b "STATEMENT ON A NONPROPRIETARY NAME ADOPTED BY THE USAN COUNCIL" (PDF). Retrieved 21 November 2018.
- ^ a b c d e "Gamifant injection, for intravenous use. Package Insert" (PDF). gamifant.com. Swedish Orphan Biovitrum. Retrieved 21 November 2018.
- ^ "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.
- ^ "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.
- ^ a b "Emapalumab Approved for Rare Primary Hemophagocytic Lymphohistiocytosis". Rare Disease Report. Retrieved 22 November 2018.
- ^ "Novimmune's NI-0501 Granted Breakthrough Therapy Designation by US FDA for Treatment of Patients With Primary Hemophagocytic Lymphohistiocytosis (HLH) - FierceBiotech". www.fiercebiotech.com.
- ^ "Emapalumab - NovImmune - AdisInsight". adisinsight.springer.com.
- ^ "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.
- ^ "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
- "Emapalumab". Drug Information Portal. U.S. National Library of Medicine.