Fitusiran

2025 年前三个季度，79 款新药首次获 NMPA 批准上市，包括全球首个获批的 GCG/GLP-1 双受体激动减重药物玛仕度肽、全球首个高选择性外周 κ 阿片受体激动剂镇痛药安瑞克芬、国产首个 EZH2 抑制剂泽美妥司他片... Q4，又有哪些重磅新药会在国内获批上市呢？ 本文将根据 Insight 数据库「中国上市策略& 时长预测」模块，选出其中 10 款重磅新药（排名不分先后）进行分享，仅供读者参阅。 信达生物 匹康奇拜单抗 2024 年 9 月 26 日，信达生物匹康奇拜单抗注射液（研发代号：IBI112）的新药上市申请获 CDE 受理，用于治疗中重度斑块状银屑病。 此次报上市是基于一项在中国中重度斑块状银屑病受试者中开展的 III 期注册临床研究 CLEAR-1（NCT05645627）的积极结果。 该研究于 2024 年 5 月顺利达成主要研究终点和全部关键次要研究终点，研究显示匹康奇拜单抗组达成皮损清除（PASI 90、PASI 75、PASI 100、sPGA 0/1 和 sPGA 0）和生活质量改善（DLQI 0/1）的受试者比例均显著优于安慰剂组。研究治疗期间，匹康奇拜单抗整体安全性良好，未发现新的安全性信号。 匹康奇拜单抗是全球首个注册 III 期临床首要研究终点中第 16 周达到 PASI 90 的受试者比例突破 80% 的 IL-23p19 抗体药物，同时在同类生物药中具有最长的维持期给药间隔（每 12 周一次），有望为中国中重度斑块状银屑病患者带来皮损清除、生活质量改善和用药便利性提升等多方面的综合获益。 除了 CLEAR-1 以外，匹康奇拜单抗当前还开展了多项临床研究，包括： 在中重度银屑病患者中开展的随机撤药再治疗的 III 期临床； 在中重度银屑病患者中开展生物制剂治疗转匹康奇拜单抗治疗的 II 期临床； 在中重度活动性溃疡性结肠炎患者中开展的 II 期临床。 恒瑞医药 瑞拉芙普-α 2024 年 9 月 20 日，恒瑞 1 类新药瑞拉芙普-α 注射液（研发代号：SHR-1701）的上市申请获 CDE 受理，适应症为联合氟尿嘧啶类和铂类药物用于局部晚期不可切除、复发或转移性胃及胃食管结合部腺癌（G/GEJA）的一线治疗。 SHR-1701 是恒瑞医药自主研发的一种抗 PD-L1/TGF-βRII 双功能融合蛋白。在剂量递增、剂量扩展和临床扩展的 I 期研究中，SHR-1701 单药用于胃癌末线人群显示出良好的抗肿瘤活性。在此基础上，恒瑞开展了一项 SHR-1701 联合化疗用于晚期 G/GEJA 的 III 期临床研究。 在 2024 年 ESMO 大会上，恒瑞首次以口头报告的形式公布了此项 III 期临床的结果。数据显示，SHR-1701 所取得的疗效数据显著，尤其是在降低疾病进展和死亡风险层面（HR 值）： 在意向治疗人群（ITT）中的中位 OS 为 15.8 个月（vs 安慰剂组 11.2 个月），HR 为 0.66，降低 34% 的疾病进展或死亡风险； 在 PD-L1 CPS≥5 人群中，中位 OS 为 16.8 个月（vs 安慰剂组 10.4 个月） ，HR 为 0.53，降低 47% 的疾病进展或死亡风险。 截图来源：Insight 数据库 诺和诺德 帕西生长素 2024 年 9 月 5 日，诺和诺德帕西生长素在国内申报上市。根据公开信息和临床试验进展，Insight 数据库推断本次申报的适应症为用于治疗因生长激素分泌不足而导致生长缓慢的儿童患者。 帕西生长素（Somapacitan）是一种每周一次长效人生长激素蛋白衍生物。与现有的每日一次生长激素相比，帕西生长素可以将每年的治疗次数从 365 次减少至 52 次，大大提升患者治疗的便利性和依从性。 目前该药已在美国、欧盟、日本等海外国家和地区获批，用于治疗儿童和成人的生长激素缺乏症。在中国，诺和诺德开展了两项帕西生长素研究，其中治疗因生长激素分泌不足而导致生长缓慢的儿童患者的 III 期临床研究已经完成。 截图来源：Insight 数据库 值得一提的是，帕西生长素已经于今年 4 月率先落地海南博鳌乐城国际医疗旅游先行区，用于在生长激素缺乏症所致生长迟缓的 3 岁及以上儿童和青少年中替代内源性生长激素，并在 5 月 24 日开出中国首张处方。 和美药业 莫米司特-Hemay005 2024 年 4 月 24 日，和美药业 1 类新药莫米司特-Hemay005 上市申请获 CDE 受理，用于中至重度斑块状银屑病。 在 2023 年美国风湿病学会年会（ACR）上，莫米司特治疗中至重度慢性斑块状银屑病的 III 期临床（NCT04839328）结果已公布。结果显示，莫米司特和安慰剂组 PASI75 比例分别为 53.6% vs 16.0%，sPGA 0/1 率分别为 31.3% vs 6.4%。 Insight 数据库显示，目前国内共有 2 款 PDE4 抑制剂获批上市，分别是阿普米司特（印度太阳制药/安进）和克立硼罗乳膏（辉瑞）。大冢制药的地法米司特、和美药业的莫米司特-Hemay005 均处于上市审评阶段，后者是首个国产 PDE4 抑制剂。 截图来源：Insight 数据库 此外，国内方面，恒瑞、中国生物制药、华东医药、济川药业、健康元等企业均在布局 PDE4 赛道，总共有 5 款 PDE4 抑制剂已进入 III 期临床。 GSK 玛贝兰妥单抗 2024 年 12 月 7 日，GSK 的 BCMA ADC 药物玛贝兰妥单抗在中国申报上市，玛贝兰妥单抗联合硼替佐米加地塞米松（BVd）治疗复发或难治性多发性骨髓瘤。 该申请基于 III 期头对头 DREAMM-7 试验的中期结果。数据显示，该试验达到了其主要终点，显示与达雷妥尤单抗联合硼替佐米和地塞米松（DVd）治疗复发或难治性多发性骨髓瘤相比，BVd 方案在 PFS 方面具有统计学显著和临床意义的改善。 在后续计划的中期分析中，DREAMM-7 试验达到了 OS 这一关键次要终点，表明与标准治疗 DVd 方案相比，BVd 方案显著降低了死亡风险。 CytokineticsAficamten 2024 年 10 月 18 日，箕星药业宣布 CDE 已正式受理艾非凯泰（Aficamten）用于治疗症状性梗阻性肥厚型心肌病（oHCM）的新药上市申请。 Aficamten 是一款在研的新一代选择性小分子心肌肌球蛋白抑制剂，其治疗 oHCM 获 FDA 和 NMPA 授予的突破性治疗药物认定。 2020 年 7 月，箕星与 Cytokinetics 就该药在大中华区的研发和商业化签订了独家许可协议。2024 年 12 月，箕星药业与赛诺菲共同宣布签订正式协议，协议约定赛诺菲将收购箕星在大中华区独家开发和商业化 Aficamten 的权益。 