SCO-483 is a promising drug candidate designed to treat Congenital Adrenal Hyperplasia (CAH) and Cushing’s disease. Its mechanism of action remains undisclosed. The drug is intended to be administered as a daily oral dose and a long-acting injection. Preliminary toxicology studies indicate that SCO-483 has a favorable safety profile, and preparations for the initiation of clinical trials are currently underway.
CAH is a group of inherited disorders that affect the adrenal glands. Approximately 95% of CAH cases are caused by a genetic mutation leading to a deficiency of the adrenal 21-hydroxylase (21-OHD). This enzyme is essential for the adrenal glands to produce cortisol and aldosterone, hormones that regulate stress responses and maintain blood pressure and salt balance. Individuals with a mutated 21-OHD gene experience hormonal imbalances, which can lead to metabolic issues and an excess of androgens. Without appropriate treatment, CAH can result in various symptoms, including adrenal insufficiency, excessive male hormones, and abnormalities in external genitalia in females. Currently, there is a significant unmet medical need as no approved non-steroidal drugs specifically designed for CAH exist.
Cushing’s disease is a rare endocrine disorder characterized by the overproduction of cortisol. This can occur when the pituitary gland overproduces adrenocorticotropin, which in turn causes the adrenal glands to produce excess cortisol. Symptoms include weight gain, memory difficulties, irritability, muscle weakness, hypertension, bone loss, and type 2 diabetes. Treatments aim to reduce cortisol levels and alleviate symptoms. Despite advancements in treating Cushing’s disease, there is a significant unmet medical need for new, more effective, and safer drugs with a novel mechanism of action.
Scientific Publication
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S-483, a Pre-clinical Program for 21-Hydroxylase Deficiency, Was Selected as a Support Program of AMED
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