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Hemophagocytic lymphohistiocytosis

Disease ID:416
Name:Hemophagocytic lymphohistiocytosis
Associated with:1 target
1 immuno-relevant ligand
Synonyms
Hemophagocytic syndrome
Database Links
Disease Ontology: DOID:0050120
OMIM: 603552
Orphanet: ORPHA158032

Targets

TRPM3
Comments:  TRPM3 is a candidate gene for this disease.
References:  3-4

Ligands

Key to terms and symbols Click ligand name to view ligand summary Click column headers to sort
Ligand References Clinical and Disease comments
emapalumab 1
Immuno Disease Comments: Approved therapy for HLH in adult and pediatric patients (FDA 2018).
Clinical Use: Emapalumab initially received FDA orphan drug designation for the treatment of hemophagocytic lymphohistiocytosis (HLH). Link here to the FDA record for this designation.
Phase 2/3 clinical trial NCT01818492 evaluated emapalumab (NI-0501) in children with primary HLH, and a long-term follow-up study of HLH patients who were given emapalumab in an earlier clinical trial is ongoing (see NCT02069899). In November 2018, the FDA granted full approval for emapalumab's use as a treament option for adult and pediatric (newborn and older) patients with refractory, recurrent or progressive primary HLH or who are intolerant to conventional HLH therapy. | View clinical data
Bioactivity Comments: IFNγ binding and neutralisation by NI-0501 and results of biological function studies are provided in patent WO2006109191 [2]. | View biological activity

References

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1. FDA. FDA approves emapalumab for hemophagocytic lymphohistiocytosis. Accessed on 21/11/2018. Modified on 21/11/2018. https://www.fda.gov, https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm626280.htm?utm_campaign=Oncology%2011%2F20%2F2018%20emapalumab%20%28GAMIFANT&utm_medium=email&utm_source=Eloqua&elqTrackId=87dd737d161843d8a913014df8c207b1&elq=e82c83f7f52f4e0aad9b0d385665ebb3&elqaid=5965&elqat=1&elqCampaignId=4818

2. Ferlin W, Fischer N, Elson G, Leger O. (2006) Human anti-interferon gamma antibodies and methods of use thereof. Patent number: WO2006109191. Assignee: Novimmune S.A.. Priority date: 27/01/2005. Publication date: 19/10/2006.

3. Lee N, Chen J, Sun L, Wu S, Gray KR, Rich A, Huang M, Lin JH, Feder JN, Janovitz EB, Levesque PC, Blanar MA. (2003) Expression and characterization of human transient receptor potential melastatin 3 (hTRPM3). J. Biol. Chem., 278 (23): 20890-7. [PMID:12672827]

4. Nilius B, Owsianik G, Voets T, Peters JA. (2007) Transient receptor potential cation channels in disease. Physiol. Rev., 87 (1): 165-217. [PMID:17237345]