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.The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication. Medical uses
Emapalumab is used to treat primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Adverse effects
In the clinical trials that lead to emapalumabs 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. 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 bodys normal catabolism. Society and culture
Legal status
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.In July 2020, and again in November 2020, the European Medicines Agency (EMA) recommended the refusal of the marketing authorization for emapalumab. Research
The research name of emapalumab was NI-0501. A phase II/III trial began in 2013 and is ongoing as of August 2018.
Acanthamoeba keratitis (AK) is a rare disease in which amoebae of the genus Acanthamoeba invade the clear portion of the front (cornea) of the eye. It affects roughly 100 people in the United States each year. Acanthamoeba are protozoa found nearly ubiquitously in soil and water and can cause infections of the skin, eyes, and central nervous system.Infection of the cornea by Acanthamoeba is difficult to treat with conventional medications, and AK may cause permanent visual impairment or blindness, due to damage to the cornea or through damage to other structures important to vision. Recently, AK has been recognized as an orphan disease and a funded project, orphan diseases Acanthamoeba keratitis (ODAK), has tested the effects of a diverse range drugs and biocides on AK. Pathogenesis
In the United States, Acanthamoeba keratitis is nearly always associated with soft contact lens use. Acanthamoeba spp. is most commonly introduced to the eye by contact lenses that have been exposed to the organism through the use of contaminated lens solution, using homemade saline-based solution or tap water, or from wearing contact lenses while bathing or swimming. However, it may also be introduced to the eye by exposure to soil or vegetation, or by trauma. In fact, the first case of Acanthamoeba keratitis described was due to ocular trauma. Once on the contact lens, Acanthamoeba is able to survive in the space between the contact lens and the surface of the eye.