Emapalumab-lzsg (Gamifant) got approval by the U.S. FDA for the treatment of refractory, recurrent, or progressive form of primary hemophagocytic lymphohistiocytosis in pediatric and adult patients. It also got approval for the cases that experience intolerance to conventional HLH therapy. The interferon gamma blocking antibody is the first approved treatment for this exceedingly rare medical condition that leads to excessive inflammation. Emapalumab grabs the coveted feat of being the first agent targeted to neutralize interferon gamma.
Michael Jordan, MD, a physician-scientist in the division of Bone Marrow Transplantation and Immune Deficiency at Cincinnati Children's Hospital Medical Center HLH Center of Excellence, who served as the primary investigator in the emapalumab clinical trial expressed his satisfaction at this approval. He said until this approval, there was no pharmacological agent that could specifically fulfill the objective in spite of the understanding of pivotal role of interferon gamma in this disorder. He sounded hopeful with the approval of Emapalumab as it represented a novel approach to treat primary HLH with potential to help these sick patients reach hematopoietic stem cell transplant.
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