Neurocrine Biosciences, Inc. announced the U.S. Food and Drug Administration (FDA) has accepted its two New Drug Applications (NDA) with Priority Review designations for crinecerfont in the treatment of children, adolescents and adults with classic congenital adrenal hyperplasia (CAH). The submitted crinecerfont NDAs included: the primary presentation of efficacy and safety of crinecerfont for the treatment of classic CAH as (1) a capsule formulation (NDA# 218808); and (2) as an oral solution formulation (NDA# 218820). The agency set Prescription Drug User Fee (PDUFA) target action dates of December 29 and December 30, 2024, respectively.

The FDA stated it is not currently planning to hold an advisory committee meeting to discuss these applications. Priority Review designation by the FDA accelerates the review timeline by four months - and means the agency recognizes CAH is a serious condition with high unmet medical need and crinecerfont is a treatment that provides significant benefit over current therapy. Should crinecerfont receive FDA approval, it will enable Neurocrine Biosciences to activate its Rare Pediatric Disease Designation Priority Review Voucher - a designation granted in September 2020.

Orphan Drug designation means the company will be exempt from paying PDUFA user fees, receive tax credits for qualified clinical trials, and has the potential of seven years of market exclusivity should crinecerfont be approved. Breakthrough Therapy designation is a process developed by the FDA to expedite development and review of drugs that are intended to treat a serious condition and where clinical evidence indicates that the potential drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). Crinecerfont study data demonstrate that lowering adrenal androgen levels enables lower, more physiologic dosing of glucocorticoids to manage androgen excess and thus could potentially reduce the complications associated with exposure to greater than normal glucocorticoid doses in patients with CAH.

Data from the CAHtalyst Pediatric and CAHtalyst Adult Phase 3 studies supported two New Drug Application submissions to the U.S. food and Drug Administration in April 2024. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington's disease, endometriosis and uterine fibroids, as well as a robust pipeline including multiple compounds in mid- to late-phase clinical development across core therapeutic areas. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements include: the crinecerfont NDAs may not obtain regulatory approval, such approval may be delayed, or may not receive the benefits associated with priority review; additional regulatory submissions may not occur or be submitted in a timely manner; the FDA may make adverse decisions regarding crinecerfont; crinecerfont may not be found to be safe and/or effective or may not prove to be beneficial to patients; development activities for crinecerfont may not been completed on time or at all; clinical development activities may be delayed for regulatory or other reasons, may not be successful or replicate previous and/or interim clinical trial results, or may not be predictive of real-world results or of results in subsequent clinical trials; competitive products and technological changes that may limit demand for products; uncertainties relating to patent protection and intellectual property rights of third parties; their dependence on third parties for development and manufacturing activities related to crinecerfont, and ability to manage these third parties.