Alirocumab

前 言2025年6月，《中国心血管健康与疾病报告2024概要》发布，报告显示，心血管病目前仍位居中国城乡居民的首位死因，而血脂异常正是背后“沉默的杀手”之一。作为一种可以大幅降低低密度脂蛋白胆固醇（LDL-C）的新型降脂药物，PCSK9抑制剂市场近年来发展迅猛。一方面，两款进口药物2021年纳入医保后加速放量；另一方面，2023年信达生物首款国产PCSK9抑制剂的获批打破了进口垄断的局面。根据法伯全渠道数据显示，2023年我国总体医疗机构PCSK9抑制剂药物市场规模超13亿元，同比大涨158.3%。补充说明*本文所述PCSK9抑制剂药物市场范围（按照药物解剖学、治疗学及化学分类法ATC编码）：C10A4。*中国医疗机构：包括两个医院渠道（城市医院、县域医院）和两个基层医疗机构渠道（社区卫生中心、乡镇卫生院）。国内累计7款PCSK9抑制剂药物获批在种类丰富的降脂药市场中，除了常年份额领先的“主角”他汀类药物，新型降脂药物PCSK9抑制剂无疑是值得关注的“黑马”。根据法伯全渠道数据显示，2023年我国总体医疗机构PCSK9抑制剂市场规模达13.2亿元，占整体降脂药市场的7.8%，排名仅次于其他调节胆固醇和甘油三酯类药物，且尤其在城市医院的销售贡献更大（图1）。图1-降脂药物分类及2023年分渠道销售额占比数据来源：法伯全渠道数据公开信息显示，目前国内共有7款靶向PCSK9产品获批上市，3款来自外资企业，4款来自内资。其中，安进的依洛尤单抗注射液（商品名：瑞百安）、赛诺菲的阿利西尤单抗注射液（商品名：波立达）、以及信达生物的托莱西单抗注射液（商品名：信必乐）已经纳入国家医保目录（表1）。表1-国内批准上市的PCSK9抑制剂数据来源：法伯整理销售额渠道分布方面，作为降脂领域的创新药物，PCSK9抑制剂主要在城市医院销售。法伯数据显示，2021-2023年，城市医院渠道销售额的贡献比例持续在90%上下浮动，到2023年达92.1%。从2023年同比增长来看，四个销售渠道均显著增长，其中乡镇卫生院渠道涨幅最高（图2）。图2-2021-2023年PCSK9抑制剂销售额渠道分布数据来源：法伯全渠道数据进口药品双雄争锋，纳入医保撬动市场增长2018年7月，美国安进公司的依洛尤单抗注射液（商品名：瑞百安）作为首款进口PCSK9抑制剂在国内获批上市，用于成人或12岁以上青少年的纯合子型家族性高胆固醇血症，是安进将创新药物引入中国市场的重要里程碑。此后的2019年末，赛诺菲的阿利西尤单抗注射液（商品名：波立达）获国家药监局批准上市，成为国内第二款获批上市的进口PCSK9抑制剂。然而，受限于高昂的价格和临床应用可及性等因素，PCSK9抑制剂在国内市场未实现爆发式增长，直到2021年医保谈判成为转折点，安进的瑞百安和赛诺菲的波立达均大幅降价进入医保。以瑞百安为例，公开信息显示，在纳入医保之前其每针的价格在1300元左右，进入医保之后降价至280元左右，降幅超过70%。按照每月注射两次计算，其年治疗费用从逾3万元降至近7000元，大大减轻了相关患者的用药负担。政策红利立竿见影，随着2022年1月1日《国家医保药品目录（2021年）》的落地实施，上述两个PCSK9抑制剂产品加速放量。根据法伯全渠道数据显示，2022年上述两款药物在我国总体医疗机构的销售额强劲增长，2021-2023年复合增长率分别搞到223.1%和699.5%（图3）。图3-2021-2023年总体医疗机构依洛尤单抗和阿利西尤单抗市场规模数据来源：法伯全渠道数据从不同销售渠道的产品竞争格局来看，法伯数据显示，2023年依洛尤单抗在城市医院、社区卫生中心和乡镇卫生院渠道PCSK9抑制剂市场均占据过半份额，而阿利西尤单抗则是在县域医院份额占优（图4）。图4-2023年各销售渠道PCSK9抑制剂市场产品格局数据来源：法伯全渠道数据信达打破进口垄断，国产梯队迎来收获期直到2023年8月，信达生物自主研发的首个国产PCSK9抑制剂托莱西单抗注射液（商品名：信必乐）获批上市，宣告了国内PCSK9抑制剂市场进口药物垄断的终结，目前该药物已于2024年纳入医保目录。也是8月，诺华中国宣布其创新性降脂药英克司兰钠注射液（Inclisiran）获批上市（商品名：乐可为）。相较于依洛尤单抗和阿利西尤单抗，后获批的托莱西单抗和英克司兰钠更具长效性，其中前者提供2周、4周、6周三种治疗方案，后者每年仅需注射两次，更加灵活和长效的治疗周期有利于提升患者的依从性。值得关注的是，截至2023年末获批的四款PCSK9抑制剂药物，均是重点在城市医院渠道布局，根据法伯全渠道数据显示，2023年瑞百安、波立达和信必乐城市医院销售额贡献的比例均在90%左右，乐可为则基本仅在城市医院销售（图5）。图5-2023年各PCSK9抑制剂药物渠道销售分布数据来源：法伯全渠道数据在信达托莱西单抗打响国产PCSK9抑制剂第一枪之后，其他国产自研PCSK9抑制剂药物也陆续迎来收获期：2024年9月30康方生物宣布其自主研发的1类新药伊努西单抗（商品名：伊喜宁）获国家药监局批准上市，用于治疗原发性高胆固醇血症和混合型高脂血症，以及杂合子型家族性高胆固醇血症。2024年10月11日君实生物宣布由公司自主研发的昂戈瑞西单抗注射液（商品名：君适达）获批上市，用于治疗原发性高胆固醇血症（非家族性）和混合型血脂异常的成人患者，成为君实生物第5款商业化产品。2025年1月10日恒瑞医药自主研发的1类创新药瑞卡西单抗（商品名：艾心安）获批上市。可以预见的是，随着更多药物获批并纳入医保，后续PCSK9抑制剂市场的或将掀起更大的波澜。在愈加激烈的竞争环境中，只有疗效更优、安全性更高、依从性更好、价格更实惠的具有差异性优势的产品，才有望脱颖而出，并持续在市场中站稳脚跟。往期精选*点击图片阅读详情*点击图片阅读详情*点击图片阅读详情关于法伯科技Pharbers法伯科技秉持客户第一的理念，近十年专注于为我国医药行业和客户提供数据及数据价值服务，努力提供更可靠、更便捷、更能够支持客户业务发展的医药数据。法伯科技能够有效地解决数据短缺、数据孤岛、数据指标口径不统一等行业痛点，通过提供多元数据治理、数据分析及洞察、数据决策等数据价值的全链条服务，帮助客户进行数据资产管理，最大化数据资产价值。目前法伯科技已经为国内外近百家领先的医药客户提供了数据价值服务，法伯科技的医药市场多渠道数据覆盖核心医院市场、广阔市场以及DTP、电商等新兴零售市场，能够帮助客户监测多渠道市场竞争动态、及时发现和应对市场变化；法伯科技同时提供真实世界数据研究及服务，帮助客户从更加真实、具体和精细的角度去发现业务发展的机会；此外，法伯科技还为客户提供更多基于数据的定制化解决方案，如终端潜力估算、市场销售策略等。联系我们产品/业务咨询请联系：contact@pharbers.com或致电：010-84244216公司地址北京市朝阳区东直门外斜街小关56号四层上海市虹口区星荟中心1座603单元

