Nilotinib Hydrochloride

2014年，罗氏以83亿美元天价收购InterMune，一举拿下特发性肺纤维化（IPF）药物Esbriet®。彼时，这款药物被寄予厚望，与勃林格殷格翰的Ofev®共同开创IPF治疗新时代。然而十年后，Esbriet®年销售额断崖式跌至1亿美元（2024年数据），仿制药的“价格利刃”彻底刺破其市场护城河。这一案例，恰是跨国药企在专利悬崖下生死博弈的缩影。 Esbriet®的沉浮：从明星药到市场夹缝 2014年8月，罗氏斥资83亿美元收购InterMune，抢占IPF市场先机。同年10月，Esbriet®与勃林格殷格翰的Ofev®一同获FDA批准，成为美国首批用于治疗IPF的药物。 罗氏采取精准定价策略，美国市场年费用高达9.4万美元，欧洲则因医保体系差异采取渐进式定价。得益于这一布局，Esbriet®在2018-2022年间年均销售额突破10亿美元。 然而，2022年核心专利到期，仿制药迅速涌入， 2023年Esbriet®销售额大幅下降至2.29亿美元，2024年更是跌至1亿美元，市场份额急剧缩水。面对仿制药冲击，罗氏并未硬碰硬，而是精准拆分市场，出售Esbriet®美国市场权益，保留利润率更高的非美市场。 与此同时，罗氏果断调整研发战略，自2023年第二季度起，罗氏新增了24项资产，并淘汰了34个项目，将资源集中于那些成功率更高或市场潜力更大的项目，以提升研发效率和投资回报。 面对市场剧变，罗氏的策略并非简单撤退，而是精准切割与价值重构，以最大化保留竞争优势。 仿制药来袭，跨国药企如何破局？ 如果说专利是创新药企的护城河，那么专利到期就是城门洞开的时刻——仿制药厂商蜂拥而至，以低至原研药20%的价格席卷市场。对于跨国药企而言，如何在“专利悬崖”前后保持竞争力，已成为生死攸关的命题。 Fierce Pharma数据显示，2025-2030年全球将有超过2300亿美元的药品步入专利到期周期，而跨国药企应对方式各异：有人“法律硬抗”，有人“科技突围”，也有人“战略撤退”。罗氏（Roche）对特发性肺纤维化（IPF）药物Esbriet®的调整，正是这一生存博弈的鲜活案例。 从83亿美元的豪掷收购到区域性剥离，罗氏在Esbriet®的取舍间展现了极高的战略弹性。这不仅关乎一款药物的兴衰，更映射出跨国药企在专利悬崖下的进化路径。 专利悬崖下的五大攻防战：跨国药企各显神通 Esbriet®的案例只是一个缩影，全球MNC们在面对专利悬崖时，各自找到了不同的突围方式。 辉瑞（Pfizer）——立普妥（Lipitor） 辉瑞在专利到期前五年启动“Lipitor 2.0”计划，通过支付保险公司回扣、向患者提供折扣卡等策略延缓仿制药替代。并剥离非核心业务成立Viatris。 诺华（Novartis）——格列卫（Gleevec） 诺华采取"软着陆"策略，推出二代（Tasigna）和三代（Iclusig）药物延续产品线，并聚焦高壁垒领域。 默沙东（MSD）——顺尔宁（Singulair） 顺尔宁专利到期后，默沙东大幅调整战略，将资源倾斜至PD-1抑制剂Keytruda，至2023年销售额达250亿美元，同时大力发展HPV疫苗Gardasil 9。 赛诺菲（Sanofi）——来得时（Lantus） 面对生物类似药冲击，赛诺菲推出Toujeo（超长效胰岛素）稳住市场，并大举进军罕见病。同时，退出中国胰岛素集采，专注高端市场。 艾伯维（AbbVie）——修美乐（Humira） 艾伯维先以专利战延缓仿制药上市，再通过价格捆绑绑定JAK抑制剂Rinvoq，最后推动Skyrizi（IL-23）+Rinvoq组合替代修美乐。 这些策略各有利弊，但本质上都围绕“如何在专利到期前后最大化利润”展开。 专利悬崖下的多维应对：药企的灵活策略 面对专利到期的挑战，药企如何应对已成为一项重要的战略考量。在这场博弈中，一些常见的策略浮现出来，但并没有一成不变的“唯一选择”。 一种思路是通过产品的升级与生命周期管理，探索新一代产品或剂型的可能性，以延续市场影响力；另外，市场策略调整，如降价、折扣或与保险公司谈判，也可能帮助药企在竞争中争取一段缓冲期。另一方面，法律战和专利壁垒的建立，不失为争取更多市场时间的一条路径。此外，也有药企考虑通过业务剥离，集中资源向高利润领域进军，或者通过管线升级与业务转型，寻求新的增长动力。 这些策略并非孤立，而是可以相辅相成，帮助企业在专利悬崖下找到持续发展的空间。关键在于药企如何根据自身情况，灵活运用各种策略，应对市场的不确定性。 结语：创新药时代，没有“永恒的护城河” Esbriet®从83亿收购到1亿收入的抛物线，揭示了一个残酷现实：在仿制药的围剿下，没有药物能永居神坛。但罗氏的“切割-聚焦-重生”策略，亦证明危机中的药企仍可重构竞争力——甩掉包袱、押注未来、拥抱技术革命，或将成为穿越专利悬崖的三把钥匙。 未来，唯有将“应变力”刻入基因的企业，方能在生物医药的生死竞速中，将每一次悬崖危机，转化为涅槃重生的跳板。 免责声明： 文章内容仅供参考，不构成投资建议。投资者据此操作，风险自担, 关于对文中陈述、观点判断保持中立，不对所包含内容的准确性、可靠性或完整性提供任何明示或暗示的保证。请读者仅作参考，并请自行承担全部责任。本公众号发布的各类文章重在分享，如有侵权请联系我们，我们将会删除。 联系我们 ABC 2025 展位火热预定中！ 扫码立即咨询 电话：13816031174 （同微信） 赞助形式包括但不仅限于演讲席位、会场展位、会刊彩页、晚宴赞助、会议用品宣传等。 点击此处“阅读全文”咨询更多精彩！

In the MOTION Phase 3 study, ROMVIMZA met primary endpoint of improved objective response rate (ORR) compared to placebo and all key secondary endpoints with statistically significant and clinically meaningful improvements in quality-of-life measures, and demonstrated well-tolerated safety profile OSAKA, Japan & WALTHAM, MA, USA I February 14, 2025 I Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President: Toichi Takino; “Ono”) announced that the U.S. Food and Drug Administration (FDA) has approved ROMVIMZA™ (vimseltinib), a kinase inhibitor, for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity. The FDA previously granted Fast Track designation and Priority Review for ROMVIMZA, which was developed by Deciphera Pharmaceuticals, Inc. (“Deciphera”), a wholly owned subsidiary of Ono. “The approval of ROMVIMZA provides a new, much-needed, well-tolerated, and effective treatment option for people suffering from TGCT,” said Hans Gelderblom, M.D., Ph.D., Chair of the Department of Medical Oncology at Leiden University Medical Center. “TGCT adversely affects the lives of patients, causing significant pain, limited mobility, and stiffness. The MOTION Phase 3 study demonstrated ROMVIMZA’s ability to shrink tumors along with being the first well-tolerated agent to demonstrate significant improvement in