Following a new U.S. green light for some ovarian cancer patients, Corcept Therapeutics has redeemed its lead asset, Lilacolilant, after it stumbled at the end of the line in another indication late last year.
The FDA has granted full clearance to Corcept’s class-first selective glucocorticoid receptor antagonist (SGRA) for the treatment of adults with platinum-resistant ovarian cancer. Patients had to have tried one to three previous systemic treatments, at least one of which had to include bevacizumab (Avastin).
According to a March 25 press release, the FDA’s approval covers the regimen of Lilacorilant, sold as Lifiorli, alongside the chemotherapy drug nab-paclitaxel (Abraxane). The drug is taken orally the day before, after, and after treatment with the chemotherapy component of the regimen, Corcept explained.
Rifioruri is designed to bind to glucocorticoid receptors, increasing chemotherapy sensitivity by inhibiting cortisol’s suppression of programmed cell death caused by chemotherapy, such as Abraxane, the company said.
As for release timing, a spokesperson told Fierce that Corcept is “working to make Lifiorli available as soon as possible” and expects the drug to be available within a week.
A spokesperson said the wholesale purchase cost for a 28-day treatment cycle would be $37,900 and that the company expects Rifiorli to have “broad coverage across most plans, including Medicare and Medicaid.”
A Corcept representative said the company also has patient assistance options available for those having difficulty obtaining medications.
Notably, this follows the FDA’s rejection of Lilacolilant in late 2025, despite the vastly different indications.
At the time, Corcept was aiming to greenlight an anti-glucocorticoid for Cushing’s syndrome, a rare disease caused by long-term exposure to excess cortisol. The FDA acknowledged the success of Lilacolilant’s Phase 3 trial in that indication, but emphasized the need for “further evidence of efficacy” to reach a conclusion, Corcept explained in late December.
The company said it continues to work on this indication and plans to meet with the FDA soon to address potential future directions.
For new oncology approvals, the FDA based their approval on the pivotal Rosella trial, which compared lifiorli plus Abraxane to Abraxane alone in 381 previously treated ovarian cancer patients.
The company said the study met its two primary endpoints: progression-free survival and overall survival.
Participants on Lifiorli therapy had a 35% reduced risk of death compared to patients on chemotherapy alone. Median overall survival for the study drug cohort was 16 months compared to 11.9 months for patients receiving Abraxane monotherapy.
Corcept noted that in a combined analysis of patients from mid-stage clinical trials of Rosella and Rifiorli, its drugs appeared to be well-tolerated and manageable. The drug’s label includes warnings and precautions against neutropenia, severe infections, adrenal insufficiency, worsening of conditions treated with glucocorticoids, and embryo-fetal toxicity.
“We are honored to offer a new treatment to patients who previously had few options,” Corcept CEO Dr. Joseph Belanoff said in a statement. “We have worked for years to demonstrate the potential of cortisol modulation in oncology. Today’s approval of Lifiorli is an important first step, but there is still much to explore with this new therapy.”
In fact, Belanoff’s company is aiming for more flexible development across multiple potential oncology indications, including endometrial, cervical, pancreatic and prostate cancers.
The FDA’s verdict on Lifiorli came fairly quickly, with Corcept noting earlier this year that it expected an approval decision around mid-July.
At the time, Alexander Olaweye, MD, a University of Pittsburgh physician and principal investigator on the Rosella trial, opined that Lilacolilant “is positioned to become the new standard of care for patients with platinum-resistant ovarian cancer.”
Corcept stock was up nearly 23% as of mid-afternoon Wednesday.
