FDA CRL - Corcept Therapeutics Incorporated

Corcept Therapeutics received a Corrected Complete Response Letter from the FDA regarding New Drug Application 219398 for relacorilant capsules, a treatment for endogenous Cushing’s syndrome and hypercortisolism. The FDA determined the application cannot be approved in its current form due to two primary clinical deficiencies. First, the agency found insufficient evidence of effectiveness; one Phase 3 trial (CORT125134-455) yielded unreliable results due to a highly enriched patient population and significant missing data, while a second trial (CORT125134-456) failed to meet its primary efficacy endpoint. Second, the FDA raised significant safety concerns regarding drug-induced liver injury (DILI), noting four probable cases including one severe incident of liver enzyme elevation among a small trial population.

While the document does not list specific facility inspection dates, it details clinical trial reviews conducted under the regulatory framework of 21 CFR 314.110. To address these issues, Corcept must provide new clinical data demonstrating meaningful efficacy and prove that the drug's benefits outweigh its risks. Additionally, the company must submit a risk mitigation strategy for liver injury, including proposed labeling warnings and enhanced monitoring. Corcept has one year to resubmit the application with the required data or request an extension.