The Food and Drug Administration (FDA) has granted Orphan Drug designation to dusquetide (SGX942; Soligenix) for the treatment of macrophage activation syndrome (MAS), a life-threatening complication of rheumatic disease.

MAS occurs much more frequently in patients with systemic juvenile idiopathic arthritis (SJIA), but can also be seen in systemic lupus erythematosus (SLE), Kawasaki disease, adult-onset Still’s disease, and other vasculitic syndromes. It is characterized by pancytopenia, liver insufficiency, coagulopathy and neurologic symptoms due to widespread hemophagocytosis and cytokine overproduction caused by the activation and uncontrolled proliferation of T lymphocytes and well-differentiated macrophages. Although treatment options exist, the mortality rate for MAS is about 10–20%. 

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The FDA designation for MAS was supported by preclinical efficacy and safety data in animal models, showing that dusquetide reduces pancytopenia, IL-12 responses, and improves body weight maintenance.

Dusquetide is a novel innate defense regulator (IDR) which acts to modulate the immune system, thereby accelerating bacterial clearance, resolving tissue damage and controlling inflammation following exposure to bacterial pathogens, trauma, radiation and/or chemotherapy.

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