Pimicotinib Hydrochloride

近日，国家药监局通过优先审评审批程序批准2款1类创新药上市。 国家药监局批准了默克雪兰诺（北京）医药有限公司申报的盐酸匹米替尼胶囊（商品名：贝捷迈）上市，用于治疗手术切除可能会导致功能受限或出现较严重并发症的症状性腱鞘巨细胞瘤（TGCT）成年患者。另外，批准了Cytokinetics, Incorporated申报的阿夫凯泰片（商品名：星舒平）上市，用于治疗纽约心脏协会（NYHA）心功能分级II-III级的梗阻性肥厚型心肌病（HCM）成人患者，以改善运动能力和症状。 上述创新药品上市，为相关患者提供了新的治疗选择。 来源：国家药监局网站

基于全球III期MANEUVER研究的积极数据，中国国家药品监督管理局（NMPA）批准了贝捷迈®（盐酸匹米替尼胶囊）的全球首个新药上市申请，也是中国首个自主研发的腱鞘巨细胞瘤（TGCT）系统性治疗药物。默克作为我们的合作伙伴，拥有贝捷迈®在全球的商业化权益。 贝捷迈®是和誉医药自主研发的1类新药，上市申请（NDA）获受理后仅6个多月即实现零发补批准。期间顺利通过国家药品审评检查中心（CFDI）首次赴海外临床中心开展的现场核查，不仅体现了和誉医药研发体系的严格标准，也彰显了监管机构对贝捷迈®临床价值和数据质量的高度认可。 贝捷迈®获批用于治疗TGCT，这标志着公司从开创性的创新研究迈向首个产品商业化的关键里程碑。 2025年12月22日，上海和誉生物医药科技有限公司（以下简称“和誉医药”，港交所代码：02256）今日宣布，其自主研发的CSF-1R高选择性小分子抑制剂贝捷迈®（盐酸匹米替尼胶囊）获得中国国家药品监督管理局（NMPA）批准，适用于手术切除可能会导致功能受限或出现较严重并发症的症状性腱鞘巨细胞瘤（TGCT）成年患者。获批后，默克将尽快推动该产品惠及中国TGCT患者。 腱鞘巨细胞瘤是一种罕见的局部侵袭性肿瘤，主要累及关节、腱鞘及滑囊，可导致严重的局部病变和功能障碍。其发生机制为肿瘤性滑膜细胞中过度表达集落刺激因子1（CSF-1），从而导致表达集落刺激因子1受体（CSF-1R）的炎症细胞在肿瘤中大量聚集。《中国腱鞘巨细胞瘤临床诊疗专家共识》明确指出，对于不可切除的TGCT，推荐采用CSF-1/CSF-1R通路抑制剂进行治疗（推荐等级：1级；证据等级：A级）。 “长期以来，中国有许多TGCT患者缺乏手术之外的治疗选择，而手术可能无法满足肿瘤复发或无法切除的患者的治疗需求。”北京积水潭医院骨与软组织肿瘤诊疗中心主任牛晓辉教授表示，“此次基于MANEUVER研究结果获批的贝捷迈®，为中国临床医生提供了一种有效且耐受性良好的系统性治疗方案，有望显著改变该疾病的临床治疗格局。” 贝捷迈®是一款由和誉医药独立研发的新型、可口服、高选择性且高效的小分子CSF-1R抑制剂，本次获NMPA批准基于全球III期MANEUVER研究的积极结果。研究显示，贝捷迈®在所有TGCT系统性治疗III期研究中，实现了迄今基于RECIST v1.1标准评估的最高客观缓解率（ORR）。第25周时，经盲态独立评审委员会（BIRC）基于RECIST v1.1标准评估，贝捷迈®在主要终点ORR上显著优于安慰剂对照组（54.0% vs. 3.2%，P＜0.0001）。在多项与患者生活质量密切相关的关键次要终点上，贝捷迈®均取得了具有临床意义的显著改善，包括提升关节活动度（p=0.0003）、改善躯体功能（PROMIS-PF；p=0.0074），以及减轻最严重僵硬感（p<0.0001）和最严重疼痛（p<0.0001）。2025年欧洲肿瘤内科学会（ESMO）大会上公布的长期随访数据进一步表明，贝捷迈®可以为TGCT患者带来强劲而持久的肿瘤缓解疗效。中位随访14.3个月时，自研究开始即接受贝捷迈®治疗的患者，ORR从第25周的54%显著提升至76.2%（95% CI：63.8，86.0），其中4例实现完全缓解。 和誉医药创始人兼首席执行官徐耀昌博士表示：“此次获批不仅是一个监管里程碑，更是和誉医药发展历程中具有决定性意义的时刻。它标志着我们从科学创新的开拓者，成长为能为患者带来突破性疗法的企业。秉持着解决真实临床需求的坚定信念，我们将继续推进具有突破性和差异化优势的药物研发，以期重塑中国乃至全球肿瘤及其他疾病领域的治疗格局。贝捷迈®作为中国首个自主研发获批的CSF-1R抑制剂，不仅填补了腱鞘巨细胞瘤（TGCT）治疗的关键空白，更印证了我们以全球视野践行创新理念，将前沿构想转化为临床价值的决心与能力。” 默克医药健康首席执行官、默克执行董事会成员丹卓航表示: “随着贝捷迈®在全球的首个注册获批，我们正持续践行改善罕见肿瘤患者生活的承诺。此次获批更是进一步夯实我们在罕见肿瘤领导地位的关键一步。我们为患者带来了既能改变疾病进程、又可缓解日常症状的希望。我们正在加速推进贝捷迈®早日惠及中国患者，并持续推进其在全球其他市场的注册布局。” 在MANEUVER研究中，贝捷迈®还展现出良好的安全性和耐受性。治疗期间，患者的中位剂量强度保持在88.2%的较高水平。大多数治疗相关不良事件（TEAEs）为1-2级，未观察到新的安全性信号，亦无胆汁淤积性肝毒性、药物性肝损伤或毛发/皮肤色素减退等相关证据。 关于贝捷迈®（盐酸匹米替尼胶囊） 贝捷迈®是由和誉医药独立研发的一款新型、口服、高选择性且高效的小分子CSF-1R抑制剂，其在全球III期MANEUVER研究中针对腱鞘巨细胞瘤（TGCT）的积极顶线结果已于2024年11月成功发布。目前，贝捷迈®已获中国国家药品监督管理局（NMPA）批准，适用于手术切除可能会导致功能受限或出现较严重并发症的症状性腱鞘巨细胞瘤（TGCT）成年患者。2023年12月，和誉医药与默克公司就贝捷迈®的商业化权利达成协议，默克公司会负责贝捷迈®在全球的商业化。 Abbisko Therapeutics' CSF-1R Inhibitor Pimicotinib Approved by the China NMPA, Addressing the Treatment Gap for Tenosynovial Giant Cell Tumor Based on the positive global Phase III MANEUVER study, the National Medical Products Administration (NMPA) has granted the world's first regulatory approval for pimicotinib, the first domestically developed systemic therapy for the treatment of Tenosynovial Giant Cell Tumor (TGCT), to our partner Merck KGaA, Darmstadt, Germany, which holds worldwide commercialization rights. Pimicotinib, a Class 1 innovative drug discovered and developed by Abbisko Therapeutics, received zero-deficiency approval in just over six months following NDA acceptance. During the review, the Center for Food and Drug Inspection (CFDI) conducted its first-ever overseas clinical site inspection for pimicotinib, which it successfully passed. This outcome reflects Abbisko's rigorous R&D standards and regulators' strong recognition of the therapy's clinical value and data quality. Pimicotinib's approval for the treatment of TGCT marks a key milestone in the company's advancement from pioneering innovative research to first product commercialization. 