FDA Approves Recorlev for Treatment of Endogenous Hypercortisolemia

The approval is based on efficacy and safety data from 2 positive phase 3 studies that evaluated a combined study population of 166 individuals, which represented the US population with Cushing syndrome.

Positive results from the pivotal phase 3 LOGICS and SONICS studies show the drug to be effective and safe for adults with Cushing syndrome. The FDA has approved levoketoconazole (Recorlev, Xeris Biopharma Holdings) for the treatment of endogenous hypercortisolemia for adults with Cushing syndrome for whom surgery has not been curative or is not an option.

“We are thrilled with the FDA's approval of Recorlev as a safe and effective treatment option for patients with endogenous Cushing's syndrome. With this approval, Xeris’ experienced endocrinology-focused commercial organization can begin rapidly working to help address the needs of Cushing syndrome patients in the U.S. who are treated with prescription therapy,” Paul Edick, chairman and CEO of Xeris Biopharma, a company that develops and commercializes therapies in endocrinology, gastroenterology, and neurology, said in a statement.

“Today’s announcement also reinforces the value that we saw in acquiring Strongbridge Biopharma’s attractive rare disease portfolio, which we believe will deliver compelling long-term value to our shareholders. We look forward to making Recorlev commercially available in the first quarter,” Edick said.

The approval is based on efficacy and safety data from 2 positive phase 3 studies that evaluated a combined study population of 166 individuals, which represented the US population with Cushing syndrome.

The LOGICS study, which met its primary and key secondary endpoints, confirmed the efficacy and safety of levoketoconazole when compared with a placebo. The SONICS study met its primary and key secondary endpoints, significant reducing and normalizing mean urinary free cortisol concentrations without a dose increase.

“Levoketoconazole is an important and welcome new therapeutic option for clinicians to help manage patients with endogenous Cushing syndrome, a severe, potentially life-threatening rare disease, if not appropriately treated, with multisystem signs and symptoms,” Maria Fleseriu, MD, FACE, professor of medicine and neurological surgery and director of the Pituitary Center at Oregon Health Sciences University, said in the statement.

Endogenous Cushing syndrome is a potentially fatal but rare endocrine disease caused by chronic elevated cortisol exposure, often resulting from a benign tumor of the pituitary gland.

Levoketoconazole is a cortisol synthesis inhibitor and received orphan drug designation from the FDA and the European Medicines Agency for the treatment of endogenous Cushing syndrome.

The boxed precautions and warnings include hepatotoxicity, hypercortisolism, hypersensitivity reaction, QT prolongation, and risks related to decreased testosterone.

Common adverse events are abnormal uterine bleeding, contusion, fatigue, headache, hemorrhage, hypokalemia, nausea, systemic hypertension, and vomiting.

“Today we are excited to see that the long and complicated path of rare drug development has reached FDA approval on a new therapeutic option for our underserved Cushing's community. We are grateful that the researchers worked so diligently for so long to establish the safety and efficacy of this drug,” Leslie Edwin, president of the Cushing’s Support & Research Foundation, said in the statement.

“Rare disease patients know the importance of sharing their complicated experiences as ‘expert witnesses,’ and we thank Xeris for being an early adherent to this concept,” she said.

Reference

Xeris Biopharma announces US FDA approval of Recorlev (levoketoconazole) for the treatment of endogenous hypercortisolemia in adult patients with Cushing’s syndrome. Businesswire. News release. December 30, 2021. Accessed January 3, 2022. https://www.businesswire.com/news/home/20211230005308/en