The US 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%. 

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.

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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.

Dusquetide was shown to be safe in a Phase 1 clinical study in 84 healthy human volunteers.  More recently, it has been studied in an exploratory Phase 2 clinical study with 111 patients with oral mucositis due to chemoradiation (CRT) therapy for head and neck cancer. 

Doses of 1.5 mg/kg demonstrated positive improvements in decreasing the duration of severe oral mucositis by 50% overall compared to the placebo group, from 18 days to 9 days (P=.099).  In patients at highest risk of oral mucositis, the reduction in the duration of severe oral mucositis was even more significant at 67% when treated with dusquetide 1.5 mg/kg, from 30 days to 10 days (P=.04).  


FDA Grants Soligenix Orphan Drug Designation for Dusquetide for Treatment of Macrophage Activation Syndrome. Soligenix press release. Published online August 18, 2016. Accessed August 20, 2016. 

This article originally appeared on MPR