该新药上市申请是基于关键性全球 III 期临床研究 SEQUOIA-HCM 所取得的积极结果。研究结果表明，与安慰剂相比，Aficamten 治疗能显著提高患者的运动能力，治疗效果在所有预设亚组中都是一致的，这些亚组反映了患者的基线特征和治疗策略，包括接受或未接受作为背景治疗的 β-受体阻滞剂的患者。在所有 10 个预先设定的次要终点中，也观察到具有统计学意义和临床意义的改善。 辉瑞 马塔西单抗 2024 年 8 月 13 日，马塔西单抗（Marstacimab、PF-6741086）在国内报上市，这是辉瑞研发的一种血友病新型疗法，是一种靶向组织因子途径抑制物（TFPI）K2 结构域的单克隆抗体，属于血友病再平衡疗法。目前马塔西单抗已在美国和欧盟获批用于治疗血友病 A 及血友病 B。 马塔西单抗的获批是基于 III 期 BASIS 研究。该研究不伴抑制物队列的中期分析结果显示，在不伴抑制物的血友病 A 及血友病 B 受试者中，经过马塔西单抗 12 个月的积极治疗期后，与既往因子预防（RP）和按需治疗 (OD) 相比，其经治疗出血的年化出血率 (ABR) 分别降低了 35% 和 92%。 2025 年 2 月，马塔西单抗正式落地海南博鳌乐城先行区，通过预充式注射笔皮下注射固定剂量给药，只需每周一次，有望减少血友病患者因频繁输注带来的心理负担和用药负担。 精准生物 普基仑赛 2024 年 7 月 20 日，重庆精准生物自主研发的国家一类生物新药 pCAR-19B 细胞自体回输制剂（普基仑赛注射液）上市申请获 NMPA 正式受理，该产品是国内首款针对儿童白血病的 CAR-T 产品，用于治疗 3-21 岁患有 CD19 阳性复发/难治性急性淋巴细胞白血病（ALL）的患者。 在 2024 年 ASH 大会上，该药用于 CD19 阳性 R/R B-ALL 的 II 期临床结果首次被披露。中位随访 211 天，最佳 ORR 为 90.63%，其中 78.13% 的患者达到 CR，12.5% 的患者达到 CRi，超过了当前商业化抗 CD19 CAR-T 疗法产品在 R/R B-ALL 中报道的疗效。 此外，98.27% 的 ORR 患者获得了阴性微小残留病（MRD）阴性缓解。3 个月 ORR 为 76.56%。中位缓解持续时间（DOR）为 10.61 个月，中位总生存期（OS）为 23.92 个月。 根据 Insight 数据库，目前国内已有 4 款靶向 CD19 的 CAR-T 产品获批上市，分别为恒润达生的雷尼基奥仑赛、复星凯瑞引进的的阿基仑赛、药明巨诺的瑞基奥仑赛、合源生物的纳基奥仑赛。普基仑赛（精准生物）、IM19（艺妙神州/先声）、布瑞基奥仑赛（吉利德/复星凯瑞）均处于上市审评阶段。 截图来源：Insight 数据库 Alnylam/赛诺菲 芬妥司兰钠 2024 年 5 月 7 日，siRNA 疗法芬妥司兰钠上市申请获 CDE 受理，拟用于作为常规预防治疗，用于有或无凝血因子 VIII 或 IX 抑制物的血友病 A 或 B 的成人患者和 ≥12 岁青少年患者，以预防或减少出血的发生频率。 芬妥司兰钠来自于 Alnylam，2018 年 8 月，赛诺菲和 Alnylam 公司达成合作，获得 Fitusiran 的全球开发和商业化权利。该药采用 Alnylam Pharmaceutical 的 ESC-GalNAc 偶联技术，因此在皮下给药时具有更强的效力和持久性。 2025 年 3 月 28 日，该药在美国率先获批，商品名为 Qfitlia，成为美国首个获批的治疗 A 型或 B 型血友病（无论是否含有抑制剂）的疗法，每年只需注射六次即可提供持续保护，代表着血友病治疗的重要突破。 在中国，该产品还被 CDE 纳入了《以患者为中心的罕见疾病药物研发试点工作计划（「关爱计划」）》试点项目，这是一项旨在罕见疾病药物研发领域落实以患者为中心的药物研发理念的试点工作。 Ascendis/维昇药业隆培促生长素 2024 年 3 月 8 日，维昇药业宣布 NMPA 已受理隆培促生长素的上市许可申请。这是首个在美国及欧洲获批上市的长效生长激素（每周注射一次），用于治疗儿童生长激素缺乏症（PGHD）。 隆培促生长素由 Ascendis Pharma A/S 所有，并在全球范围内开发。2018 年 11 月，维昇药业从 Ascendis 引进了该产品在中国的开发和商业化权益。 2022 年 4 月，维昇药业在中国完成了隆培促生长素治疗 PGHD 的关键 III 期临床试验。 结果显示，隆培促生长素 52 周年化生长速率（AHV）高于短效（每日注射）人生长激素，具有统计学显著差异，并可能使患有 PGHD 的儿童在有限的时间窗口内更有效达到治疗目标。第 52 周时，隆培促生长素 AHV 为 10.66 厘米/年，而短效（每日注射）人生长激素为 9.75 厘米/年（p=0.001）。 如欲了解更多国内新药获批情况，可以扫描文末二维码，前往 Insight 数据库查询。 封面来源：站酷海洛题图 免责声明：本文仅作信息分享，不代表 Insight 立场和观点，也不作治疗方案推荐和介绍。如有需求，请咨询和联系正规医疗机构。 编辑：ccai PR 稿对接：微信 insightxb 投稿：微信 insightxb；邮箱 insight@dxy.cn

iStock, Deagreez As the industry loses one of its key female leaders in GSK CEO Emma Walmsley, BioSpace profiles the women leading the industry’s smaller biopharmas. With the departure of GSK CEO Emma Walmsley, the industry loses one of only two female leaders in charge of a top 20 pharma company—the other being Vertex’s Reshma Kewalramani. But as you move down the list into the world of smaller biopharmas, you see many more female faces. Ranking companies by market cap, BioSpace took a look at the top women in charge. Vertex topped the list with an estimated worth of $94.3 