Neurocrine(US:NBIX) Prnewswire·2024-12-13 23:55Core Insights - CRENESSITY is the first new treatment for classic congenital adrenal hyperplasia (CAH) in 70 years, approved by the FDA as an adjunctive treatment to glucocorticoid replacement [1][21] - The approval is based on the largest clinical trial program for CAH, known as the CAHtalyst studies, which demonstrated significant efficacy and safety [5][18] - CRENESSITY is expected to be commercially available within a week and will be distributed through a specialty pharmacy [2][3] Company Overview - Neurocrine Biosciences has a long history of research in the role of corticotropin-releasing factor and its receptor in CAH, marking the approval of CRENESSITY as a significant milestone for the CAH community [2] - The company is committed to supporting patients with access to CRENESSITY through a comprehensive assistance program [3] Clinical Trial Data - The CAHtalyst studies included 285 patients and demonstrated that CRENESSITY significantly reduced androgen levels and allowed for lower glucocorticoid doses [16][18] - In the pediatric study, patients taking CRENESSITY experienced approximately four times greater reduction in androstenedione and steroid doses compared to placebo [8] - In the adult study, 63% of patients achieved glucocorticoid doses in the physiologic range while maintaining or improving androgen levels, compared to 18% in the placebo group [9] Treatment Mechanism - CRENESSITY functions as a selective CRF1 antagonist, reducing excess ACTH and adrenal androgen production, which allows for glucocorticoid dose reduction [19][20] - The treatment is available in both capsule and oral solution forms, with specific dosing recommendations based on patient age and weight [20] Safety and Tolerability - CRENESSITY was well tolerated in clinical trials, with no serious treatment-related adverse events reported [10] - Common side effects included headache, abdominal pain, and fatigue, which were generally mild to moderate in severity [8][9]