Pexidartinib Hydrochloride

On February 14, 2025, Ono Pharmaceuticals secured FDA approval for vimseltinib, a therapy for tenosynovial giant cell tumor (TGCT), which will be marketed as ROMVIMZA. This approval places Ono in direct competition with another Japanese pharmaceutical company, Daiichi Sankyo, in the TGCT market. LAS VEGAS, March 4, 2025 /PRNewswire/ -- Tenosynovial giant cell tumor, also known as pigmented villonodular synovitis (PVNS) or giant cell tumor of the tendon sheath (GCT-TS), is primarily treated through surgical resection. For localized TGCT, the typical approach involves direct removal of the tumor nodule. In contrast, diffused-type TGCT (DTGCT) tends to present as larger tumors affecting major joints, making treatment more complex. Management options for DTGCT often include total synovectomy, joint replacement, or, in extremely rare cases, amputation. Among the 7MM, the United States had the highest prevalence of tenosynovial giant cell tumor, accounting for approximately 45% of total cases. In 2023, localized TGCT in the U.S. was most commonly found in the digits, representing around 75% of cases. Depending on whether the tumor is localized or diffuse, giant cell tumors can also occur in the knee, ankle, hip, and other areas. According to DelveInsight analysts, diffuse TGCT was most frequently observed in the knee, with an estimated 30,000 cases in the US in 2023. The primary approach to treating TGCT is surgery, often supplemented by alternative options like Radiation Therapy, Radiosynovectomy, and Systemic Therapies, including CSF1 inhibitors and Tyrosine Kinase Inhibitors. TURALIO (pexidartinib, previously known as PLX3397) is a targeted therapy developed by Daiichi Sankyo for adult patients with symptomatic tenosynovial giant cell tumor that leads to significant morbidity or functional limitations and cannot be effectively treated with surgery. This orally administered small-molecule tyrosine kinase inhibitor acts on colony-stimulating factor 1 receptor (CSF1R), KIT proto-oncogene receptor tyrosine kinase (KIT), and FMS-like tyrosine kinase 3 (FLT3) with an internal tandem duplication (ITD) mutation. Overexpression of the CSF1R ligand contributes to excessive cell proliferation and accumulation in the synovium. The US FDA approved TURALIO in August 2019 for the treatment of TGCT in adults with severe symptoms and limited surgical options. Before this, in October 2015, pexidartinib received Breakthrough Therapy Designation (BTD) from the FDA for TGCT treatment. TURALIO carries a risk of serious and potentially life-threatening liver toxicity and is available only through a restricted Risk Evaluation and Mitigation Strategy (REMS) program. Learn more about the FDA-approved TGCT drugs @ Drugs for Tenosynovial Giant Cell Tumors Treatment Recently, in February 2025, Ono Pharmaceuticals secured FDA approval for vimseltinib for the treatment of tenosynovial giant cell tumor. Marketed under the brand name ROMVIMZA, the drug places Ono in