A drug originally developed to treat a rare hormonal disorder is now offering a new avenue of hope for patients battling one of the most aggressive forms of ovarian cancer, following the results of a major international trial.
How Blocking a Stress Hormone Can Fight Cancer
The drug, called relacorilant, is a selective glucocorticoid receptor antagonist. It was designed for Cushing’s syndrome, a condition of excess cortisol, but its mechanism has shown a surprising benefit in oncology. Researchers believe that cortisol, often elevated in cancer patients, can suppress the immune system and contribute to tumours becoming resistant to chemotherapy. Relacorilant works by blocking the receptor for this hormone, potentially re-sensitising cancer cells to treatment and boosting the body’s own defences.
The Phase 3 ROSELLA trial, which enrolled 381 women with platinum-resistant ovarian cancer who had received up to three prior lines of therapy, tested this approach. Patients received standard chemotherapy (nab-paclitaxel) either with or without relacorilant. According to the study published in the Lancet, the combination met its primary goals, significantly extending both the time patients lived without their cancer worsening and overall survival.
The data showed a 30% reduction in the risk of progression or death. Crucially, the median overall survival for those on the relacorilant combination was 16 months, compared to 11.9 months for chemotherapy alone—an extension of 4.1 months and a 35% reduction in the risk of death. Based on these results, the U.S. Food and Drug Administration (FDA) approved relacorilant, branded as Lifyorli, in March 2026 for this specific group of patients. An application has also been submitted to the European Medicines Agency.
The treatment is administered orally around the time of chemotherapy infusions. The ROSELLA trial indicated it was generally well-tolerated, though serious adverse reactions including neutropenia, infections, and pleural effusion were noted. The drug is contraindicated for patients who require life-saving corticosteroids and carries warnings about adrenal insufficiency. Notably, relacorilant’s use does not depend on a specific tumour biomarker, meaning it could be suitable for a broad patient population.
A Second New Option: Boosting the Immune System
In a separate development, another treatment has also shown significant promise for extending life in platinum-resistant disease. The Phase 3 KEYNOTE-B96 trial involved 643 patients and tested the immunotherapy drug pembrolizumab (Keytruda) alongside chemotherapy.
Pembrolizumab works by targeting the PD-1 receptor, effectively taking the “brakes” off the immune system so it can better attack cancer cells. The trial found that adding the drug improved median overall survival to 18.2 months, compared to 14 months with chemotherapy and a placebo. The FDA approved this regimen in February 2026, and the European Union followed with its approval in April 2026, but it is restricted to patients whose tumours test positive for the PD-L1 biomarker.
The safety profile was consistent with known effects of pembrolizumab, which includes warnings for immune-mediated adverse reactions. While approved for other cancers in the UK, its use for ovarian cancer, like relacorilant, requires further regulatory review by British authorities.
The Challenge of Ovarian Cancer in the UK
These advances are targeted at a critical unmet need. Ovarian cancer is the sixth most common cancer among women in the UK, with approximately 7,600 new cases diagnosed annually—a figure projected to rise to around 9,400 per year by 2040. It accounts for about 4% of all new female cancer cases and leads to roughly 3,900 deaths each year.
Survival rates paint a stark picture of the disease’s challenge. While over 90% of women diagnosed at the earliest stage (Stage I) survive for five years or more, the majority of cases are detected at an advanced stage (III or IV), where five-year survival plummets to around 17%. Overall, only about 43% of women in the UK survive ovarian cancer for five years, a rate lower than the European average.
The new trial results focus specifically on platinum-resistant ovarian cancer, which occurs when the disease progresses within six months of first-line platinum chemotherapy. This form of the disease is particularly difficult to treat, with patients typically facing a prognosis of about one year post-diagnosis. Current treatment options are limited to alternative non-platinum chemotherapies and, for some, newer targeted therapies like antibody-drug conjugates.