Novartis' Innovative Therapy Isturisa (osilodrostat) Receives FDA Approval for Adult Cushing’s Disease

Recently, the U.S. FDA announced the approval of Isturisa (osilodrostat) oral tablets for the treatment of adult patients with Cushing’s disease who are unable to undergo pituitary surgery or who continue to suffer from the disease after pituitary surgery. Isturisa is an 11β-hydroxylase inhibitor developed by Novartis. Last July, Italy’s Recordati acquired the global development and commercialization rights for Isturisa from Novartis.

Cushing's disease is caused by a pituitary tumor that releases excessive adrenocorticotropic hormone (ACTH), leading to overproduction of cortisol by the adrenal glands. This condition most commonly affects adults aged 30–50 years, and women are three times more likely to be affected than men. Cushing's disease can lead to serious health problems, including hypertension, obesity, type 2 diabetes, blood clots in the legs and lungs, bone loss, weakened immune system, and depression.

Isturisa is the first FDA-approved therapy that directly addresses excessive cortisol production by blocking 11β-hydroxylase, thereby inhibiting cortisol synthesis. It has previously been granted orphan drug designation by the FDA.

▲Molecular structure of Isturisa (Image source: Ed (Edgar181) / Public domain)

The efficacy and safety of Isturisa were evaluated in a study involving 137 adult patients. The mean age of these patients was 41 years, and the majority had either failed to achieve disease remission following pituitary surgery or were ineligible for surgical intervention. In this 24-week, single-arm, open-label clinical trial, nearly half of the patients achieved normalization of cortisol levels after 24 weeks of treatment.

Subsequently, 71 patients were enrolled in an 8-week randomized, double-blind study to continue receiving either Isturisa or placebo. At the end of the treatment period, 86% of patients maintained on Isturisa had cortisol levels within the normal range, compared with 30% in the placebo group.

“The FDA supports the development of safe and effective treatments for rare diseases. This innovative therapy can help patients with Cushing’s disease,” said Mary Thanh Hai, M.D., acting director of the Office of Drug Evaluation II in the Center for Drug Evaluation and Research at the FDA. “By helping patients achieve normal cortisol levels, this drug provides an important treatment option for adults with Cushing’s disease.”

References:

[1] FDA Approves New Treatment for Adults with Cushing’s Disease. Retrieved March 6, 2020, from https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-adults-cushings-disease

Flash | FDA Approves Novartis’ Innovative Therapy for Rare Endocrine Disease

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