direct competition with fellow Japanese pharmaceutical company Daiichi Sankyo in the TGCT space. Like TURALIO, which became the first FDA-approved TGCT treatment in 2019, ROMVIMZA is a colony-stimulating factor 1 receptor (CSF1R) inhibitor. As a kinase inhibitor, it blocks the CSF1 receptor to slow the growth of cells expressing this protein, potentially limiting tumor progression in TGCT. Ono Pharmaceuticals acquired ROMVIMZA through its $2.4 billion purchase of Deciphera Pharmaceuticals last year, making the U.S.-based firm a subsidiary. At the time, Ono's CEO, Gyo Sagara, described the acquisition as a strategic effort to expand the company's targeted oncology pipeline, strengthen its presence in the U.S. and European markets, and enhance its kinase drug discovery expertise. To compete with TURALIO's early market entry, Ono is positioning ROMVIMZA as a more convenient TGCT treatment. Unlike Daiichi Sankyo's drug, which requires twice-daily dosing, ROMVIMZA is administered only twice a week, with at least 72 hours between doses. Additionally, while TURALIO carries a boxed warning for hepatotoxicity due to the risk of severe liver damage, ROMVIMZA does not have this safety warning. To know more about TGCT treatment options, visit @ New Treatment for Tenosynovial Giant Cell Tumors TGCT that is not amenable to improvement from surgery. Several companies, such as SynOx Therapeutics (Emactuzumab), AmMax Bio (AMB-05X), Merck KGaA (pimicotinib), and others, are involved in developing therapies for TGCT. Discover which therapies are expected to grab major TGCT market share @ Tenosynovial Giant Cell Tumors Market Report Pimicotinib is an investigational, orally administered small-molecule inhibitor designed to selectively and effectively target the colony-stimulating factor-1 receptor. Developed by Abbisko, it remains under clinical evaluation. In December 2023, Abbisko Therapeutics agreed with Merck, granting Merck exclusive rights to commercialize pimicotinib-based products for all indications in mainland China, Hong Kong, Macau, and Taiwan, with the possibility of expanding to global markets, including the US. Pimicotinib has been granted Breakthrough Therapy Designation (BTD) by both the China National Medical Products Administration (NMPA) and the FDA, as well as Priority Medicine (PRIME) designation by the European Medicines Agency (EMA) for the treatment of patients with TGCT who are not candidates for surgery. In November 2024, Merck reported that the Phase III MANEUVER trial, conducted by Abbisko Therapeutics, met its primary endpoint. The study showed a significant improvement in the objective response rate (ORR) for patients with tenosynovial giant cell tumor. By week 25, pimicotinib achieved an ORR of 54.0%, compared to 3.2% in the placebo group (p<0.0001). Emactuzumab is a monoclonal antibody that serves as a potent and selective inhibitor of CSF-1R. By interacting with two key cytokines, CSF-1 and IL-34, the CSF-1 receptor plays a crucial role in regulating