FDA rejects Corcept’s relacorilant, citing need for more effectiveness data

Corcept Therapeutics (NASDAQ:CORT) shares plunged Wednesday after the U.S. Food and Drug Administration (FDA) rejected its New Drug Application (NDA) for relacorilant, a treatment for hypertension caused by excessive cortisol levels.

The FDA’s Complete Response Letter (CRL) acknowledged that Corcept's pivotal GRACE trial met its primary endpoint—showing that relacorilant significantly improved blood pressure control compared to a placebo.

The Agency also noted that the GRADIENT trial provided confirmatory evidence.

However, regulators concluded they could not approve the drug without "additional evidence of effectiveness," effectively stalling the company's effort to move beyond its sole commercial product, Korlym.

Relacorilant is a selective glucocorticoid receptor (GR) antagonist designed to modulate the activity of cortisol, the "stress hormone," without the off-target effects of earlier therapies.

Unlike Korlym (mifepristone), which binds to progesterone receptors and can cause endometrial thickening or irregular bleeding, relacorilant was designed to be highly specific.

The rejection is a major blow to the Redwood City-based biotech, which had been banking on relacorilant to diversify its revenue stream as Korlym faces potential generic competition in the coming years.

Investors reacted sharply to the news; the stock, which had been under pressure leading up to the December 30 PDUFA date, fell more than 20% in morning trading.

Despite the setback in endocrinology, Corcept is continuing to advance relacorilant in other indications.

The company recently filed a separate NDA for the drug in platinum-resistant ovarian cancer, with a target action date set for July 11, 2026.

Corcept remains in a strong financial position with over $500 million in cash, which analysts say should provide enough runway to conduct any additional trials the FDA may require for the hypertension indication.