Emapalumab


Emapalumab, marketed under the trade name Gamifant, is an anti-interferon-gamma antibody used for the treatment of hemophagocytic lymphohistiocytosis, which currently has no cure.

Adverse effects

In the clinical trials that lead to emapalumab's FDA approval, the most commonly reported adverse effects were infections, high blood pressure, infusion reactions, and fever. Serious adverse effects occurred in about half of the subjects studied in the clinical trial that led to its FDA approval.

Pharmacology

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.

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.

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.

Research

The research name of emapalumab was NI-0501. A phase II/III trial began in 2013 and is ongoing as of 2018. 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.