macrophages and related cells across various biological processes and organ systems. This makes it a compelling target with wide-ranging therapeutic potential. SynOx Therapeutics is currently assessing emactuzumab in the Phase III TANGENT clinical trial. In April 2024, the company secured a $35 million loan facility from Hercules Capital, bolstering its financial position as it advances the trial. Additionally, SynOx successfully completed a $75 million Series B funding round. Earlier, in August 2022, the European Medicines Agency (EMA) granted orphan medicinal product designation to emactuzumab for both localized and diffuse TGCT. AMB-05X is a powerful monoclonal antibody targeting CSF1R, designed as a therapeutic platform for severe macrophage-driven inflammatory, fibrotic, and neovascular conditions. It has received Fast Track designation from the FDA and PRIME designation from the EMA for TGCT. Currently, it is being evaluated in a Phase II clinical trial to assess its safety, tolerability, efficacy, and pharmacokinetics when administered via intra-articular injection in TGCT patients. Discover more about drugs for TGCT in development @ Tenosynovial Giant Cell Tumors Clinical Trials The anticipated launch of these emerging therapies for TGCT are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the TGCT market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for TGCT is expected to grow from USD 300 million in 2023, with a significant CAGR by 2034. This growth can be attributed to the approval of new therapies with improved safety and tolerability compared to pexidartinib, which will significantly impact the treatment landscape in the coming years. This will lead to increased adoption of these new therapies, with patients transitioning from off-label treatments to these newer options during the forecast period (2023‒2034). DelveInsight's latest published market report, titled as Tenosynovial Giant Cell Tumors Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the TGCT country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The TGCT market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Prevalent Cases of TGCT Growth Pattern-specific Prevalent Cases of TGCT Gender-specific Prevalent Cases of Localized TGCT Gender-specific Prevalent Cases of Diffuse TGCT Tumor Location-specific of Localized TGCT Tumor Location-specific Cases of Diffuse TGCT Total Treated Cases of TGCT The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM TGCT market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this TGCT market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the TGCT market. Also, stay abreast of the mitigating factors to improve your market position in the TGCT therapeutic space. Related Reports Tenosynovial Giant Cell Tumors Epidemiology Tenosynovial Giant Cell Tumors Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted TGCT epidemiology in the 7MM, i.e., the United States, EU4 (Germany, Spain, Italy, and France), the United Kingdom, and