The U.S. Food and Drug Administration (FDA) ended 2025 by issuing complete response letters to Corcept Therapeutics and Outlook Therapeutics, leading to significant declines in both companies' stock prices.
Corcept Therapeutics had sought FDA approval for relacorilant, intended for patients with high blood pressure due to hypercortisolism, also known as Cushing’s syndrome. The company reported that while one of its two Phase III trials met the primary endpoint, the other did not show a statistically significant benefit over placebo. The FDA acknowledged these results but concluded that more evidence is needed to demonstrate relacorilant’s effectiveness and establish a favorable benefit-risk profile for the drug.
Truist Securities analysts noted in a communication to investors that the FDA's request for additional data "may require additional trial(s), significantly dimming [Corcept’s] outlook in Cushing’s." They also lowered their price target for Corcept from $135 to $50 after shares dropped 50% to $34.80 at market close on December 31. However, they indicated that the rejection does not affect the ongoing FDA review of relacorilant for ovarian cancer, which is expected to conclude by July 11.
Corcept CEO Joseph Belanoff commented, “confident we will find a way to get relacorilant to the patients it could help,” adding that the company plans to meet with the FDA soon.
Outlook Therapeutics also received an FDA rejection for its ophthalmic formulation of bevacizumab intended for wet age-related macular degeneration (AMD). This marks the third time Outlook has been denied approval for this indication. Although Roche originally developed Avastin (bevacizumab) as a cancer therapy, physicians have used it off-label in eye diseases such as wet AMD.
Outlook had previously been rejected by the FDA in August due to insufficient evidence of effectiveness. In its resubmission, Outlook included new mechanistic and natural history data along with results from a pivotal trial and confirmatory studies, but the agency again requested more evidence supporting efficacy. Following this latest decision, Outlook’s stock fell 61% to 62 cents in premarket trading on January 2.
Outlook CEO Bob Jahr stated that the company remains “fully committed to taking all necessary steps to receive approval in the United States.” He added that Outlook is now exploring all available pathways toward potential approval. The drug has already received approval in Europe under the name Lytenava.
These rejections followed another recent complete response letter issued by the FDA regarding Sanofi’s multiple sclerosis candidate, tolebrutinib. Houman Ashrafian, head of R&D at Sanofi, said: “the FDA’s decision is a significant and meaningful change in direction from the feedback the agency previously provided.”
