Armodafinil

2026年7月21日，国家药品监督管理局（NMPA）批准武田制药奥博雷通片（Oveporexton，TAK-861）上市，用于治疗16岁及以上1型发作性睡病（NT1）。这不仅是一款新药的获批，更是全球多中心研发的原创靶点新药在中国实现首报首发的历史性突破。 01.中国首报首发：打破"时差"的历史性突破 长期以来，全球创新药研发存在一个"时差"问题——跨国药企通常先在美国或欧洲提交上市申请，待FDA或EMA批准后再在中国提交，这个过程往往需要3至5年。对于罕见病患者而言，这种等待意味着漫长的煎熬。 奥博雷通片彻底打破了这一惯例。该药于2026年1月在中国首次申报上市，同年2月、6月才分别在美国和日本申报——首次申报选在中国，首次获批也发生在中国。从申报到获批，审评仅历时6个月。这意味着中国患者成为全球首批用上这款新药的人。 6个月 中国审评周期 首发 全球首个批准国家 3国 中美日同步申报 国家药监局药品注册司化学药品处处长徐晓强对此评价："这是中国药品监管改革和医药创新生态吸引力增强的里程碑事件。"中美均授予该药突破性疗法认定，在日本获先驱和孤儿药资格，欧盟也授予了孤儿药资格——其临床价值已获全球公认。 02.发作性睡病：被忽视的24小时疾病 1型发作性睡病（NT1）是一种罕见且严重的慢性神经系统疾病，已列入我国《第二批罕见病目录》。其病因是下丘脑中约7万个产生食欲素（Orexin，又称下丘脑分泌素）的神经元被自身免疫系统大量破坏，导致脑内食欲素水平严重下降或缺失。 NT1远不止"白天爱睡觉"那么简单，它是一种24小时疾病，症状贯穿昼夜： 1 日间过度嗜睡（EDS）：无法控制地陷入睡眠，严重影响工作和学习 2 猝倒（Cataplexy）：由强烈情绪触发的突然肌肉无力，轻则面部松弛，重至全身瘫软 3 夜间睡眠紊乱：睡眠碎片化、入眠幻觉、睡眠瘫痪 4 认知功能受损：注意力、执行功能和记忆力下降 更令人忧心的是，NT1极易被误诊。有研究显示，患者从发病到确诊平均需要8-10年，常被误诊为癫痫、精神分裂症或抑郁症。中国患病率约为0.033%，看似数字不高，但受累患者的生活质量遭受毁灭性打击。 💡 现有治疗的困境 当前NT1的治疗手段均为"对症治疗"——莫达非尼/阿莫达非尼促进清醒，羟基丁酸钠改善夜间睡眠，抗抑郁药减少猝倒。这些药物作用机制均位于食欲素信号的下游，没有任何一款药物针对NT1的根本病因——食欲素缺乏。多药联用、副作用明显、疗效有限，成为NT1患者的日常困境。 03.TAK-861：从根源修复食欲素信号 如果说现有药物是"在断电的房子里用发电机"，那么TAK-861就是"直接把电接回电路"。 奥博雷通（TAK-861）是武田自主研发的全球首创（First-in-Class）口服选择性食欲素2型受体（OX2R）激动剂。其核心创新在于： 🧬 作用机制：精准替代缺失信号 NT1患者大脑中产生食欲素的神经元大量丢失，导致食欲素信号缺失。TAK-861直接激活OX2R——食欲素促进清醒的主要受体——模拟内源性食欲素的功能，从上游恢复整条信号通路，同时改善日间嗜睡、猝倒、认知障碍和夜间睡眠紊乱等广泛症状谱。 💊 口服 vs 注射 此前武田开发的danavorexton（TAK-925）为静脉注射制剂，给药不便。TAK-861实现口服给药，每日两次，极大提升了患者依从性和用药便利性。 🎯 3000倍选择性 TAK-861对OX2R的EC₅₀为2.5 nM，对OX1R为7500 nM，对OX2R的选择性是OX1R的3000倍，最大限度减少脱靶效应，安全性优势显著。 值得一提的是，TAK-861的发现过程本身就是药物化学的精彩案例。武田此前开发的口服OX2R激动剂TAK-994虽然有效，但存在肝毒性风险（被认为属脱靶效应）。研究团队通过提高分子效力和降低有效剂量，成功开发出效力约为TAK-99410倍的TAK-861，有效规避了前代化合物的安全性问题。 04.临床数据：全面改写治疗标准 TAK-861的获批基于两项全球关键性III期研究——FirstLight（TAK-861-3001）和RadiantLight（TAK-861-3002），这是迄今为止NT1领域规模最大、最全面的临床开发项目。 两项研究在19个国家开展，共入组273例患者（FirstLight 168例，RadiantLight 105例），入组在6个月内即告完成，足见NT1领域未被满足的治疗需求之迫切。 📊 核心疗效数据（12周，vs 安慰剂） 觉醒维持（MWT）：2mg/2mg剂量组平均睡眠潜伏期较基线变化为+20.09分钟，2/2mg剂量组63%的患者达到≥20分钟的正常范围（安慰剂仅3%） 日间嗜睡（ESS）：ESS评分较基线改善-9.53分，近85%的患者达到≤10分的正常水平 猝倒（WCR）：每周猝倒发生率较安慰剂降低75%（发生率比0.25），猝倒天数从基线的0天/周改善至4-5天/周 认知功能：约70%的患者报告无显著认知困难（安慰剂仅约15%） 更值得关注的是，两项研究的14项主要和次要终点全部达标，所有剂量组p值均<0.001。FirstLight研究主要研究者Emmanuel Mignot博士指出："奥博雷通展示了将疾病管理超越增量症状缓解、转向整体疾病改善的潜力。" 安全性方面，TAK-861总体耐受性良好。最常见不良事件为尿频（61.4%）和失眠（57.1%），未见严重治疗相关不良事件。超过95%的完成研究者进入长期扩展研究，也从一个侧面反映了患者对治疗的认可。 05.中国首发获批的深层意义 TAK-861在中国首发获批，其意义远超一款新药的上市。 1 审评提速的制度保障：该药获得"突破性治疗品种"认定并被纳入"优先审评审批"通道，6个月完成审评，展现了NMPA对高临床价值创新药的加速审评能力。这不是"降低标准"，而是"提质增效"。 2 全球同步开发模式的验证：TAK-861采用"全球研发一盘棋、中国同步设计、同步申报、同步商业化"的新模式，标志着跨国药企在中国的研发路径从"境外上市后引进"升级为"全球首发布局"。 3 患者可及性的根本改善：过去中国NT1患者需等待数年才能用上海外新药，如今不仅与全球同步，更是率先获益。这款全球首创的对因治疗药物，让中国患者第一次拥有了从根源上对抗疾病的可能。 4 中国创新药生态的吸引力跃升：中国从全球医药创新的"跟进者"向"首发者"迈进，体现了监管体系的成熟和中国市场在全球新药开发战略中地位的实质性提升。 06.展望：一个新起点的开始 TAK-861的获批是一个里程碑，也是一个新起点。 对NT1患者而言，这是首个从病因层面干预的治疗药物，有望从根本上改变"治标不治本"的困境。但我们也应看到，该药目前仅获批用于16岁及以上患者，而NT1好发于学龄期儿童，儿科适应症的拓展将是下一步的关键。此外，食欲素受体激动剂对2型发作性睡病（NT2，脑脊液食欲素水平正常）的疗效尚在探索中，这也是武田食欲素管线面临的科学挑战。 对中国创新药审评而言，TAK-861的"中国首报首发"模式将成为标杆。它证明了中国监管体系完全具备支持全球同步首发的能力，未来有望吸引更多跨国药企将中国纳入创新药全球首发布局。今年国产创新药密集取得突破，而这款全球同步首发的原创靶点药，则标志着中国正从全球医药创新的"跟进者"向"首发者"迈进。 对武田而言，TAK-861是武田食欲素管线的核心资产。武田CEO Christophe Weber称其为"武田在食欲素生物学领域领导地位和构建多资产食欲素特许经营组合的体现"，将为武田的长期增长奠定基础。FDA已受理其NDA并授予优先审评，PDUFA日期在今年第三季度，全球上市在即。 "发作性睡病不是由单一症状定义的疾病，这就是为什么我们设计了全面的III期项目来评估奥博雷通对广泛疾病影响的效果。" — Sarah Sheikh，武田神经科学治疗领域负责人 从"跟跑"到"并跑"再到"首发"，中国医药创新的脚步从未停歇。TAK-861的中国首报首发，是一个信号：中国患者，正在越来越早，甚至率先用上全球最前沿的创新药。 参考来源： 1. NMPA官方公告，2026年7月21日2. Takeda, "New Pivotal Study Data Show Oveporexton Improved Daily Function, Cognition and Nighttime Sleep for People with Narcolepsy Type 1", 2026年6月15日3. Dauvilliers Y, et al. Efficacy and Safety of Oveporexton (TAK-861) for the Treatment of Narcolepsy Type 1: Results from a Phase 3 Randomized Study. Neurology. 2026;106(11 Suppl 1)4. Mitsukawa K, et al. TAK-861, a potent, orally available orexin receptor 2-selective agonist. Sci Rep. 2024;14:208385. Takeda, "Takeda Announces Positive Results from Two Pivotal Phase 3 Studies of Oveporexton", BusinessWire, 2025年7月14日 📌 更多医药行业深度解读，关注公众号查看~

