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FDA Approves Gamifant for Primary Hemophagocytic Lymphohistiocytosis

First and Only Treatment for Rare Hyper-Inflammation Syndrome With High Mortality

The FDA has approved Gamifant (emapalumab-lzsg, Sobi and NovImmune SA), an interferon gamma (IFNγ) blocking antibody for the treatment of pediatric and adult patients who have primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or an intolerance to conventional therapy.

Primary HLH is an extremely rare, rapidly progressive, and often-fatal syndrome in which massive overexpression of IFNy is believed to drive immune system hyperactivation, which ultimately leads to organ failure.

Diagnosis of primary HLH is challenging––it’s estimated that fewer than 100 cases are diagnosed each year in the U.S.––due to the variability in signs and symptoms. These may include fevers, liver and spleen swelling, extremely low blood cell counts, bleeding disorders, infections, neurological symptoms, and organ dysfunction and failure. Symptoms usually begin to appear during the first year of life and can quickly become lethal if untreated. The median survival rate is less than two months.

The immediate goal of treatment is to get the hyperinflammation under control as quickly as possible and to prepare for hematopoietic stem cell transplantation, which is the sole cure. Emapalumab, a monoclonal antibody (mAB) that binds to and neutralizes IFNy, offers a wholly new approach to treating primary HLH and could enable seriously ill patients to obtain hematopoietic stem cell transplantation. Emapalumab is expected to be available in the first quarter of 2019.

Source:, November 20, 2018.

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