22 November 2025, Shanghai – Abbisko Therapeutics Co., Ltd. ("Abbisko Therapeutics" hereafter, HKEX code: 02256.HK) today announced that the China National Medical Products Administration (NMPA) has approved pimicotinib for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause functional limitation or severe morbidity. Following this approval, Merck KGaA, Darmstadt, Germany, is working to ensure timely patient access in China. TGCT is a rare and locally aggressive tumor primarily affecting joints, tendon sheaths, and bursae with considerable morbidity and functional impairment. TGCT is driven by overexpression of colony-stimulating factor-1 (CSF-1) in neoplastic synovial cells which leads to accumulation of colony-stimulating factor-1 receptor (CSF-1R)–expressing inflammatory cells in the tumor. The Chinese expert consensus on clinical diagnosis and treatment of TGCT recommends CSF-1/CSF-1R pathway inhibitors as the preferred systemic treatment for patients with unresectable TGCT (Recommendation Level I; Evidence Level A). "Many people living with TGCT in China have faced a long and difficult journey due to the lack of approved options beyond surgery, which may not address the needs of patients whose tumors recur or are not amenable to resection," said Prof. Niu Xiaohui, Director of the Bone and Soft Tissue Tumour Diagnosis and Research Centre at Beijing Jishuitan Hospital. "With the approval of pimicotinib based on the results of the global MANEUVER study, healthcare professionals in China will soon have the opportunity to prescribe their patients an effective and well-tolerated systemic treatment option, offering a much-needed advance in how they manage this challenging condition." Pimicotinib is a novel, oral, highly selective, and potent small-molecule CSF-1R inhibitor independently developed by Abbisko Therapeutics. The approval by the China NMPA is based on the positive results from the global Phase III MANEUVER study, in which pimicotinib demonstrated the highest objective response rate (ORR) based on RECIST v1.1 seen in a Phase III clinical trial for the treatment TGCT. At week 25, pimicotinib demonstrated a statistically significant improvement in the primary endpoint of ORR compared with placebo (54.0% vs. 3.2%, respectively; p<0.0001) as assessed by Blinded Independent Review Committee (BIRC) based on RECIST v1.1. pimicotinib also demonstrated clinically meaningful and statistically significant improvements across secondary endpoints relevant to patient daily life, improving relative Range of Motion (p=0.0003) and physical function as measured by the PROMIS-PF scale (p=0.0074), as well as reducing Worst Stiffness NRS (p<0.0001) and BPI Worst Pain NRS (p<0.0001). Longer-term follow-up data presented at the 2025 European Society for Medical Oncology (ESMO) Congress further demonstrated that pimicotinib provides robust and durable tumor responses for patients with TGCT. With a median follow-up of 14.3 months, ORR per RECIST v1.1 increased from 54% at week 25 to 76.2% (95% CI: 63.8, 86.0), including four patients achieving a Complete Response (CR). Dr. Yaochang Xu, Founder and Chief Executive Officer of Abbisko Therapeutics, stated: "The approval of pimicotinib is far more than a regulatory milestone—it is a defining moment in Abbisko's journey. It reflects our evolution from pioneers in scientific innovation to a company capable of bringing meaningful therapies to patients. Guided by an unwavering commitment to addressing real clinical