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Recordati's Isturisa is expected to launch in the second or third quarter. (Getty) As part of a small 2019 deal, Italian drugmaker Recordati snagged a trio of underperforming Novartis endocrinology meds, including a late-stage candidate for Cushing's disease. Less than a year later, that drug is cleared for market after an FDA green light. The FDA on Friday approved Recordati's Isturisa (osilodrostat) to treat Cushing's disease—a rare disease in which patients' adrenal glands produce too much cortisol—in those who have undergone a prior pituitary gland surgery or are not eligible for one. Isturisa, a cortisol synthesis inhibitor, will come with the FDA's orphan drug designation, providing market exclusivity for seven years, Recordati said (PDF) in a release. The drug is expected to be commercially available in the second or third quarter. The FDA based its review on phase 3 data showing 86% of patients treated with Isturisa showed normal cortisol levels in their urine after eight weeks, compared with 29% of patients treated with placebo, the drugmaker said. Recordati is "actively building its commercial, medical, and market access teams" to accommodate Isturisa's launch through its recently created U.S. endocrinology business unit, it said. The drugmaker will launch the drug with a "comprehensive distribution model" through specialty pharmacies. Novartis, once the owner of Isturisa, turned the asset over to Recordati in 2019 as part of a $390 million offload of some of the Swiss drugmaker's endocrinology portfolio. Recordati received Signifor, long-acting sister Signifor LAR and Isturisa, positioned as a successor drug to Signifor. The purchase included milestone payments tied to Isturisa. Recordati talked up the buy of the Cushing's disease trio as a boon for its rare disease portfolio, calling it a "key and historical milestone" at the time. From https://www.fiercepharma.com/pharma/recordati-scores-fda-nod-for-cushing-s-disease-med-isturisa -
The U.S. Food and Drug Administration today approved Isturisa (osilodrostat) oral tablets for adults with Cushing's disease who either cannot undergo pituitary gland surgery or have undergone the surgery but still have the disease. Cushing's disease is a rare disease in which the adrenal glands make too much of the cortisol hormone. Isturisa is the first FDA-approved drug to directly address this cortisol overproduction by blocking the enzyme known as 11-beta-hydroxylase and preventing cortisol synthesis. "The FDA supports the development of safe and effective treatments for rare diseases, and this new therapy can help people with Cushing's disease, a rare condition where excessive cortisol production puts them at risk for other medical issues," said Mary Thanh Hai, M.D., acting director of the Office of Drug Evaluation II in the FDA's Center for Drug Evaluation and Research. "By helping patients achieve normal cortisol levels, this medication is an important treatment option for adults with Cushing's disease." Cushing's disease is caused by a pituitary tumor that releases too much of a hormone called adrenocorticotropin, which stimulates the adrenal gland to produce an excessive amount of cortisol. The disease is most common among adults between the ages of 30 to 50, and it affects women three times more often than men. Cushing's disease can cause significant health issues, such as high blood pressure, obesity, type 2 diabetes, blood clots in the legs and lungs, bone loss and fractures, a weakened immune system and depression. Patients may have thin arms and legs, a round red full face, increased fat around the neck, easy bruising, striae (purple stretch marks) and weak muscles. Isturisa's safety and effectiveness for treating Cushing's disease among adults was evaluated in a study of 137 adult patients (about three-quarters women) with a mean age of 41 years. The majority of patients either had undergone pituitary surgery that did not cure Cushing's disease or were not surgical candidates. In the 24-week, single-arm, open-label period, all patients received a starting dose of 2 milligrams (mg) of Isturisa twice a day that could be increased every two weeks up to 30 mg twice a day. At the end of this 24-week period, about half of patients had cortisol levels within normal limits. After this point, 71 patients who did not need further dose increases and tolerated the drug for the last 12 weeks entered an eight-week, double-blind, randomized withdrawal study where they either received Isturisa or a placebo (inactive treatment). At the end of this withdrawal period, 86% of patients receiving Isturisa maintained cortisol levels within normal limits compared to 30% of patients taking the placebo. The most common side effects reported in the clinical trial for Isturisa were adrenal insufficiency, headache, vomiting, nausea, fatigue and edema (swelling caused by fluid retention). Hypocortisolism (low cortisol levels), QTc prolongation (a heart rhythm condition) and elevations in adrenal hormone precursors (inactive substance converted into a hormone) and androgens (hormone that regulates male characteristics) may also occur in people taking Isturisa. Isturisa is taken by mouth twice a day, in the morning and evening as directed by a health care provider. After treatment has started, a provider may re-evaluate dosage, depending upon the patient's response. Isturisa received Orphan Drug Designation, which is a special status granted to a drug intended to treat a rare disease or condition. The FDA granted the approval of Isturisa to Novartis. Media Contact: Monique Richards, 240-402-3014 Consumer Inquiries: Email, 888-INFO-FDA The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products. SOURCE U.S. Food and Drug Administration Related Links http://www.fda.gov From https://www.prnewswire.com/news-releases/fda-approves-new-treatment-for-adults-with-cushings-disease-301019293.html
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The U.S. Food and Drug Administration has approved the clinical use of a magnetic resonance imaging (MRI) scanner — the ultra-high-field 7T Terra MRI — with unprecedented resolution that allows for more reliable images of the brain. The approach recently allowed the precise localization of a small tumor in the pituitary gland, which standard MRI had failed to spot, in a patient with Cushing’s disease. So far, only one scanner of this kind exists in the U.S.. It was installed in February 2017 at the Mark and Mary Stevens Neuroimaging and Informatics Institute (INI) of the Keck School of Medicine, University of Southern California (USC). The new scanner has an increased magnetic field strength of 7 Tesla, which is more than four times that of conventional MRI. This property greatly improves the instrument’s signal-to-noise ratio, dramatically increasing the spatial resolution and contrast of its images so that scientists can visualize the human living brain in high-definition and with unprecedented detail. The 7T Terra is ideal for high-resolution neuroimaging, exploration of neurodegenerative diseases such as Alzheimer’s and Parkinson’s, and diagnosis and treatment of other brain diseases, a USC news story by Zara Greenbaum states. Earlier this year, a report described the case of women with Cushing’s disease with a pituitary adenoma (slow-growing, benign tumor in the pituitary gland) that was possible to localize only with the new 7T MRI. Based on laboratory analysis that revealed high levels of adrenocorticotropic hormone(ACTH) and cortisol, the doctors suspected a pituitary adenoma and recommended the patient for surgery. However, they ignored the precise location of the tumor, which failed to be detected by standard MRI scanners (1T and 3T). Two hours before surgery, the woman underwent a 7T MRI scan which finally identified with high precision the location of the adenoma, a very small tumor of 8 mm on the right side of the pituitary gland. “The 7T may save patients an invasive procedure. It also makes it easier for neurosurgeons to selectively remove a tumor without damaging surrounding areas,” said Gabriel Zada, MD, associate professor of neurological surgery at the Keck School. Since its arrival, the device has supported exploratory research into both healthy and diseased brains. Now the scanner’s advanced imaging technology can be used to help with diagnosis, treatment and monitoring of patients with neurological diseases, including Cushing’s disease. “This device, which has already made its mark as a powerful tool to advance research in the neurosciences, is now accessible to clinical populations in addition to researchers,” said Arthur W. Toga, PhD, provost professor and chair at the Keck School and director of the USC Stevens INI. “Clinicians across the university and beyond can now leverage all the benefits of increased spatial resolution to serve patients in need,” he said. Adapted from https://cushingsdiseasenews.com/2018/11/06/fda-oks-high-resolution-mri-better-spotting-pituitary-tumor-cushings/