SOUTH SAN FRANCISCO, Calif., June 30, 2026 (GLOBE NEWSWIRE) -- Quince Therapeutics, Inc. (Nasdaq: QNCX), a clinical stage biopharmaceutical company focused on the development of novel, disease modifying therapies for serious underserved diseases, today announced the formation of a Scientific Advisory Board (SAB) of leading clinicians to support the advancement of LAM-001, an inhaled formulation of rapamycin (mTOR inhibitor) designed to enhance pulmonary delivery and reduce systemic exposure. Comprised of internationally recognized experts in pulmonary medicine, the SAB will be instrumental in progressing the development of LAM-001 across multiple pulmonary indications including pulmonary hypertension associated with interstitial lung disease (PH-ILD), bronchiolitis obliterans syndrome post lung transplant (BOS), and sarcoidosis associated pulmonary hypertension (SAPH). “I am honored to serve as the Chairman of Quince’s Scientific Advisory Board to help guide the development of LAM-001 across a range of pulmonary conditions where evidence strongly suggests it has a direct effect on the underlying disease pathophysiology,” commented Paul Yu, M.D., Ph.D., Director, Cardiovascular Research Center, Massachusetts General Hospital. “On behalf of the SAB, we are encouraged by the clinically relevant improvement seen in measures of lung function in patients with functional class III pulmonary hypertension treated with LAM-001. With continued development, we believe LAM-001 has the potential to provide patients with a much needed treatment option that, importantly, may be utilized in conjunction with currently available therapies to achieve better health outcomes.” Scientific Advisory Board members: Paul Yu, M.D., Ph.D. (Chairman of SAB) Dr. Paul Yu is Director of the Cardiovascular Research Center at Massachusetts General Hospital and Associate Professor of Medicine at Harvard Medical School, where he holds the Charles and Elizabeth Sanders Endowed Chair. A physician-scientist trained in immunology and cardiovascular medicine, Dr. Yu’s research focuses on BMP and TGF-β signaling in cardiovascular homeostasis, repair, and disease. His work spans pulmonary vascular disease, cardiovascular rheumatology, and rare disorders such as fibrodysplasia ossificans progressiva. Dr. Yu has translated several scientific discoveries into therapeutics, including contributions to sotatercept for pulmonary arterial hypertension and the clinical repositioning of saracatinib for fibrodysplasia ossificans progressiva (FOP). He has published over 120 peer-reviewed studies and has been elected to the American Society for Clinical Investigation. Aaron Waxman, M.D., Ph.D. Dr. Aaron Waxman is Director of the Pulmonary Vascular Disease Program at Brigham and Women's Hospital and Associate Professor of Medicine at Harvard Medical School. Dr. Waxman’s clinical practice and research programs center on pulmonary arterial hypertension (PAH), right heart failure, thromboembolic disease, and the broader spectrum of