Emapalumab, sold under the brand name Gamifant, is an anti-interferon-gamma (IFNγ) antibody medication used for the treatment of hemophagocytic lymphohistiocytosis (HLH), which currently has no cure. Hemophagocytic lymphohistiocytosis (HLH) is a severe systemic inflammatory syndrome that can be fatal. This syndrome can sometimes occur in normal people with medical problems that can cause a strong activation of the immune system, such as infection or cancer. HLH in these settings is called Secondary HLH. Patients with secondary HLH may also have medical conditions which contribute to the predisposition to HLH, such as rheumatologic diseases or primary immune deficiencies.
HLH occurs in other patients because they have specific genetic defects which more directly cause HLH, in which case it is called Primary HLH. Primary HLH is also sometimes called Familial HLH. The genetic mutations that cause Primary HLH are present at birth, and patients often become ill with HLH in the first few years of life. If not detected and treated, primary HLH is usually fatal, typically within a few months. Even with treatment the prognosis is sometimes only a few years, unless a bone marrow transplant can be successfully performed. If secondary HLH is detected promptly and treated aggressively, the prognosis may be better.
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