Japan. Tenosynovial Giant Cell Tumors Pipeline Tenosynovial Giant Cell Tumors Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key TGCT companies, including AmMax Bio, Inc., Deciphera Pharmaceuticals SynOx Therapeutics, Abbisko Therapeutics Co, Ltd., Hutchison Medipharma Limited, Elixiron Immunotherapeutics, among others. Rheumatoid Arthritis Market Rheumatoid Arthritis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key rheumatoid arthritis companies, including Taisho Pharmaceuticals, R-Pharm, GlaxoSmithKline, Aclaris Therapeutics, Pfizer, Abivax, Bristol Myers Squibb, Oscotec, Genosco, Mesoblast, Pfizer Akros Pharma, Japan Tobacco, Abbvie, Horizon Therapeutics, Eli Lilly and Company, Taiho Pharmaceutical, Gilead Sciences, Kiniksa Pharmaceuticals, Istesso, SynAct Pharma, Cyxone, among others. Rheumatoid Arthritis Pipeline Rheumatoid Arthritis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key rheumatoid arthritis companies, including Taisho Pharmaceutical, Zhejiang Hisun Pharmaceutical, SinoMab, Jiangsu Hengrui Medicine, RemeGen, GlaxoSmithKline, Modern Biosciences, Jiangsu Alphamab Biopharmaceuticals, Akros Pharma, Hanlim Pharm, Galapagos NV, Chong Kun Dang Pharmaceutical, Bristol Myers Squibb, AstraZeneca, Aclaris Therapeutics, Abivax, AbbVie, Synact Pharma, Gilead sciences, Mesoblast, Rottapharm Biotech, Eli Lilly and Company, Kiniksa Pharmaceuticals, Abcentra, Cyxone, Yungjin Pharm. Co., Ltd. Taiho Pharmaceutical, Oscotec Inc., Pfizer, Janssen Research & Development, Cipher Pharmaceuticals Inc., KLUS Pharma Inc., Revolo Biotherapeutics, Lynk Pharmaceuticals, Beijing Baylx Biotech, Hope Biosciences, Kangstem Biotech, Celltex Therapeutics, I-Mab Biopharma Co. Ltd., Synermore Biologics Co., Ltd, Oryn Therapeutics, Arthrogen, Bayer, Amgen, Draconis Pharma, Biozeus, Landos Biopharma, Imcyse, Kymera Therapeutics, RedHill Biopharma, 4D Pharma, CV6 Therapeutics, VYNE Therapeutics, MyMD Pharmaceuticals, Brickell Biotech Inc, Worg Pharmaceuticals, Almirall, Standigm, Metrion Biosciences, Jnana Therapeutics, Silo Pharma, SFA Therapeutics, Neutrolis Therapeutics, Ahead Therapeutics, Fresenius Kabi, Kidswell Bio, BioXpress Therapeutics, CinnaGen, Alvotech, Hetero Biopharma, Shanghai Henlius Biotech, Cadila Pharmaceuticals, Innovent Biologics, Torrent Pharmaceuticals, Emcure Pharmaceuticals, mAbxience, Shanghai Junshi Biosciences, Enosi Therapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

▎药明康德内容团队报道 每年的罕见病日，都是我们关注这些“少数群体”的重要时刻。对于罕见病患者来说，新药的研发和获批意味着生命的希望。近几个月，中国国家药监局药品审评中心（CDE）将多款罕见病药物纳入优先审评，涉及适应症涵盖症状性腱鞘巨细胞瘤、重症肌无力、巨细胞动脉炎、特发性肺纤维化等。这些药物有望加速获批，为患者带来新的治疗选择。本文将根据公开资料梳理7款新药的研发进展，期待它们早日上市，点亮罕见病患者的未来。（根据CDE官网，自2024年9月以来，CDE对于纳入优先审评的新药加入“是否为罕见病药物”标签。本文将统计2024.9~2025.2期间，正式被CDE纳入优先审评的罕见病药物） 图片来源：123RF 第一三共（Daiichi Sankyo）：盐酸吡昔替尼胶囊 作用机制：CSF1R小分子抑制剂 适应症：症状性腱鞘巨细胞瘤（TGCT） 吡昔替尼（pexidartinib，PLX3397）是一款CSF1R小分子抑制剂，它同时也会抑制c-kit和FLT3-ITD的活性。该药物于2024年10月被CDE纳入优先审评，适用于治疗伴有重度病变或功能受限且无法通过手术改善的症状性TGCT成年患者。今年1月，其上市申请获得CDE受理。 吡昔替尼此前已经于2019年8月获得美国FDA批准上市，用于治疗症状性TGCT成人患者。