Dusquetide Granted Orphan Drug Status to Treat Life-Threatening Immune Disorder

The U.S. Food and Drug Administration (FDA) granted orphan drug designation to dusquetide (SGX942, Soligenix) for the treatment of macrophage activation syndrome (MAS), a life-threatening complication of rheumatic disease. MAS 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. MAS occurs much more frequently in patients with systemic juvenile idiopathic arthritis, but is also seen in systemic lupus erythematosus, Kawasaki disease, adult-onset Still’s disease and other vasculitis syndromes, with a mortality rate of approximately 10 percent to 20 percent.

Dusquetide is a novel innate defense regulator that acts to modulate the immune system to accelerate bacterial clearance, resolving tissue damage and controlling inflammation following exposure to bacterial pathogens, trauma, radiation and/or chemotherapy. The FDA designation was given after preclinical efficacy and safety data in animal models showed that dusquetide reduces pancytopenia and IL-12 responses and improves body weight maintenance.