Emapalumab

{{Short description|Monoclonal antibody}} {{Use dmy dates|date=November 2019}} {{cs1 config |name-list-style=vanc |display-authors=6}} {{Infobox drug | type = mab | image = | width = | alt = | caption =

| mab_type = mab | source = u | target = IFN-gamma

| pronounce ={{IPAc-en|ˈ|ɛ|m|ə|p|ə|l|ˌ|u|m|ə|b}} {{respell|EM|a|PAL|eu|mab}}<ref name="SearchUSAN">{{cite web |title=STATEMENT ON A NONPROPRIETARY NAME ADOPTED BY THE USAN COUNCIL |url=https://searchusan.ama-assn.org/usan/documentDownload?uri=%2Funstructured%2Fbinary%2Fusan%2Femapalumab.pdf |access-date=21 November 2018}}</ref> | tradename = Gamifant | Drugs.com = {{drugs.com|monograph|emapalumab-lzsg}} | MedlinePlus = a619024 | licence_EU =  | DailyMedID = Emapalumab | licence_US =  | pregnancy_AU =  | pregnancy_AU_comment = | pregnancy_category= | routes_of_administration = Intravenous | class = | ATCvet = | ATC_prefix = L04 | ATC_suffix = AG09 | ATC_supplemental =

| legal_AU =  | legal_AU_comment = | legal_BR =  | legal_BR_comment = | legal_CA =  | legal_CA_comment = | legal_DE =  | legal_DE_comment = | legal_NZ =  | legal_NZ_comment = | legal_UK =  | legal_UK_comment = | legal_US = Rx-only | legal_US_comment = <ref name="Gamifant FDA label">{{cite web | title=Gamifant (- emapalumab-lzsg injection | website=DailyMed | date=30 June 2020 | url=https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=a865e0ef-8685-4f69-8838-648c4f3bab47 | access-date=7 October 2020}}</ref> | legal_EU = | legal_EU_comment = | legal_UN =  | legal_UN_comment = | legal_status =

| C = 6430 | H = 9898 | N = 1718 | O = 2038 | S = 46 }}

'''Emapalumab''', sold under the brand name '''Gamifant''', is an anti-interferon-gamma (IFNγ) antibody medication used for the treatment of hemophagocytic lymphohistiocytosis (HLH),<ref name="FDA emapalumab">{{cite web | title=FDA approves emapalumab for hemophagocytic lymphohistiocytosis | website=U.S. Food and Drug Administration (FDA) | date=20 November 2018 | url=https://www.fda.gov/drugs/fda-approves-emapalumab-hemophagocytic-lymphohistiocytosis | access-date=19 March 2021}}{{dead link|date=May 2025|bot=medic}}{{cbignore|bot=medic}} {{PD-notice}}</ref><ref name="BW Gamifant">{{cite press release |title=FDA Approves Gamifant (emapalumab-lzsg), the First and Only Treatment Indicated for Primary Hemophagocytic Lymphohistiocytosis (HLH) |date=20 November 2018 |url=https://www.businesswire.com/news/home/20181120005454/en/FDA-Approves-Gamifant%C2%AE-emapalumab-lzsg-Treatment-Primary-Hemophagocytic |via=Business Wire |publisher=Sobi |access-date=21 November 2018 }}</ref><ref name="pmid32648854">{{cite journal | vauthors = Cheloff AZ, Al-Samkari H | title = Emapalumab for the treatment of hemophagocytic lymphohistiocytosis | journal = Drugs of Today | volume = 56 | issue = 7 | pages = 439–446 | date = July 2020 | pmid = 32648854 | doi = 10.1358/dot.2020.56.7.3145359 | s2cid = 220473902 }}</ref> which has no cure.<ref>{{ClinicalTrialsGov|NCT02069899|Long-term Follow-up of HLH Patients Who Received Treatment With NI-0501, an Anti-interferon Gamma Monoclonal Antibody}}</ref>

The most common side effects include infections, hypertension, infusion-related reactions, and pyrexia.<ref name="FDA emapalumab" />

The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.<ref>{{cite report | title=New Drug Therapy Approvals 2018 | website=U.S. Food and Drug Administration (FDA) | date=January 2019 | url=https://www.fda.gov/media/120357/download | archive-url=https://web.archive.org/web/20190826114431/https://www.fda.gov/media/120357/download | url-status=dead | archive-date=26 August 2019 | format=PDF | access-date=16 September 2020}}</ref>

In June 2025, the U.S. Food and Drug Administration (FDA) approved emapalumab-lzsg for the treatment of macrophage activation syndrome (MAS) in patients with Still’s disease.<ref name=":0">{{Cite web |date=2025-06-30 |title=FDA approves Sobi’s Gamifant to treat severe inflammatory arthritis complication - PMLiVE |url=https://pmlive.com/pharma_news/fda-approves-sobis-gamifant-to-treat-severe-inflammatory-arthritis-complication/ |access-date=2025-06-30 |website=pmlive.com |language=en-GB}}</ref>

