U.S. regulators rejected Corcept Therapeutics’ bid to market Relacorilant for Cushing syndrome–related hypertension on Wednesday, citing insufficient evidence of effectiveness, a decision that erased about half the company’s market value.
The Food and Drug Administration declined to approve Relacorilant after determining it could not reach a favorable benefit-risk assessment without additional proof of efficacy, Corcept said in a statement. The decision surprised investors and analysts, who had expected approval based on mixed but promising late-stage trial results.
Shares of the Redwood City, California-based biotech closed at $34.51, down 50.8% from Tuesday’s close of $70.20. The selloff wiped out about $3.7 billion in market capitalization in a single session.
FDA Cites Insufficient Efficacy Despite One Successful Trial
Corcept said the FDA acknowledged that one Phase three study of Relacorilant in patients with hypercortisolism, also known as Cushing syndrome, met its primary endpoint. However, a second Phase three trial focused on patients with Cushing syndrome and hypertension failed to meet its main efficacy goal, although several secondary measures showed improvement.
“The FDA could not arrive at a favorable benefit-risk assessment for Relacorilant without Corcept providing additional evidence of effectiveness,” the company said. The language suggests regulators were primarily concerned about whether the drug lowered blood pressure consistently enough to justify approval.
Relacorilant is designed to block cortisol’s effects without the hormonal side effects associated with older therapies. Cushing syndrome is a rare disorder caused by prolonged exposure to high cortisol levels and can lead to severe complications, including diabetes, hypertension, and cardiovascular disease.
Stock Loses Half Its Value as Investors Reassess Growth Outlook
The rejection marked a sharp reversal for Corcept Therapeutics, which has long promoted Relacorilant as its next major growth driver. On an earnings call in November, Sean Maduck, president of the company’s endocrinology business, said Relacorilant could “generate $3 billion to $5 billion in annual revenue in hypercortisolism alone” within three to five years.
Analysts had also been bullish. FactSet estimates put Corcept’s 2025 revenue at $821 million, largely from its only marketed product, Korlym, which is used to control high blood sugar in certain Cushing patients. Consensus projections had Relacorilant sales in Cushing syndrome reaching $1.9 billion by 2028.
“The FDA decision was clearly a shock to the market,” said Piper Sandler analyst David Amsellem. “In our chat with management, it was clear that the crux of the rejection is that Relacorilant did not demonstrate enough efficacy to justify an approval.”
Company Pivots Focus to Cancer Uses as Analysts Weigh Rebound
Amsellem said the FDA appears to want Corcept to conduct another clinical study, a step that could delay a Cushing-related approval by several years. “Another study would set Relacorilant back a few years,” he wrote in a research note.
Despite the setback, some analysts see a path forward. Corcept is also testing Relacorilant in ovarian and endometrial cancers, areas with significant unmet medical need. The FDA is scheduled to decide by mid-July whether to approve the drug for platinum-resistant ovarian cancer.
Amsellem said sales in that cancer indication alone could exceed $1 billion annually and maintained an overweight rating on the stock. “We would argue that CORT shares can easily rebound and are still well-positioned longer-term,” he wrote.
Corcept Therapeutics did not provide guidance on whether it plans to immediately pursue another hypertension study but said it would work with regulators to determine next steps. For now, the company remains reliant on Korlym as it seeks to rebuild investor confidence after one of the steepest single-day declines in its history.