pulmonary vascular disease. Across local and national efforts, Dr. Waxman has served as Principal Investigator on more than 15 clinical trials in pulmonary hypertension. These include the Phase 2 SPECTRA trial of sotatercept, which helped establish activin signaling inhibition as a novel therapeutic pathway in PAH, and the INCREASE trial of inhaled treprostinil in pulmonary hypertension associated with interstitial lung disease—a study that defined a new treatment standard for a previously underserved patient population. He has authored more than 180 peer-reviewed publications, and his research has been supported by the National Institutes of Health, including the National Heart, Lung, and Blood Institute. Steven Nathan, M.D., F.C.C.P. Dr. Steven Nathan is Schar Chair of the Advanced Lung Disease and Lung Transplant Program, Inova Health System and Professor of Education at the University of Virginia. The author of more than 500 publications and co-editor of two books on idiopathic pulmonary fibrosis, Dr. Nathan is internationally recognized for his expertise in advanced lung diseases. Dr. Nathan is a reviewer for multiple journals and is on the editorial board for the journal, Thorax. He has served on multiple committees, including U.S. Food and Drug Administration advisory boards as well as steering committees for clinical trials in IPF and pulmonary hypertension, where he has also served as chair. Dr. Nathan is the recipient of the 2023 American College of Chest Physicians’ 2023 College Medalist Award, and is a member of several professional medical associations, including the American Thoracic Society, the American College of Chest Physicians, and the International Society for Heart and Lung Transplantation. Steve Hays, M.D. Dr. Steven R. Hays is Medical Director of the UCSF Advanced Lung Disease and Lung Transplant Program and Professor of Medicine at the University of California, San Francisco. Under his leadership, the program has grown to one of the nation’s largest transplant centers while consistently achieving superior outcomes, receiving INTERLINK’s Chairman’s Award for Transplant Excellence for three consecutive years. Dr. Hays brings deep clinical expertise across the full spectrum of advanced lung disease, including cystic fibrosis, alpha-1 antitrypsin deficiency, interstitial lung disease, and emphysema. A dedicated clinical investigator, Dr. Hays has served as principal investigator on NIH-funded, industry-sponsored, and investigator-initiated trials. His research portfolio spans lung transplant immunology, chronic lung allograft dysfunction, infectious disease, and transplant digital health. He has authored more than 110 peer-reviewed publications and has been an invited speaker at the American Thoracic Society, the International Society of Heart and Lung Transplantation, and the American Society of Transplantation, among others. He is a Fellow of the American College of Chest Physicians, and a member of the American Thoracic Society and the International Society of Heart and Lung Transplantation, where he serves on the Pulmonary and Infectious Disease Councils. He is a recipient of the American Thoracic Society’s Outstanding Clinician Award and a member of the prestigious UCSF Council of Master Clinicians. Robert Baughman, M.D. Dr. Baughman is Professor Emeritus at the University of Cincinnati. Along with his longtime collaborator Dr. Elyse Lower, he has performed numerous investigator initiated trials of novel treatments for sarcoidosis, including