Information about Emapalumab
Emapalumab is a human monoclonal antibody to interferon gamma which acts to block its binding to cell surface receptors and activation of inflammatory signals.
Clinical use of Emapalumab
Emapalumab is used to treat the severe inflammatory condition of hemophagocytic lymphohistiocytosis (HLH) in which serum gamma interferon levels are elevated.
Liver safety of Emapalumab
Emapalumab therapy is associated with mild and transient serum enzyme elevations during therapy, but has not been linked to instances of clinically apparent acute liver injury.
Mechanism of action of Emapalumab
Emapalumab (em" a pal' ue mab) is a recombinant, human IgG1 monoclonal antibody to gamma interferon, which inhibits its binding to cell surface interferon receptors and the subsequent activation of intracellular proinflammatory signaling pathways. Gamma interferon levels are known to be elevated in patients with hemophagocytic lymphohistiocytosis (HLH), a life-threatening inflammatory syndrome marked by fever, enlargement of liver and spleen, macrophage update of red blood cells (hemophagocytosis) in bone marrow, spleen and lymph nodes, low natural killer cell activity and high levels of serum ferritin, triglycerides and multiple cytokines. HLH has primary genetic causes but can also arise secondarily as a complication of viral infections, cancer, severe rheumatic diseases and immunosuppression after transplantation. In clinical trials, emapalumab was found to reduce hemolysis and the need for blood transfusions with subsequent improvement in symptoms and quality of life in children and adults with recurrent HLH.
FDA approval information for Emapalumab
Emapalumab was approved for use in HLH in the United States in 2018.
Dosage and administration for Emapalumab
Emapalumab is available as a solution in single dose vials of 10 mg in 2 mL or 50 mg in 10 mL (5 mg/mL) under the commercial name Gamifant. The recommended starting dose is 1 mg/kg as an intravenous infusion twice per week. Doses can be modified based upon clinical and laboratory results and therapy continued until hematopoietic cell transplantation, or unacceptable toxicity or until it is no longer thought to be needed. Premedication with dexamethasone is recommended as is prophylaxis for herpes zoster, Pneumocystis jirovecii and fungal infections.
Side effects of Emapalumab
Side effects can include infusion site reactions, infections, fever and hypertension. Uncommon severe complications include severe infusion reactions and severe infections, including reactivation of tuberculosis, herpes zoster, pneumocystis jirovecii and fungal infections. Generally, however, the symptoms and complications of the underlying HLH overshadow the adverse effects of inhibition of gamma interferon.
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