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Neurocrine Biosciences Announces U.S. FDA Accepts New Drug Applications and Grants Priority Review for Crinecerfont for Pediatric and Adult Patients with CAH
PDUFA Target Action Dates in Late December 2024Highly Selective CRF1 Antagonist is the Potential First New Treatment for CAH in 70 YearsSAN DIEGO, July 1,
About this update from Neurocrine Biosciences, Inc.
[{"type":"text","content":"PDUFA Target Action Dates in Late December 2024Highly Selective CRF1 Antagonist is the Potential First New Treatment for CAH in 70 YearsSAN DIEGO, July 1, 2024 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today 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). If approved, crinecerfont would be the first new treatment option for CAH in 70 years and a first-in-class therapy, with a novel approach for the treatment of this rare and serious endocrine disorder.\n\n \n \n \n \n \n \n\n \nThe 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.\nPriority 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.\n\"Receipt of a Priority Review reflects the FDA's agreement that CAH is a serious condition and there is an urgent need for patients to have access to new treatments,\" said Eiry W. Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences. \"Crinecerfont's compelling efficacy results and excellent safety profile support our filing, and we look forward to working with the FDA as we head toward the PDUFA dates at the end of 2024.\"\nCrinecerfont previously was granted Orphan Drug designation in March 2019 and Breakthrough Therapy designation in December 2023.\nOrphan Drug designation means the company will be exempt from paying PDUFA user fees, receive tax credits for qualified clinical trials, and has the po...