Press release

January 03, 2022
2 min read

FDA approves levoketoconazole for Cushing’s syndrome


Press release

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The FDA approved the steroidogenesis inhibitor levoketoconazole for adults with Cushing’s syndrome for whom surgery is not an option or has not been curative, according to an industry press release.

The approval of levoketoconazole (Recorlev, Xeris Biopharma) follows positive efficacy and safety results from the multinational phase 3 SONICS and LOGICS studies evaluating levoketoconazole as a potential treatment for endogenous hypercortisolemia in adults with Cushing’s syndrome. As Healio previously reported, the FDA accepted a new drug application for levoketoconazole in May.

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Despite the recent approval of novel therapies, management of Cushing’s disease remains challenging. The disorder is associated with significant comorbidities and has high mortality if left uncontrolled.

The approval of Recorlev was based upon safety and efficacy data from two positive phase 3 studies that evaluated a combined study population of 166 patients, which was representative of the adult drug-treated U.S. population with Cushing’s syndrome. The SONICS study met its primary and key secondary endpoints, significantly reducing and normalizing mean urinary free cortisol concentrations without a dose increase. LOGICS, a double-blind, placebo-controlled randomized-withdrawal study that met its primary and key secondary endpoints, confirmed the efficacy and safety of Recorlev in normalizing and maintaining therapeutic response compared with placebo.

Top-line findings from the LOGICS study demonstrated that levoketoconazole improved and normalized morning urinary free cortisol concentrations for adults with endogenous Cushing’s disease compared with placebo. The drug was generally well tolerated, with safety data mirroring those from the earlier phase 3 SONICS trial.

Endogenous Cushing’s syndrome — caused by chronic hypercortisolism — is rare, with estimates ranging from 40 to 70 people per million affected worldwide, according to the National Institute of Diabetes and Digestive and Kidney Diseases.

Maria Fleseriu

“Levoketoconazole is an important and welcome new therapeutic option for clinicians to help manage patients with endogenous Cushing's syndrome, a severe, potentially life-threatening rare disease, if not appropriately treated, with multisystem signs and symptoms,” Maria Fleseriu, MD, FACE, a Healio | Endocrine Today Co-editor, professor of medicine and neurological surgery and director of the Pituitary Center at Oregon Health & Science University in Portland, said in the release. “In prospective clinical studies, treatment with levoketoconazole was shown to be effective for reducing and normalizing cortisol.”

In November, the Pituitary Society published an updated guideline for the treatment of Cushing’s disease, focusing on new therapeutic options and an algorithm for screening and diagnosis, along with best practices for managing disease recurrence.