methotrexate, thalidomide, leflunomide, infliximab, rituximab, repository corticotropin injection, pirfenidone, roflumilast, and armodafinil. For sarcoidosis associated pulmonary hypertension, he has conducted trials on bosentan, iloprost, riociguat, tadalafil, selexipag, and inhaled nitric oxide. His publications include over 350 original papers and over 70 review articles and/or book chapters. Additionally, Dr. Baughman has edited multiple books on sarcoidosis and interstitial lung disease. A recognized leader in his field, Dr. Baughman led a multi-national registry of sarcoidosis associated pulmonary hypertension (ReSAPH) and is past president of the World Association of Sarcoidosis and Other Granulomatous Disorders (WASOG). About LAM-001 LAM-001 is a proprietary, investigational, once-daily inhaled formulation of sirolimus, also known as rapamycin. LAM-001’s potential as a disease-modifying agent in pulmonary hypertension stems from its ability to inhibit mTOR-mediated pulmonary arterial smooth muscle cell proliferation. The mTOR pathway has been shown to be activated in the pulmonary arterial smooth muscle cells of patients with pulmonary hypertension, and mTOR inhibition with rapamycin has been shown to reverse smooth muscle cell hyperproliferation and attenuate pulmonary vascular remodeling and cardiopulmonary dysfunction in multiple nonclinical models. Additionally, mTOR signaling promotes fibroblast activation, myofibroblast differentiation, and extracellular matrix deposition in injured or inflamed lung tissue, and mTOR inhibition has been shown to exert direct anti-fibrotic activity, reducing collagen accumulation, suppressing profibrotic cytokine signaling, and attenuating parenchymal fibrosis. These effects are particularly relevant in PH-ILD, where vascular remodeling and progressive fibrosis evolve in parallel and amplify pulmonary vascular load. LAM-001 is designed to enhance pulmonary delivery and reduce systemic exposure, offering a promising potential disease-modifying therapy for pulmonary disease. LAM-001 is currently being studied in multiple indications including PH-ILD, a serious and progressive condition affecting an estimated ~86K patients in the U.S. and ~120K in Europe. Based on compelling Phase 2a data presented at the American Thoracic Society (ATS) in May 2026, the company is advancing LAM-001 into a Phase 2b trial in PH-ILD, with initiation planned for mid-2026 and data anticipated in the first quarter of 2028. LAM-001 is also being evaluated in a Phase 2 study in BOS, a serious complication following lung transplantation affecting an estimated ~17K patients in the U.S. and ~11K in Europe. The trial is fully enrolled, with data anticipated in the first quarter of 2027. In late 2026, the company also plans to initiate a Phase 2 study of LAM-001 in SAPH, a severe complication of sarcoidosis with no approved therapy affecting an estimated ~60K patients in the U.S. and Europe. About Quince Therapeutics, Inc. Quince Therapeutics, Inc. is committed to transforming the lives of patients facing serious, underserved diseases by developing disease-modifying therapies to treat their conditions. The company is currently developing LAM-001 for the treatment of PH-ILD, BOS, and SAPH. A Phase 2a study in PH patients has been completed, a Phase 2 clinical study in BOS patients is ongoing, a Phase 2b study in PH-ILD is anticipated to begin in mid-2026, and a Phase 2 study in SAPH is anticipated to begin in late-2026. By pioneering innovative