On November 20, 2018, the U.S. Food and Drug Administration approved Gamifant (emapalumab-lzsg) for the treatment of pediatric (newborn and above) and adult patients with primary hemophagocytic lymphohistiocytosis (HLH) who have refractory, recurrent or progressive disease or intolerance to conventional HLH therapy1,2. This is the first FDA approval for a drug specifically for HLH.
The approval is based on data from pivotal phase 2/3 study which enrolled patients with primary HLH. The efficacy of Gamifant was studied in a clinical trial of 27 pediatric patients with suspected or confirmed primary HLH with either refractory, recurrent or progressive disease during conventional HLH therapy or who were intolerant of conventional HLH therapy. The median age of the patients in the trial was 1 year old. The study showed that 63 percent of patients experienced a response and 70 percent were able to proceed to stem cell transplant.
The FDA granted Priority Review, Breakthrough Therapy designation and also Orphan Drug designation to Gamifant earlier. The FDA approval of Gamifant is granted to Novimmune SA.
About HLH
HLH is a condition in which the body's immune cells do not work properly. The cells become overactive releasing molecules, which leads to inflammation. The immune cells start to damage the body's own organs, including the liver, brain and bone marrow. It can be inherited, which is known as primary or "familial" HLH. It can also have non-inherited causes. People with primary HLH usually develop symptoms within the first months or years of life. Symptoms may include fever, enlarged liver or spleen and decreased number of blood cells.
About Gamifant (emapalumab)
Emapalumab is a monoclonal antibody (mAb) that binds and neutralises interferon gamma (IFNy). In the US, Gamifant is indicated for paediatric (newborn and older) and adult primary haemophagocytic lymphohistiocytosis (HLH) patients with refractory, recurrent or progressive disease, or intolerance to standard-of-care HLH therapy. Gamifant is the first and only medicine approved in the US for primary HLH, an ultra-rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. Gamifant is indicated to be administered through intravenous (IV) infusion over one hour twice per week until haematopoietic stem cell transplant (HSCT).
Note - Gamifant is developed and submitted for approval to the USFDA by Nonimmune, and now Sobi will commercialise the Gamifant globally.
Footnotes
1 https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm626263.htm
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