Neurocrine Biosciences' Crinecerfont Receives Priority Review for CAH Treatment: FDA Decision Expected by Year-End
Neurocrine Biosciences' Crinecerfont Receives Priority Review for CAH Treatment: FDA Decision Expected by Year-End
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.
神经分泌生物科学公司（NASDAQ:NBIX）今天宣布，美国食品和药物管理局（FDA）已接受了其两个新药申请（NDA），并授予了优先审查地位，用于治疗患有经典先天性肾上腺增生症（CAH）的儿童、青少年和成人。如果获得批准，crinecerfont将成为CAH治疗70年来的首个新治疗选择，是一种首个治疗方法，采用了治疗这种罕见且严重内分泌紊乱的新方法。
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.
提交的crinecerfont新药申请包括：（1）以胶囊剂型（NDA# 218808）为主要效果和安全性演示；以及（2）以口服溶液剂型（NDA# 218820）为主要效果和安全性演示。该机构设置了处方药用户费用(PDUFA)的目标行动日期分别为2024年12月29日和12月30日。FDA表示，目前不打算召开顾问委员会会议讨论这些申请。
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.
FDA的优先审查地位加快了四个月的审查时间，这意味着该机构认识到CAH是一种高度未满足的医疗需求的严重疾病，而crinecerfont是一种提供相对于当前治疗方案显著效益的治疗法。如果crinecerfont获得FDA批准，这将使神经分泌生物科学公司激活其罕见儿科疾病指定优先审查券——该指定于2020年9月被授予。