支持这项批准的国际多中心3期临床试验结果显示，39%的患者在25周的吡昔替尼治疗后获得缓解，部分缓解率为23%，完全缓解率为15%，而安慰剂组的缓解率为0%。根据FDA彼时的新闻稿介绍，吡昔替尼是其批准的首个治疗TGCT的治疗方法。 强生（Johnson & Johnson）：尼卡利单抗注射液 作用机制：FcRn抗体 适应症：重症肌无力 尼卡利单抗（nipocalimab）是一款靶向新生儿Fc受体（FcRn）的抗体疗法，其已在临床试验中显示可降低免疫球蛋白（IgG）水平，包括致病性IgG，这被认为是自身抗体疾病的根本原因之一。2024年11月，该疗法被CDE纳入优先审评，适用于治疗自身抗体阳性的全身型重症肌无力（gMG）成人患者和青少年患者（大于12 岁）。gMG是一种慢性的罕见自身抗体疾病。 此前，强生公司已先后向FDA和欧洲药品管理局（EMA）提交了尼卡利单抗的上市许可申请（MAA），寻求gMG的适应症批准。该公司在今年1月公布了一项旨在评估尼卡利单抗在广泛的抗体阳性（抗AChR、抗MuSK和抗LRP4）的gMG成年患者中疗效的关键性3期临床试验结果。研究达到主要终点，在24周内，接受尼卡利单抗治疗患者的gMG疾病指标日常生活活动评分（MG-ADL）获得统计学显著和具有临床意义的改善。值得一提的是，这款疗法还于今年年初被行业媒体Evaluate列为今年有望上市的10大潜在重磅疗法之一。  荣昌生物：注射用泰它西普 作用机制：BLyS/APRIL双靶点融合蛋白 适应症：重症肌无力 泰它西普是一款双靶抗体融合蛋白，可同时靶向BLyS和APRIL，直击致病性抗体产生的源头——B细胞及浆细胞，从而减少致病性抗体的产生，发挥治疗作用。泰它西普此前在中国已获批两项适应症，包括系统性红斑狼疮（SLE）和类风湿关节炎。2024年11月，泰它西普针对重症肌无力的新适应症上市申请被CDE纳入优先审评。 近年来，B细胞靶向治疗在重症肌无力中显示出较好的治疗前景，研究发现，相比传统免疫治疗方案，B细胞靶向治疗可显著降低重症肌无力的复发率，且停药率低。泰它西普治疗gMG的中国3期临床研究已经达到主要研究终点。临床研究结果显示其可持续有效改善gMG患者的临床状况，并显示出良好的疗效和安全性。 艾伯维（AbbVie）：乌帕替尼缓释片 作用机制：JAK抑制剂 适应症：成人巨细胞动脉炎（GCA） 乌帕替尼缓释片是一种选择性JAK抑制剂，此前已在中国获批用于治疗多种适应症。前期研究显示，该产品对JAK1显示出的抑制效力大于对JAK2、JAK3和TYK2的抑制效力。2024年12月，乌帕替尼缓释片针对成人GCA适应症的上市申请被CDE纳入优先审评。艾伯维此前新闻稿介绍，乌帕替尼缓释片有可能成为GCA患者的第一个口服治疗药物选择。 GCA是一种自身免疫性疾病，导致颞动脉和其他颅内动脉、主动脉以及其他大中型动脉的炎症。乌帕替尼缓释片此前已在一项针对GCA患者开展的3期研究中取得了积极结果，46%接受乌帕替尼联合26周激素递减方案治疗的患者实现了持续缓解，相比之下，接受安慰剂联合52周激素递减方案治疗的患者中这一数值为29%。 维亚臻生物：普乐司兰钠注射液 作用机制：靶向APOC3靶点的小核酸药物 适应症：家族性乳糜微粒血症综合征（FCS） 普乐司兰钠注射液（VSA001）是一款肝脏靶向的小干扰RNA（siRNA）药物，通过高效且持久地沉默载脂蛋白C-III（APOC3）的mRNA水平，以降低APOC3蛋白的表达，进而通过脂蛋白脂酶（LPL）依赖性和非依赖性双重途径，有效降低血清甘油三酯（TG）和富含TG的脂蛋白（TRL）及其降解残留物水平。根据维亚臻新闻稿介绍，多项临床试验表明该药物具有高效、持久的降TG效果，安全耐受性良好，以及每三个月给药一次的用药便利性。 2024年12月，普乐司兰钠被CDE纳入优先审评，在饮食控制基础上，用于降低家族性乳糜微粒血症综合征（FCS） 成人患者的甘油三酯水平，进而减少急性胰腺炎的发生风险。FCS是一种危及患者生命的罕见病，主要表现为极重度升高的TG水平和餐后重度腹痛及皮肤黄色瘤等，并可由此引起包括急性胰腺炎在内的多种严重并发疾病。常规降脂治疗不能满足该疾病患者的治疗需求，全球范围内尚缺少安全有效的药物治疗选择。2025年1月，普乐司兰钠针对该适应症的上市申请也已经正式获得CDE受理。 勃林格殷格翰（Boehringer Ingelheim）：那米司特片 作用机制：PDE4B抑制剂 适应症：特发性肺纤维化 那米司特片（nerandomilast，BI 1015550）是勃林格殷格翰在研的一款口服PDE4B抑制剂，对PDE4B具有优先的酶促抑制作用。研究表明，该疗法具有抗炎和抗纤维化的双重作用机制，有望治疗肺纤维化和进行性纤维化性间质性肺疾病相关炎症。2022年，FDA曾授予该产品突破性疗法认定，凸显了其在IPF治疗中的潜力。 2025年1月，那米司特片被CDE纳入优先审评，治疗特发性肺纤维化（IPF）。该药的上市申请也于近日（2月25日）获CDE正式受理。IPF是一种严重的致死性肺部疾病，特征性的表现为肺纤维化程度持续增加、肺功能持续下降、呼吸症状加重，严重影响患者生活质量。IPF的自然病程多变且无法预测，预后不佳，患者在确诊后的中位生存期约为3年，比大多数癌症的5年生存率都低。目前，IPF直接的致病途径尚不明确，治疗手段一直十分有限。 翰森制药：伊奈利珠单抗注射液 作用机制：抗CD19单抗 适应症：免疫球蛋白G4相关疾病 伊奈利珠单抗是一款抗CD19单抗，翰森制药此前通过合作获得于中国内地、香港、澳门开发及商业化该产品的独家许可。该产品此前已经在美国、日本、欧洲和中国获批上市，适用于抗水通道蛋白4（AQP4）抗体阳性的视神经脊髓炎谱系障碍（NMOSD）成人患者的治疗。2025年1月，伊奈利珠单抗注射液用于免疫球蛋白G4相关性疾病（IgG4-RD）的新适应症上市申请被CDE纳入优先审评。 IgG4-RD是一种慢性、全身性、免疫介导的纤维炎症性疾病，可影响身体的多个器官。根据翰森制药新闻稿介绍，该次纳入基于其全球关键性3期试验MITIGATE的积极结果，与安慰剂相比，伊奈利珠单抗将患者的复发风险降低87%，57.4%的患者在第52周达到无复发、无需治疗的完全缓解，同时显著减少糖皮质激素使用，证实了其通过CD19+B细胞耗竭机制的安全性和有效性。MITIGATE研究的结果此前已经发表于《新英格兰医学杂志》（NEJM），FDA也已经根据该研究结果授予伊奈利珠单抗用于IgG4相关性疾病的突破性疗法认定，目前相关监管手续正在申报中。 每一款新药的诞生，都是科学与希望的交汇。我们期待这些被纳入优先审评的罕见病药物能够早日获批，为患者带来更多治疗选择，也为他们的生活注入更多光明与可能。愿未来的每一天，罕见病患者都能感受到医学进步的温暖，愿我们共同努力，让“罕见”不再“无助”。 本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权或其他合作需求，请联系wuxi_media@wuxiapptec.com。 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。