a number of other important quality-of-life measures without any observed liver injury as seen with other approved TGCT treatment. ROMVIMZA is a differentiated treatment that has the potential to address the significant unmet needs of the TGCT community.” “The FDA approval of ROMVIMZA for TGCT is a crucial advancement for the TGCT community and we believe ROMVIMZA has the potential to become the new standard of care for people with TGCT for which surgical resection will potentially cause worsening functional limitation or severe morbidity. This is also an important milestone for our organization, as it is the second approved therapy discovered using Deciphera’s proprietary switch-control kinase inhibitor platform,” said Ryota Udagawa, President and Chief Executive Officer of Deciphera Pharmaceuticals. “I’d like to extend my gratitude to the patients, families, caregivers, and healthcare providers who contributed to the success in ROMVIMZA’s clinical studies. Their commitment, along with the dedication of the Deciphera and Ono teams, enabled us to advance this impactful new treatment, which we look forward to delivering to patients.” TGCT is a rare, non-malignant tumor that forms within or near joints. TGCT arises from the dysregulation of the CSF1 gene, resulting in an overproduction of CSF1. If left untreated or if the tumor repeatedly recurs, it can lead to damage and degeneration in the affected joint and surrounding tissues, potentially causing significant disability. The FDA approval was based on the efficacy and safety results from the pivotal Phase 3 MOTION study of ROMVIMZA in patients with TGCT not amenable to surgery with no prior anti-CSF1/CSF1R therapy (prior therapy with imatinib or nilotinib allowed), compared to placebo, as well as the Phase 1/2 study of ROMVIMZA. In MOTION, ROMVIMZA demonstrated a statistically significant and clinically meaningful ORR at Week 25 in the intent-to-treat (ITT) population, as assessed by blinded independent radiologic review (IRR) per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1), versus placebo (40% in ROMVIMZA arm vs 0% in placebo arm, p <0.0001). The primary endpoint was supported by statistically significant and clinically meaningful improvements in active range of motion, patient-reported physical functioning, and patient-reported pain observed in the vimseltinib arm compared to the placebo arm at week 25. The safety profile of ROMVIMZA is manageable and consistent with results previously disclosed in the Phase 1/2 clinical trial. Deciphera Pharmaceuticals plans to make ROMVIMZA commercially available in the U.S. next week. Learn more at www.ROMVIMZA.com . In July of 2024, the Company announced the marketing authorization application (MAA) for ROMVIMZA for the treatment of patients with TGCT was accepted and is under review by the European Medicines Agency (EMA). Deciphera is committed to supporting TGCT patients and providers in navigating coverage and access to ROMVIMZA. As part of that commitment, Deciphera AccessPoint TM , a patient support program, is available to provide comprehensive access and financial assistance programs for eligible patients. For more information, visit DecipheraAccessPoint.com or call 1-833-4DACCES (1-833-432-2237), Monday-Friday, 8:00 AM to 8:00 PM Eastern Time (ET). About MOTION Study The MOTION study is a two-part, randomized, double-blind, placebo-controlled Phase 3 clinical study to assess the efficacy and safety of vimseltinib in patients with TGCT not amenable to surgery with no prior anti-CSF1/CSF1R therapy (prior therapy with imatinib or nilotinib allowed). The primary endpoint of the study is an objective response