The U.S. Food and Drug Administration has approved a novel combination therapy that significantly extends survival for women with a difficult-to-treat form of ovarian cancer. The new regimen offers a fresh option for patients whose disease has stopped responding to platinum-based chemotherapy.
The approval is for Lifyorli (relacorilant), used in combination with the chemotherapy nab-paclitaxel. It is designated for adults with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received one to three prior lines of therapy. This approval marks the first FDA-cleared selective glucocorticoid receptor antagonist, a type of drug that works by blocking the effects of cortisol. This stress hormone can inadvertently help tumors resist chemotherapy, and modulating it represents a new mode of action in oncology treatment.
The decision was based on results from the phase 3 ROSELLA trial involving 381 patients. The trial met both its primary goals, showing the combination therapy improved both how long patients lived without their cancer worsening and how long they lived overall. Patients receiving Lifyorli plus nab-paclitaxel saw a 35% reduction in the risk of death compared to those on chemotherapy alone. Median overall survival was 16.0 months versus 11.9 months, a gain of 4.1 months. The combination also reduced the risk of disease progression by 30%.
Regarding safety, the prescribing information includes warnings for neutropenia, severe infections, and adrenal insufficiency. The most common adverse reactions included decreased hemoglobin, decreased neutrophils, fatigue, and nausea. Serious adverse reactions occurred in 35% of patients receiving the combination therapy.
Oncologists and patient advocates have welcomed the new treatment. "Data demonstrate that Lifyorli plus nab-paclitaxel provides a clinically meaningful benefit in overall survival for patients with platinum-resistant ovarian cancer and is well tolerated," said Dr. Rob Coleman of Texas Oncology, a special adviser to the GOG Foundation. "Lifyorli is positioned to become a new standard-of-care treatment." The Ovarian Cancer Research Alliance also called it "urgently needed" news for the community.
Looking ahead, the developer, Corcept Therapeutics, indicates this approval is just an initial step, with more research planned to explore the full potential of cortisol modulation in cancer care. For patients facing limited options after platinum therapy fails, this approval provides a new and effective therapeutic pathway grounded in a novel scientific approach.