FDA授予失眠药daridorexant突破性疗法认定：针对10至18岁患者，儿童失眠尚无获批药物
瑞士生物制药公司 Idorsia 于2026年11月4日宣布，美国食品药品监管局（FDA）已授予其失眠药 daridorexant 突破性疗法认定，用于治疗10岁至未满18岁患者的失眠障碍。这是 FDA 首次以这一快速通道认定儿童青少年失眠用药，目前该年龄段没有任何获 FDA 批准的药物治疗。
突破性疗法认定用于加速针对严重疾病的药物开发与审评，前提是初步临床证据显示该药可能在一个或多个重要临床终点上带来实质改善。获得认定后，Idorsia 将得到 FDA 更密集的指导与更频繁的沟通。daridorexant 是一种双重食欲素受体拮抗剂，选择性降低清醒驱动，而非广泛抑制大脑活动；成人剂量25毫克与50毫克的疗效已发表于《柳叶刀·神经病学》。
认定依据是一项多中心、随机、双盲、安慰剂对照的二期剂量探索研究（NCT05423717）：165名10岁至未满18岁的失眠障碍患者分别接受 daridorexant 10毫克、25毫克、50毫克或安慰剂，疗程两周，以多导睡眠监测测得的客观总睡眠时间为主要终点。结果显示总睡眠时间出现统计学显著的剂量依赖改善（p=0.0185）；单次50毫克使总睡眠时间总体增加50分钟，在伴有神经发育障碍的患者中增加64分钟，清晨困倦呈数值下降。安全性方面，即使在成人最高推荐剂量50毫克下，其耐受性与安慰剂相近，治疗期间未见提示药物滥用的不良事件，停药后未见戒断症状迹象。
儿童失眠的特点是入睡或维持睡眠困难，并造成日间功能损害，患病率约8%至12%，在孤独症谱系障碍与注意缺陷多动障碍等神经发育障碍患者中负担尤重。Idorsia 首席医学官 Amer Joseph 表示，这一认定凸显了儿童青少年失眠患者重大的未满足医疗需求与 daridorexant 迄今令人鼓舞的临床证据，该药有望成为首个获 FDA 批准的儿童失眠药物治疗。
Idorsia 说明，daridorexant 的儿童用途目前仍属研究性质，在任何国家均未获批上市；完整数据集论文正在接受一家高影响力学术期刊评审，公司正汇总全球卫生管理机构对三期临床方案的意见，预计2027年下半年启动三期项目。（来源：Idorsia）
FDA Grants Breakthrough Therapy Designation to Daridorexant for Insomnia in Patients Ages 10 to Under 18
Swiss biopharmaceutical company Idorsia announced on November 4, 2026 that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to its insomnia drug daridorexant for the treatment of insomnia disorder in patients aged 10 to under 18. It is the first such expedited designation for a pediatric insomnia medicine, and no drug treatment is currently approved by the FDA for this age group.
Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious conditions where preliminary clinical evidence indicates a substantial improvement on a clinically significant endpoint. The designation gives Idorsia intensive FDA guidance and more frequent interaction. Daridorexant is a dual orexin receptor antagonist that selectively reduces wake drive rather than broadly inhibiting brain activity; its efficacy in adults at 25 mg and 50 mg doses was published in The Lancet Neurology.
The designation rests on a multicenter, randomized, double-blind, placebo-controlled Phase 2 dose-finding study (NCT05423717) in which 165 children and adolescents aged 10 to under 18 with insomnia disorder received daridorexant 10 mg, 25 mg or 50 mg, or placebo, over two weeks, with objective total sleep time measured by polysomnography as the primary endpoint. The study showed a statistically significant dose-dependent improvement in total sleep time (p=0.0185); a single 50 mg dose increased sleep time by 50 minutes overall and by 64 minutes in patients with neurodevelopmental disorders, with a numerical decrease in morning sleepiness. Even at 50 mg, the highest recommended adult dose, tolerability remained similar to placebo, with no adverse events denoting drug abuse and no indication of withdrawal symptoms on discontinuation.
Pediatric insomnia, characterized by difficulty initiating or maintaining sleep with significant daytime impairment, has a prevalence of about 8 to 12 percent and a particularly high burden in patients with neurodevelopmental disorders such as autism spectrum disorder and ADHD. Idorsia Chief Medical Officer Amer Joseph said the designation underscores the significant unmet medical need and the encouraging clinical evidence, and that daridorexant could become the first FDA-approved pharmacological treatment for pediatric insomnia disorder.
Idorsia noted that daridorexant for pediatric use is investigational and is not approved or marketed in any country. The full dataset manuscript is under evaluation at a high-impact scientific journal, and the company expects to initiate its Phase 3 program in the second half of 2027. (Source: Idorsia Ltd)
来源：Idorsia
发布：GSM·美国（发布链接）
图片来源：图：位于 Maryland 州 Silver Spring 的美国食品药品监管局（FDA）白橡树园区总部大楼。美国食品药品监管局，公共领域。 / Photo: FDA White Oak campus headquarters in Silver Spring, Maryland. U.S. Food and Drug Administration, public domain.
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