Search the Community
Showing results for tags 'adults'.
The FDA accepted for review a new drug application for the steroidogenesis inhibitor levoketoconazole for the treatment of endogenous Cushing’s syndrome, according to an industry press release. “We are pleased with the FDA’s acceptance for filing of the Recorlev new drug application,” John H. Johnson, CEO of Strongbridge Biopharma, said in the release. “We believe this decision reflects the comprehensive clinical evidence that went into the NDA submission, including the positive and statistically significant efficacy and safety results from the multinational phase 3 SONICS and LOGICS studies evaluating Recorlev as a potential treatment option for adults with endogenous Cushing’s syndrome. We are advancing our commercial readiness plans and look forward to potentially bringing a new therapeutic option to the Cushing’s syndrome community in the first quarter of 2022.” As Healio previously reported, top-line findings from the LOGICS study demonstrated that levoketoconazole (Recorlev, Strongbridge Biopharma) 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. The FDA set a Prescription Drug User Fee Act target action date of Jan. 1, 2022, for levoketoconazole, according to the company. The FDA letter made no mention of a plan to hold an advisory committee meeting. From https://www.healio.com/news/endocrinology/20210513/fda-accepts-nda-for-novel-cushings-syndrome-treatment
Metyrapone treatments helped patients with Cushing syndrome reach normal, urinary-free cortisol levels in the short-term and also had long-term benefits, according to a study published in Endocrine. This observational, longitudinal study evaluated the effects of the 11β -hydroxylase inhibitor metyrapone on adult patients with Cushing syndrome. Urinary-free cortisol and late-night salivary cortisol levels were evaluated in 31 patients who were already treated with metyrapone to monitor cortisol normalization and rhythm. The average length of metyrapone treatment was 9 months, and 6 patients had 24 months of treatment. After 1 month of treatment, the mean urinary-free cortisol was reduced from baseline by 67% and mean late-night salivary cortisol level decreased by 57%. Analyzing only patients with severe hypercortisolism, after 1 month of treatment, the mean urinary-free cortisol decreased by 86% and the mean late-night salivary cortisol level decreased 80%. After 3 months, normalization of the mean urinary-free cortisol was established in 68% of patients. Mean late-night salivary cortisol levels took longer to decrease, especially in severe and very severe hypercortisolism, which could take 6 months to drop. Treatment was more successful at normalizing cortisol excretion (70%) than cortisol rhythm (37%). Nausea, abdominal pain, and dizziness were the most common adverse events, but no severe adverse event was reported. Future research is needed to evaluate a larger cohort with randomized dosages and stricter inclusion criteria to evaluate metyrapone's effects on cortisol further. Study researchers conclude that metyrapone was successful and safe in lowering urinary-free cortisol after just 1 month of treatment and controlling long-term levels in patients with Cushing syndrome. This study was supported by Novartis. Reference Ceccato F, Zilio M, Barbot M, et al. Metyrapone treatment in Cushing's syndrome: a real-life study [published online July 16, 2018]. Endocrine. doi: 10.1007/s12020-018-1675-4 From https://www.endocrinologyadvisor.com/general-endocrinology/metyrapone-cushing-syndrome/article/786716/
Frontiers in Endocrinology, 04/24/2013 Reed ML et al. – Deficiency of growth hormone (GH) in adults results in a syndrome characterized by decreased muscle mass and exercise capacity, increased visceral fat, impaired quality of life, unfavorable alterations in lipid profile and markers of cardiovascular risk, decrease in bone mass and integrity and increased mortality. The potential of GH to act as a mitogen has resulted in concern over the possibility of increased de novo tumors or recurrence of pre–existing malignancies in individuals treated with GH. Though studies of adults who received GHRT in childhood have produced conflicting reports in this regard, long term surveillance of adult GHRT has not demonstrated increased cancer risk or mortality. Read more: http://www.mdlinx.com/endocrinology/news-article.cfm/4588746/growth-hormone-adult-growth-hormone-deficiency#ixzz2RODfAgDb