|Other names||NI-0501, emapalumab-lzsg|
|Chemical and physical data|
|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), which has no cure.
The most common side effects include infections, hypertension, infusion-related reactions, and pyrexia.
Emapalumab is used to treat primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy.
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%). Serious adverse effects occurred in about half of the subjects studied in the clinical trial that led to its FDA approval.
Mechanism of action
In the setting of HLH, over-secretion of IFN-γ is thought to contribute to the pathogenesis of the disease. Emapalumab binds and neutralizes IFN-γ, preventing it from inducing pathological effects.
Society and culture
The U.S. Food and Drug Administration (FDA) granted orphan drug designations in 2010 and 2020, and breakthrough therapy designation in 2016, on the basis of preliminary data from the phase II trial.
The research name of emapalumab was NI-0501. A phase II/III trial began in 2013 and is ongoing as of August 2018[update]. The trial targets patients under the age of 18 who have failed to improve on conventional treatments. This study was realised in the context of an EU-funded FP7 project, named FIGHT-HLH (306124).
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- 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.
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- "Gamifant: Pending EC decision". European Medicines Agency. 24 July 2020. Retrieved 21 September 2020.
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- 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.