billion. Following Kewalramani is Alnylam’s Yvonne Greenstreet, who leads the RNAi specialist that’s valued at $60.3 billion. Below Greenstreet is Akeso’s Michelle Xia, who BioSpace profiled last month as part of a special focus on Chinese biotech. Editorial GSK CEO Emma Walmsley’s Departure Reseals Pharma’s Glass Ceiling After Emma Walmsley steps down as GSK CEO in January, Vertex Pharma’s Reshma Kewalramani will be the sole female CEO at a top-20 pharma company. Still, there are many prominent women in pharma that could someday break through again. September 29, 2025 · 1 min read · Annalee Armstrong Read More We would also be remiss if we didn’t mention incoming Takeda CEO Julie Kim , who will take the reins next summer. While she isn’t included this time around, her succession to the top of the C-suite will mark a pivotal moment for the industry. Read on for profiles of the top five women in charge of the world’s most valuable biopharmas. Maybe one day we’ll see them climb even higher. Reshma Kewalramani, Vertex CEO Courtesy Vertex, Dina Rudick/Anthem Multimedia Reshma Kewalramani Company: Vertex Pharmaceuticals Value: $94.3 billion A physician by trade, Reshma Kewalramani was trained in internal medicine and nephrology at Massachusetts General Hospital and Brigham and Women’s Hospital. She jumped to pharma in 2004, spending 12 years at Amgen until she rose to vice president and head of the U.S. Medical Organization. Kewalramani’s trip to the CEO chair was fairly quick once she arrived at Vertex in February 2017 as a senior vice president. A little over a year later, she became chief medical officer—a role that sees a woman in charge almost as infrequently as CEO. Another two years later, she was named the chief executive. In her time as Vertex’s top dog, the company’s market cap has risen nearly 80%. She has overseen the broadening of the company’s cystic fibrosis portfolio, including approvals for Trykafta and Alyftrek, both CFTR modulators—a modality that has been credited as the “biggest advance in the past decade” for cystic fibrosis. Vertex has also faced criticism, however, for a lack of access to the lifesaving treatments, which can cost upwards of $300,000 per year. Kewalramani has also overseen a diversification of Vertex’s portfolio to better balance the CF work with other indications. The company has worked with CRISPR Therapeutics since 2017 on a gene therapy for blood disorders. That was approved as Casgevy in December 2023, along with bluebird bio’s Lyfgenia, as the first gene therapies for sickle cell disease. Casgevy is the first CRISPR-based gene editing therapy ever to receive an FDA authorization. And then there is Journavx, which became the first new non-opioid pain medicine in its class to receive an FDA nod in January. Yvonne Greenstreet, Alnylam CEO Courtesy