== Medical uses == Emapalumab is used to treat primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy.<ref name="FDA emapalumab" />

==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%).<ref name="Gamifant FDA label" /><ref name="RDR">{{cite web |title=Emapalumab Approved for Rare Primary Hemophagocytic Lymphohistiocytosis |url=https://www.raredr.com/news/emapalumab-approved-for-rare-primary-hemophagocytic-lymphohistiocytosis |website=Rare Disease Report |access-date=22 November 2018 |archive-date=22 November 2018 |archive-url=https://web.archive.org/web/20181122132019/https://www.raredr.com/news/emapalumab-approved-for-rare-primary-hemophagocytic-lymphohistiocytosis |url-status=dead }}</ref> Serious adverse effects occurred in about half of the subjects studied in the clinical trial that led to its FDA approval.<ref name="RDR"/>

==Pharmacology== ===Mechanism of action=== In the setting of HLH, over-secretion of IFN-γ is thought to contribute to the pathogenesis of the disease.<ref name="Gamifant FDA label" /> Emapalumab binds and neutralizes IFN-γ, preventing it from inducing pathological effects.<ref name="Gamifant FDA label" />

===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.<ref name="Gamifant FDA label" />

==Society and culture== === Legal status === The U.S. Food and Drug Administration (FDA) granted orphan drug designations in 2010 and 2020,<ref>{{cite web | title=Emapalumab Orphan Drug Designations and Approvals | website=U.S. Food and Drug Administration (FDA) | date=25 September 2020 | url=https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=766720 | archive-url=https://web.archive.org/web/20231215122942/https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=766720 | url-status=dead | archive-date=15 December 2023 | access-date=19 March 2021}}</ref><ref>{{cite web | title=Emapalumab Orphan Drug Designations and Approvals | website=U.S. Food and Drug Administration (FDA) | date=26 March 2010 | url=https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=302910 | archive-url=https://web.archive.org/web/20231211193853/https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=302910 | url-status=dead | archive-date=11 December 2023 | access-date=19 March 2021}}</ref> and breakthrough therapy designation in 2016, on the basis of preliminary data from the phase II trial.<ref>{{cite web|url=http://www.fiercebiotech.com/biotech/novimmune-s-ni-0501-granted-breakthrough-therapy-designation-by-us-fda-for-treatment-of|title=Novimmune's NI-0501 Granted Breakthrough Therapy Designation by US FDA for Treatment of Patients With Primary Hemophagocytic Lymphohistiocytosis (HLH) - FierceBiotech|website=www.fiercebiotech.com|date=16 March 2016}}</ref>

In July 2020, and again in November 2020, the European Medicines Agency (EMA) recommended the refusal of the marketing authorization for emapalumab.<ref>{{cite web | title=Gamifant: Pending EC decision | website=European Medicines Agency | date=24 July 2020 | url=https://www.ema.europa.eu/en/medicines/human/summaries-opinion/gamifant | access-date=21 September 2020 | archive-date=23 September 2020 | archive-url=https://web.archive.org/web/20200923013240/https://www.ema.europa.eu/en/medicines/human/summaries-opinion/gamifant | url-status=dead }}</ref><ref>{{cite web | title=Gamifant EPAR | website=European Medicines Agency (EMA) | date=21 July 2020 | url=https://www.ema.europa.eu/en/medicines/human/EPAR/gamifant | access-date=19 March 2021}}</ref>

In June 2025, the FDA approved emapalumab-lzsg for the treatment of macrophage activation syndrome (MAS) in patients with Still’s disease.<ref name=":0" />

==Research== The research name of emapalumab was NI-0501.<ref name="SearchUSAN" /> A phase II/III trial began in 2013 and is ongoing {{as of|2018|August|lc=y}}.<ref>{{ClinicalTrialsGov|NCT01818492|A Study to Investigate the Safety and Efficacy of an Anti-IFNγ mAb in Children Affected by Primary Haemophagocytic Lymphohistiocytosis}}</ref> The trial targets patients under the age of 18 who have failed to improve on conventional treatments.<ref>{{cite web|url=https://www.cincinnatichildrens.org/service/c/clinical-trials/studies/ni-0501-study|title=NI-0501: A Study to Investigate the Safety and Efficacy of an Anti-IFN? mAb in Children Affected by Primary Hemophagocytic Lymphohistiocytosis|website=www.cincinnatichildrens.org}}</ref> This study was realised in the context of an EU-funded FP7 project, named FIGHT-HLH (306124).{{Citation needed|date=November 2018}}

== References == {{reflist}}

==External links== * {{ClinicalTrialsGov|NCT01818492|A Study to Investigate the Safety and Efficacy of an Anti-IFNγ mAb in Children Affected by Primary Haemophagocytic Lymphohistiocytosis}}

Category:Experimental monoclonal antibodies Category:Orphan drugs