needs, we will continue advancing breakthrough, differentiated medicines that have the potential to reshape the future of oncology and other disease areas in China and around the world. As the first domestically independently developed CSF-1R inhibitor approved in China, pimicotinib not only fills a crucial gap for patients with TGCT, but also exemplifies what we can achieve when we pursue bold ideas with global ambition and a relentless focus on impact." "We are continuing to deliver on our commitment to improving the lives of patients with rare tumors with this first-in-the-world regulatory approval of pimicotinib," said Danny Bar-Zohar, CEO Healthcare and Member of the Executive Board of Merck KGaA, Darmstadt, Germany. "This approval is a significant step forward in further strengthening our leadership in rare tumors, while offering patients the opportunity to change the course of their disease and help alleviate symptoms that impact their daily lives. We are now working to make pimicotinib available to patients in China as quickly as possible, as we continue to progress applications with regulatory authorities in additional markets." In the MANEUVER study, pimicotinib also demonstrated a generally well-tolerated safety profile. The median dose intensity remained high at 88.2% during treatment. Most treatment-emergent adverse events (TEAEs) were Grade 1-2. No new safety signals were observed with longer-term follow-up, and there was no evidence of cholestatic hepatotoxicity, drug-induced liver injury, or hair/skin hypopigmentation. About Pimicotinib Pimicotinib is a novel, oral, highly selective, and potent small-molecule CSF-1R inhibitor independently developed by Abbisko Therapeutics. Positive results from the global Phase III MANEUVER study of pimicotinib for the treatment of Tenosynovial Giant Cell Tumor (TGCT) were announced in November 2024. Currently, pimicotinib has been approved by the National Medical Products Administration (NMPA) in China for the treatment of adult patients with symptomatic TGCT for which surgical resection may potentially cause functional limitation or relatively severe morbidity. In December 2023, Abbisko entered into an agreement with Merck KGaA, Darmstadt, Germany, pertaining to the commercial rights to pimicotinib, pursuant to which Merck KGaA, Darmstadt, Germany, is responsible for the commercialization of pimicotinib globally. Outside of China, pimicotinib has been granted Breakthrough Therapy Designation by the US Food and Drug Administration (FDA) and PRIME Designation by the European Medicines Agency (EMA). About Abbisko Therapeutics Founded in April 2016, Abbisko Therapeutics Co., Ltd. (HKEX: 02256.HK), is an oncology-focused biopharmaceutical company based in Shanghai that is dedicated to the discovery and development of innovative medicines to treat unmet medical needs in China and globally. The Company was established by a group of seasoned drug hunters with rich research & development and managerial expertise from top multinational pharmaceutical companies. Since its founding, Abbisko Therapeutics has built an extensive pipeline of innovative programs focused on precision oncology and immuno-oncology. Please visit www.abbisko.com for more information. 关于和誉 和誉医药（香港联交所代码：02256）成立于2016年，是一家立足中国，着眼全球的创新药研发公司。公司的创始人和管理团队拥有多年顶尖跨国药企的研发和管理经验，并参与了多个临床及上市新药的研发。和誉医药专注于肿瘤新药研发，以小分子肿瘤精准治疗和小分子肿瘤免疫治疗药物为核心，着眼病患及医药市场的需求，秉承国际新药开发的理念和标准，致力于开发新颖及高潜力药物靶点的潜在first-in-class或best-in-class创新药物，用于改善中国及全球病人的生活质量。自成立以来，和誉医药已经建立了丰富的创新产品管线，涵盖肿瘤精准治疗领域以及肿瘤免疫治疗领域。 更多信息，欢迎访问 www.abbisko.com。