Neurocrine Biosciences Announces U.S. FDA Accepts New Drug Applications and Grants Priority Review for Crinecerfont for Pediatric and Adult Patients with CAH

Core Viewpoint - Neurocrine Biosciences has received FDA acceptance for two New Drug Applications (NDA) for crinecerfont, which could be the first new treatment for congenital adrenal hyperplasia (CAH) in 70 years, highlighting a significant advancement in addressing a serious endocrine disorder [1][2][3] Group 1: FDA Review and Designations - The FDA has set Prescription Drug User Fee Act (PDUFA) target action dates for the NDAs on December 29 and December 30, 2024, respectively [2] - Crinecerfont has been granted Priority Review designation, which accelerates the review timeline by four months, indicating the serious nature of CAH and the high unmet medical need [2][3] - The drug has also received Orphan Drug designation and Breakthrough Therapy designation, which provide various benefits including exemption from PDUFA fees and potential market exclusivity [3][4] Group 2: Clinical Data and Mechanism of Action - Crinecerfont is a selective corticotropin-releasing factor type 1 receptor (CRF1) antagonist aimed at reducing excess adrenocorticotropic hormone (ACTH) and adrenal androgens through a glucocorticoid-independent mechanism [8] - The CAHtalyst Phase 3 studies demonstrated strong efficacy and safety results, supporting the NDAs submitted to the FDA [4][9] - The drug aims to enable lower, more physiologic dosing of glucocorticoids, potentially reducing complications associated with higher doses currently used in treatment [8] Group 3: Background on Congenital Adrenal Hyperplasia - Congenital adrenal hyperplasia (CAH) is a rare genetic condition primarily caused by a deficiency of the enzyme 21-hydroxylase, leading to severe health issues if untreated [5] - Current treatments involve glucocorticoids at supraphysiologic doses, which can result in significant complications, including metabolic and psychological issues [6][7]