FDA approves ovarian cancer treatment

The FDA has approved a new treatment for ovarian cancer in adults with platinum-resistant disease after one to three earlier systemic treatments.

The approval covers Lifyorli, also known as relacorilant, in combination with the chemotherapy nab-paclitaxel for adults with platinum-resistant epithelial ovarian, fallopian tube or primary peritoneal cancer. Patients must have received one to three prior systemic treatment regimens, with at least one including bevacizumab.

Lifyorli, developed by Corcept Therapeutics, is the first FDA-approved selective glucocorticoid receptor antagonist. This type of drug blocks the effects of cortisol, a stress hormone that can help tumours resist chemotherapy.

Joseph Belanoff, Corcept’s chief executive officer, said: “It is a privilege to bring forward a new treatment for patients who historically have had few options.

“We have worked for years to demonstrate the potential of cortisol modulation in oncology.

“Today’s approval of Lifyorli is an important first step, but there is much more to explore with this new mode of treatment.

“We extend our deep appreciation to the patients and healthcare providers who participated in the clinical trials that made this approval possible.”

Approval was based on the ROSELLA trial, which enrolled 381 patients with platinum-resistant ovarian cancer who had received one to three prior lines of therapy, with at least one including bevacizumab.

Patients were randomised one to one to receive either Lifyorli plus nab-paclitaxel or nab-paclitaxel alone. No biomarker selection was required.

The trial met its dual primary endpoints of progression-free and overall survival.

Patients treated with Lifyorli plus nab-paclitaxel saw a 35 per cent reduction in the risk of death compared with patients treated with nab-paclitaxel alone, with median overall survival of 16.0 months compared with 11.9 months, a difference of 4.1 months.

Patients who received Lifyorli plus nab-paclitaxel also saw a 30 per cent reduction in the risk of disease progression compared with patients treated with nab-paclitaxel alone.

The safety of Lifyorli was assessed in a pooled analysis of patients from ROSELLA and the drug’s phase 2 trial.

The prescribing information includes warnings and precautions for neutropenia and severe infections, adrenal insufficiency, exacerbation of conditions treated with glucocorticoids and embryo-fetal toxicity.

The most common adverse reactions in more than 20 per cent of patients, including laboratory abnormalities, were decreased haemoglobin, decreased neutrophils, fatigue, nausea, diarrhoea, decreased platelets, rash and decreased appetite.

Serious adverse reactions occurred in 35 per cent of patients who received Lifyorli in combination with nab-paclitaxel.

Rob Coleman, of Texas Oncology and special adviser to the president of the GOG Foundation, said: “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.

“Lifyorli is positioned to become a new standard-of-care treatment.

“Having a new treatment for this advanced, recurrent disease will provide clinicians with a compelling option to help patients with this extremely difficult-to-treat cancer.”

Sarah DeFeo, chief programme officer at Ovarian Cancer Research Alliance, added: “The FDA’s approval of Lifyorli in combination with nab-paclitaxel is welcome news to all of us in the ovarian cancer community.

“We are grateful to everyone who participated in the clinical trials, their families and the physicians who helped advance this urgently needed treatment option for patients with platinum-resistant ovarian cancer.”