rate (ORR) at Week 25 in the intent-to-treat (ITT) population, as assessed by blinded independent radiologic review (IRR) per using Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1), versus placebo. The secondary endpoints include ORR per tumor volume score (TVS), active range of motion (ROM), physical function, stiffness, quality of life, and pain, all assessed at Week 25. This study consists of two Parts. In Part 1, patients were randomized to receive either vimseltinib or placebo for 24 weeks. In Part 2, patients randomized to placebo in Part 1 have the option to receive vimseltinib, and all patients receive vimseltinib for a long-term period in an open-label setting. ROMVIMZA (vimseltinib) capsules INDICATIONS AND USAGE ROMVIMZA is indicated for treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity. IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS Hepatotoxicity Embryo-Fetal Toxicity: Allergic Reactions to FD&C Yellow No.5 (Tartrazine) and No. 6 (Sunset Yellow FCF): Increased Creatinine without Affecting Renal Function: Adverse Reactions: The most common (≥20%) adverse reactions, including laboratory abnormalities that occurred in patients receiving ROMVIMZA were increased AST, periorbital edema, fatigue, rash, increased cholesterol, peripheral edema, face edema, decreased neutrophils, decreased leukocytes, pruritus, and increased ALT. Drug Interactions: Lactation: Advise females not to breastfeed during treatment with ROMVIMZA. Please see the accompanying full Prescribing Information . About Tenosynovial Giant Cell Tumor (TGCT) TGCT is a rare disease caused by a translocation in colony-stimulating factor 1 (CSF1) gene resulting in overexpression of CSF1 and recruitment of colony-stimulating factor 1 receptor (CSF1R)-positive inflammatory cells into the lesion. TGCT is a rare, non-malignant tumor that develops inside or near joints. TGCT is caused by dysregulation of the CSF1 gene leading to overproduction of CSF1. TGCT is also known as giant cell tumor of the tendon sheath (GCT-TS) or pigmented villonodular synovitis (PVNS), a diffuse-type of TGCT. Although non-malignant, these tumors can grow and cause damage to surrounding tissues and structures inducing pain, swelling, and limitation of movement of the joint. Surgery is the main treatment option; however, these tumors tend to recur, particularly in diffuse-type TGCT. If untreated or if the tumor continually recurs, damage and degeneration may occur in the affected joint and surrounding tissues, which may cause significant disability. For a subset of patients, surgical resection will potentially cause worsening functional limitation or severe morbidity, systemic treatment options are limited and a new therapeutic option for TGCT is needed. About Deciphera Pharmaceuticals Inc. (As of June 11, 2024, Deciphera became a member of Ono Pharmaceutical Co., Ltd.) Deciphera is a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer. We are leveraging our proprietary switch-control kinase inhibitor platform and deep expertise in kinase biology to develop a broad portfolio of innovative medicines. In addition to advancing multiple product candidates from our platform in clinical studies, QINLOCK ® (ripretinib) is Deciphera’s switch-control inhibitor approved in many countries including the European Union and the United States for the treatment of fourth-line gastrointestinal stromal tumor (GIST). ROMVIMZA™ (vimseltinib) is a kinase inhibitor approved in the United States for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity. For more information, visit www.deciphera.com and follow us on LinkedIn and Twitter (@Deciphera). SOURCE: Ono Pharmaceutical