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The US Food and Drug Administration (FDA) has granted orphan drug designation for dusquetide as a treatment for macrophage activation syndrome (MAS).
Dusquetide is an innate defense regulator, a new class of short, synthetic peptides that accelerate bacterial clearance and resolution of tissue damage while modulating inflammation following exposure to bacterial pathogens, radiation, chemotherapy, and other agents.
According to researchers, dusquetide has demonstrated preclinical efficacy and safety in several animal models.
In a mouse model of MAS, dusquetide was shown to reduce pancytopenia, inhibit IL-12 responses, and improve body weight maintenance.
SGX942, the drug product containing dusquetide, has demonstrated safety in a phase 1 study of 84 healthy volunteers.
In addition, SGX942 has demonstrated preliminary efficacy and safety in an exploratory phase 2 study of 111 patients with oral mucositis due to chemoradiation therapy for head and neck cancer.
SGX942 is being developed by Solgenix, Inc.
About orphan designation
The FDA grants orphan designation to drugs and biologics intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.
The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the drug is approved.
About MAS
MAS is a life-threatening complication of rheumatic disease that, for unknown reasons, frequently occurs in individuals with systemic juvenile idiopathic arthritis. MAS also occurs in patients with systemic lupus erythematosus, Kawasaki disease, adult-onset Still’s disease, and various vasculitic syndromes.
MAS is characterized by pancytopenia, liver insufficiency, coagulopathy, and neurologic symptoms.
MAS is thought to be caused by the activation and uncontrolled proliferation of T lymphocytes and well-differentiated macrophages, leading to widespread hemophagocytosis and cytokine overproduction. 
Image from Flickr
The US Food and Drug Administration (FDA) has granted orphan drug designation for dusquetide as a treatment for macrophage activation syndrome (MAS).
Dusquetide is an innate defense regulator, a new class of short, synthetic peptides that accelerate bacterial clearance and resolution of tissue damage while modulating inflammation following exposure to bacterial pathogens, radiation, chemotherapy, and other agents.
According to researchers, dusquetide has demonstrated preclinical efficacy and safety in several animal models.
In a mouse model of MAS, dusquetide was shown to reduce pancytopenia, inhibit IL-12 responses, and improve body weight maintenance.
SGX942, the drug product containing dusquetide, has demonstrated safety in a phase 1 study of 84 healthy volunteers.
In addition, SGX942 has demonstrated preliminary efficacy and safety in an exploratory phase 2 study of 111 patients with oral mucositis due to chemoradiation therapy for head and neck cancer.
SGX942 is being developed by Solgenix, Inc.
About orphan designation
The FDA grants orphan designation to drugs and biologics intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.
The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the drug is approved.
About MAS
MAS is a life-threatening complication of rheumatic disease that, for unknown reasons, frequently occurs in individuals with systemic juvenile idiopathic arthritis. MAS also occurs in patients with systemic lupus erythematosus, Kawasaki disease, adult-onset Still’s disease, and various vasculitic syndromes.
MAS is characterized by pancytopenia, liver insufficiency, coagulopathy, and neurologic symptoms.
MAS is thought to be caused by the activation and uncontrolled proliferation of T lymphocytes and well-differentiated macrophages, leading to widespread hemophagocytosis and cytokine overproduction.
Image from Flickr
The US Food and Drug Administration (FDA) has granted orphan drug designation for dusquetide as a treatment for macrophage activation syndrome (MAS).
Dusquetide is an innate defense regulator, a new class of short, synthetic peptides that accelerate bacterial clearance and resolution of tissue damage while modulating inflammation following exposure to bacterial pathogens, radiation, chemotherapy, and other agents.
According to researchers, dusquetide has demonstrated preclinical efficacy and safety in several animal models.
In a mouse model of MAS, dusquetide was shown to reduce pancytopenia, inhibit IL-12 responses, and improve body weight maintenance.
SGX942, the drug product containing dusquetide, has demonstrated safety in a phase 1 study of 84 healthy volunteers.
In addition, SGX942 has demonstrated preliminary efficacy and safety in an exploratory phase 2 study of 111 patients with oral mucositis due to chemoradiation therapy for head and neck cancer.
SGX942 is being developed by Solgenix, Inc.
About orphan designation
The FDA grants orphan designation to drugs and biologics intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.
The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the drug is approved.
About MAS
MAS is a life-threatening complication of rheumatic disease that, for unknown reasons, frequently occurs in individuals with systemic juvenile idiopathic arthritis. MAS also occurs in patients with systemic lupus erythematosus, Kawasaki disease, adult-onset Still’s disease, and various vasculitic syndromes.
MAS is characterized by pancytopenia, liver insufficiency, coagulopathy, and neurologic symptoms.
MAS is thought to be caused by the activation and uncontrolled proliferation of T lymphocytes and well-differentiated macrophages, leading to widespread hemophagocytosis and cytokine overproduction.