Corcept Applies for Relacorilant Approval to Treat Hypercortisolism

REDWOOD CITY, Calif.--Corcept Therapeutics Incorporated, a company at the forefront of developing treatments for severe disorders influenced by the hormone cortisol, has officially submitted a new drug application to the U.S. Food and Drug Administration (FDA). This application is for relacorilant, a selective cortisol modulator designed to aid patients suffering from endogenous hypercortisolism, commonly known as Cushing’s syndrome.

The new drug application is supported by encouraging results from several pivotal studies, including the GRACE trial, which is a critical component of the research. Additionally, supportive data comes from the Phase 3 GRADIENT study, its long-term extension studies, and a Phase 2 study specifically targeting hypercortisolism. Participants in these trials who were treated with relacorilant demonstrated significant improvements in various symptoms associated with hypercortisolism, maintaining a favorable safety profile. Importantly, none of the patients experienced drug-induced adrenal insufficiency, hypokalemia, or QT prolongation, which are adverse effects seen with current medications. Furthermore, there were no side effects linked to activity at the progesterone receptor, such as endometrial thickening or vaginal bleeding.

According to Joseph Belanoff, MD, the Chief Executive Officer of Corcept, relacorilant’s effectiveness and safety may enable it to become the primary treatment option for patients with hypercortisolism. He emphasized the company's dedication to improving the health of those affected by this condition and expressed optimism about relacorilant’s potential benefits.

Relacorilant operates as a selective cortisol modulator with the ability to bind to glucocorticoid receptors without engaging the body's other hormone receptors. This specificity is noteworthy as it allows the compound to be explored for treating various serious disorders beyond Cushing’s syndrome, including ovarian, adrenal, and prostate cancers. Corcept owns the proprietary rights to relacorilant, safeguarded by multiple patents, and the drug holds orphan drug designation in both the United States and the European Union for treating Cushing’s syndrome.

Cushing’s syndrome, or hypercortisolism, results from excessive cortisol activity, leading to a range of symptoms. Patients may experience hypertension, central obesity, elevated blood sugar, unmanaged type 2 diabetes, extreme fatigue, and muscle weakness. Psychological effects such as irritability, anxiety, and depression are also common, with the potential to impact every organ system. Without effective treatment, the condition can be fatal.

For over two decades, Corcept Therapeutics has concentrated on the modulation of cortisol, exploring its potential in treating various serious health conditions. This focus has led to the discovery of over a thousand proprietary selective cortisol modulators. The company is currently engaged in advanced clinical trials for conditions such as hypercortisolism, solid tumors, amyotrophic lateral sclerosis (ALS), and liver disease. Notably, in 2012, Corcept introduced Korlym®, the first FDA-approved treatment for endogenous hypercortisolism.

Corcept Therapeutics is headquartered in Redwood City, California, where it continues its pioneering work in the field of endocrine therapies.