Alnylam Yvonne Greenstreet Company: Alnylam Pharmaceuticals Value: $60.3 billion Yvonne Greenstreet is only the second CEO in Alnylam’s history. After joining in 2016 as chief operating officer, she succeeded founding CEO John Maraganore in January 2022. Prior to Alnylam, Greenstreet spent three years at Pfizer leading a division worth $16 billion, followed by 18 years at GSK, where she served as chief of strategy for research and development and chief medical officer for Europe, among other roles. She received a medical degree from the University of Leeds and was a practicing OB/GYN prior to her pharma days. Alnylam is noted in the industry as a leader in RNAi interference, a gene silencing technology based on Nobel Prize–winning science. Since Greenstreet took the helm, the company has received approvals for cholesterol drug Leqvio, ATTR amyloidosis therapy Amvuttra and Qfitlia for hemophilia. Amvuttra has since been expanded into additional indications. Greenstreet has emerged as a leader on issues facing pharma, such as the drug pricing provisions of the Inflation Reduction Act. She’s also a vocal supporter of biotech achievements—recently celebrating uniQure’s positive data in Huntington’s disease in a LinkedIn post despite the company being Alnylam’s rival in the space. In 2023, Alnylam achieved $1 billion in net product revenue for the first time. The company’s market cap has risen 190% during Greenstreet’s time at the top. Akeso CEO Michelle Xia Courtesy Akeso Michelle Xia Company: Akeso Value: $16.2 billion Michella Xia is overseeing one of China’s most exciting biotechs, which has produced one of the most talked-about cancer drugs. Already approved in China, ivonescimab is now moving through clinical development in the U.S. with partner Summit Therapeutics. Xia never intended to become a CEO. A biochemistry graduate of Sun Yat-sen University, she also picked up postgraduate degrees from Newcastle University and Glasgow University in the U.K., then headed to the U.S. to focus on cancer immunotherapy at the University of Louisville. She joined industry in 2006 as a senior scientist at Bayer, before jumping to a contract research organization focused on oncology and immuno-oncology called Crown Bioscience. When Crown opened a subsidiary in China, Xia returned home and found a whole new world of innovation. Seeing the potential in the homegrown talent, she struck out on her own in 2012, launching Akeso. Despite tough beginnings, Xia has helped grow the biotech into a world-renowned company with much more to offer than just ivonescimab. Akeso’s market cap has climbed 594% since it debuted on the Chinese stock market in 2020. Martine Rothblatt, United Therapeutics CEO Wikimedia Commons, Joe Andrucyk Martine Rothblatt Company: United Therapeutics Value: $15.1 billion Perhaps the CEO