approaches, the company aims to offer new hope and improved quality of life to patients worldwide. Forward Looking Statements Statements in this news release contain “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 as contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are subject to the “safe harbor” created by those sections. All statements, other than statements of historical facts, may be forward-looking statements. Forward-looking statements contained in this news release may be identified by the use of words such as “believe,” “may,” “should,” “expect,” “aim,” “anticipate,” “plan,” “believe,” “estimated,” “potential,” “intend,” “will,” “can,” “seek,” or other similar words. Examples of forward-looking statements include, among others, statements relating to expectations that the SAB will be instrumental in progressing the development of LAM-001 across multiple pulmonary indications; the design and potential benefits of LAM-001, including as a disease-modifying therapy for pulmonary disease and as a treatment option that may be utilized in conjunction with currently available therapies to achieve better health outcomes; anticipated regulatory and development processes and timelines, including the expected timing to initiate the planned Phase 2b trial of LAM-001 in PH-ILD and the planned Phase 2 trial of LAM-001 in SAPH, the expected timing for data readouts from the ongoing Phase 2 trial of LAM-001 in BOS and the planned Phase 2b trial of LAM-001 in PH-ILD; the Phase 2a data of LAM-001 in PAH and PH-ILD supporting continued development of LAM-001 in PH-ILD and the potential benefit across exercise capacity, pulmonary hemodynamics, cardiac stress and lung function; observed improvements in 6MWD and PVR in the Phase 2a trial potentially suggesting broader effects on cardiopulmonary physiology; the estimated patient populations in the U.S. and Europe for PH-ILD, BOS and SAPH; and the potential advantages of mTOR inhibitors in PH-ILD. Forward-looking statements are based on Quince’s current expectations and are subject to inherent uncertainties, risks, and assumptions that are difficult to predict and could cause actual results to differ materially from what the company expects. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. Factors that could cause actual results to differ include, but are not limited to, clinical results may not be indicative of results that may be observed in the future, including in larger populations; potential safety and other complications related to LAM-001; the ability to obtain and maintain regulatory approval; competition in the company’s industry; the scope, progress and expansion of developing LAM-001; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis alternative therapies; the company’s ability to attract or retain key management, members of the board of directors and other personnel; the impacts of general macroeconomic and geopolitical conditions on the company’s business and financial position; and other risks and uncertainties described in the section titled “Risk Factors” in the company’s Annual Report on Form 10-K, filed with the Securities and Exchange Commission (SEC) on April 10, 2026, and other reports as filed with the SEC. Forward-looking statements contained in this news release are made as of this date, and Quince undertakes no duty to update such information except as required under applicable law. Contacts Corey Davis, Ph.D. LifeSci Advisors, LLC cdavis@lifesciadvisors.com 212-915-2577