FDA Reviews NDA for Relacorilant in Treating Ovarian Cancer

On July 14, 2025, Corcept Therapeutics announced the submission of a New Drug Application (NDA) for relacorilant, a novel treatment aimed at patients with platinum-resistant ovarian cancer, to the United States Food and Drug Administration (FDA). This submission is backed by promising results from the pivotal Phase 3 ROSELLA trial (NCT05257408), which demonstrated significant survival benefits when relacorilant was administered in conjunction with nab-paclitaxel compared to nab-paclitaxel alone.

The ROSELLA trial, a randomized study, enrolled patients diagnosed with epithelial ovarian, primary peritoneal, or fallopian tube cancer who exhibited disease progression within six months after the completion of platinum-based therapy. Participants were administered either relacorilant (150 mg orally) alongside intravenous nab-paclitaxel (80 mg/m² on days 1, 8, and 15 of each 28-day cycle) or nab-paclitaxel alone at a dose of 100 mg/m². The primary endpoints of the study were progression-free survival (PFS) and overall survival (OS), evaluated by a blinded independent central review.

Results indicated that the combination of relacorilant and nab-paclitaxel yielded a median PFS of 6.54 months (95% CI, 5.55–7.43) compared to 5.52 months (95% CI, 3.94–5.88) for the control group, resulting in a hazard ratio (HR) of 0.70 (P = 0.0076). Additionally, the six-month and twelve-month PFS rates were reported at 52% and 25% in the combination arm, as opposed to 42% and 13% in the control arm, respectively.

Dr. Joseph K. Belanoff, CEO of Corcept Therapeutics, remarked, "This submission is an important milestone for Corcept as we now have two new drug applications before the FDA: Relacorilant in combination with nab-paclitaxel as a treatment for people with platinum-resistant ovarian cancer and relacorilant as a treatment for patients with hypercortisolism." He emphasized the crucial need for improved treatment options for patients facing these challenging conditions.

At 50% OS maturity, the median OS for the combination group was reported at 15.97 months (95% CI, 13.47 to not reached), in contrast to 11.50 months (95% CI, 10.02–13.57) for the nab-paclitaxel monotherapy, yielding an HR of 0.69 (P = 0.0121). The twelve-month OS rates were 60% for the combination group versus 49% for the control group, suggesting a meaningful survival advantage.

The secondary endpoints of the trial further corroborated the efficacy of relacorilant, with the objective response rate reaching 36.9% in the combination group, including 3.2% complete responses, compared to 30.1% in the monotherapy group (2.1% complete responses). The clinical benefit rate was also favorable for the relacorilant group, standing at 51.1% versus 38.9% in the control group.

Despite these promising outcomes, safety concerns were flagged, with higher rates of treatment-emergent adverse events (TEAEs) reported in the combination arm. Grade 3 or higher TEAEs occurred in 74.5% of patients receiving relacorilant compared to 59.5% in the control group. Notably, the combination treatment necessitated more dose modifications, with 48.4% of patients requiring reductions in nab-paclitaxel doses compared to 31.6% in the control arm.

The findings from the ROSELLA trial and the subsequent NDA submission come at a time when effective treatment options for platinum-resistant ovarian cancer remain limited. The FDA's review of this NDA will be crucial in determining the availability of relacorilant as a potentially transformative therapeutic option for patients battling this challenging form of cancer. As noted by Dr. Olawaiye and colleagues in a study published in the Journal of Clinical Oncology, establishing effective treatments for ovarian cancer remains a high priority in oncology research (Olawaiye et al., J Clin Oncol, 2025).

As the oncology community awaits the FDA's decision, the implications for the treatment landscape of platinum-resistant ovarian cancer could be significant, paving the way for improved patient outcomes where options have previously been scarce.