FDA Approves Lifyorli for Platinum-Resistant Ovarian Cancer

Corcept Therapeutics has announced that the U.S. Food and Drug Administration has approved Lifyorli (relacorilant) in combination with nab-paclitaxel for the treatment of adults with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer. The approval marks a significant milestone, as Lifyorli becomes the first approved selective glucocorticoid receptor antagonist (SGRA) for this indication.

The decision is based on results from the pivotal ROSELLA Phase 3 clinical trial, which enrolled 381 patients with platinum-resistant ovarian cancer who had previously received one to three lines of therapy, including bevacizumab. Participants were randomized to receive either Lifyorli plus nab-paclitaxel or nab-paclitaxel alone.

The study successfully met its dual primary endpoints of progression-free survival and overall survival. Patients receiving the combination therapy experienced a 35% reduction in the risk of death compared to those receiving chemotherapy alone, with median overall survival improving to 16.0 months versus 11.9 months. Additionally, the combination reduced the risk of disease progression by 30%, as confirmed by blinded independent central review.

Importantly, the trial did not require biomarker-based patient selection, suggesting that the therapy could benefit a broad population of patients with this difficult-to-treat cancer subtype. Platinum-resistant ovarian cancer is known for its poor prognosis and limited treatment options, making new therapeutic advances particularly significant.

Safety data from the ROSELLA trial and earlier Phase 2 studies indicated that the Lifyorli combination was generally well tolerated and manageable. The most common adverse events included decreased blood counts, fatigue, nausea, diarrhea, and rash. However, the prescribing information includes warnings for serious risks such as neutropenia, severe infections, adrenal insufficiency, and embryo-fetal toxicity.

Clinical experts have highlighted the potential of this new therapy to reshape treatment standards. Rob Coleman noted that the combination delivers a meaningful survival benefit and could become a new standard of care for patients with advanced, recurrent disease. Similarly, Sarah DeFeo welcomed the approval as an important step forward for the ovarian cancer community, emphasizing the urgent need for new treatment options.

Corcept CEO Joseph Belanoff described the approval as the culmination of years of research into cortisol modulation as a novel approach in oncology. He added that the company sees further potential in exploring this mechanism across additional cancer types.

The ROSELLA trial was conducted globally across multiple regions, with results previously presented at the American Society of Clinical Oncology annual meeting and published in The Lancet. Full findings are expected to be presented at the upcoming Society of Gynecologic Oncology meeting.

With this approval, Lifyorli offers a promising new option for patients facing limited alternatives, potentially improving survival outcomes in a challenging disease setting.