Corcept Therapeutics submits FDA application for relacorilant to treat Cushing’s syndrome

Corcept Therapeutics Incorporated has taken a significant step in addressing the unmet needs of patients with hypercortisolism, commonly known as Cushing’s syndrome. The biopharmaceutical company has formally submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for relacorilant, its proprietary selective cortisol modulator. This development signals hope for a safer and more effective treatment option in a complex therapeutic landscape.

Why Relacorilant Represents a Breakthrough in Hypercortisolism Care

Relacorilant’s innovative approach distinguishes it from existing hypercortisolism therapies. Designed to selectively bind to the glucocorticoid receptor without interacting with other hormone receptors, the drug reduces cortisol’s harmful effects without triggering adverse side effects often associated with current treatments. Unlike some therapies, relacorilant avoids issues such as adrenal insufficiency, hypokalemia, and QT prolongation, which can significantly complicate patient outcomes.

In pivotal clinical trials, including the GRACE and GRADIENT studies, as well as in long-term extension and Phase 2 trials, relacorilant demonstrated its efficacy by improving a range of hypercortisolism symptoms. Patients reported better management of hypertension, weight gain, and glucose tolerance—hallmarks of the disease—without severe safety concerns.

What Sets Relacorilant Apart

Corcept Therapeutics has emphasised relacorilant’s safety profile as a critical factor in its potential approval. The absence of drug-induced adrenal insufficiency, a serious risk associated with existing treatments, highlights the drug’s precision. Additionally, relacorilant avoids complications linked to progesterone receptor activity, such as endometrial thickening and vaginal bleeding, making it a promising candidate for broader adoption.

Joseph Belanoff, MD, Corcept’s Chief Executive Officer, described the drug’s combination of efficacy and safety as a significant advancement, expressing optimism that it could become the standard of care for hypercortisolism patients. The company has framed its FDA submission as a culmination of years of focused research into cortisol modulation.

Hypercortisolism Treatment: The Unmet Need

Cushing’s syndrome, or endogenous hypercortisolism, is caused by prolonged exposure to elevated cortisol levels, often resulting in life-threatening complications such as diabetes, cardiovascular disease, and infections. Despite the availability of therapies, many patients experience limited efficacy or significant side effects, underscoring the urgent need for alternatives.

Relacorilant’s precision in targeting glucocorticoid receptors offers a solution that aligns with the physiological complexities of the condition, providing hope to patients for better disease management with fewer risks.

The Broader Potential of Relacorilant

Beyond hypercortisolism, Corcept is exploring relacorilant as a treatment for various cancers, including ovarian, adrenal, and prostate cancers, where abnormal cortisol activity contributes to disease progression. With orphan drug designation in both the United States and the European Union for Cushing’s syndrome, relacorilant is positioned to make a significant impact in multiple therapeutic areas.

What Lies Ahead

The FDA’s review of Corcept’s NDA will determine whether relacorilant becomes an approved treatment for hypercortisolism. If approved, the drug could revolutionise the standard of care, offering patients a more targeted, safer, and effective option. This milestone also solidifies Corcept Therapeutics’ reputation as a leader in the development of cortisol-modulating therapies for complex diseases.

Expert Insights and Industry Implications

Relacorilant’s potential approval could reshape how hypercortisolism is treated globally. The drug’s development reflects a growing emphasis on precision medicine, where treatments are tailored to address specific pathways without causing collateral harm. Experts suggest that relacorilant’s safety and efficacy profile could encourage further investment and innovation in cortisol modulation research.

Corcept’s focus on hypercortisolism also highlights the company’s strategic commitment to addressing underserved conditions, particularly those where current options leave significant gaps.