with the most diverse background on this list, Martine Rothblatt didn’t start her journey to biotech in the halls of a hospital. She was instead educated in communications satellite law and went on to found what is now known as SiriusXM Satellite Radio. Just two years after the company formed in 1990, she resigned as CEO to create what would become United Therapeutics. The company, which officially emerged in 1996, was started to find a cure for Rothblatt’s daughter, who suffered from what is now known as pulmonary arterial hypertension. United succeeded in its mission to bring a therapy through the FDA, achieving approval for Remodulin in 2002. The drug was followed in 2009 by Tyvaso and Adcirca, the same year United achieved $1 billion in assets. United now has five approved medicines and has expanded its portfolio to include therapies for xenotransplantation via the acquisition of Revivicor’s platform in 2011. Professional achievements aside, Rothblatt has been a champion of LGBTQ rights. She came out as transgender in 1994 and transitioned at age 40, using her platform since then to establish health laws standards for transgender people and fight discriminatory legislation. With the longest tenure on this list, Rothblatt has seen United rise from humble beginnings. The company’s market cap is 15,240% higher than it was in 2000, which is the earliest data available in S&P Capital IQ’s database. Sarah Boyce, Avidity CEO Courtesy Avidity Sarah Boyce Company: Avidity Biosciences Value: $5.2 billion Sarah Boyce has been a biotech leader through-and-through since 2010, when she joined Alexion as vice president of nephrology. Since then, she has climbed the C-suite ladder, ultmately becoming CEO at RNA therapeutics specialist Avidity Biosciences. Boyce took that role in October 2019, working immediately to help secure a $100 million series C that was announced just months later. But she didn’t rest on the fundraising front. Boyce moved quickly to turn that crossover round into a market debut. Avidity moved to go public in June 2020 , seeking $259.2 million. The IPO overshot that, closing at $298.1 million . Avidity now has three therapies in registration-enabling clinical trials for rare muscular disorders. Since debuting, the company’s market cap has risen 530%. Prior to Avidity, Boyce held roles at Ionis Pharmaceuticals, Forest Laboratories, Novartis Oncology and Roche, in addition to Alexion. She has a degree in microbiology from the University of Manchester in England. She’s a member of the Biotech Sisterhood and has signed on to letter writing campaigns to support reproductive rights and the FDA’s regulatory authority. Editor’s Note: This list was developed using S&P Capital IQ’s industry classification for biotechnology but excluding biotechnology companies without a healthcare focus.