Lynozyfic is a bispecific antibody directing T cells to kill multiple myeloma cancer cells; multiple myeloma is the second most common blood cancer Approval based on pivotal Phase 1/2 LINKER-MM1 trial; results reported were among the highest overall (70%) and complete response rates (45%) for bispecific antibodies in heavily pre-treated multiple myeloma patients Lynozyfic provides a patient-centric response-adapted dosing regimen TARRYTOWN, NY, USA I July 02, 2025 I Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Lynozyfic™ (linvoseltamab-gcpt) to treat adult patients with relapsed or refractory (R/R) multiple myeloma (MM) who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti‑CD38 monoclonal antibody. Lynozyfic was granted accelerated approval based on response rate and durability of response in the LINKER-MM1 trial. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Lynozyfic is the first FDA-approved BCMAxCD3 bispecific antibody that can be dosed every two weeks starting at week 14, and every four weeks if a very good partial response (VGPR) or better is achieved following completion of at least 24 weeks of therapy. The regimen includes hospitalization for safety during the step-up dosing period (one 24-hour period after the first step-up dose, and another 24-hour period after the second step-up dose). “The FDA approval of Lynozyfic represents meaningful progress for the multiple myeloma community. Lynozyfic demonstrated early, deep and durable responses in heavily pre-treated patients, which I saw firsthand in clinical trials,” said Sundar Jagannath, M.D., Network Director of the Center of Excellence for Multiple Myeloma at Mount Sinai in New York City and a trial investigator. “Lynozyfic has a convenient response-adapted dosing regimen, which provides the potential to extend time between doses. This is a significant patient-centric advancement that could help reduce treatment burden.” The FDA approval is based on results from the pivotal Phase 1/2 LINKER-MM1 trial investigating linvoseltamab in R/R MM in which patients (n=80) experienced a: The prescribing information for Lynozyfic has a Boxed Warning for cytokine release syndrome (CRS) and neurologic toxicity – including immune effector cell-associated neurotoxicity syndrome – in addition to warnings and precautions for infections, neutropenia, hepatotoxicity and embryo-fetal toxicity. The most common adverse reactions (≥20%) in the safety population of LINKER-MM1 (n=117) were musculoskeletal pain, CRS, cough, upper respiratory tract infection, diarrhea, fatigue, pneumonia, nausea, headache and dyspnea. The most common Grade 3 or 4 laboratory abnormalities (≥30%) were decreased lymphocyte count, decreased neutrophil count, decreased hemoglobin and decreased white blood cell count. Lynozyfic is available only through a restricted program called the Lynozyfic Risk Evaluation and Mitigation Strategy (REMS). Details of the Important Safety Information are included below. “The FDA approval of Lynozyfic reinforces the strength of our bispecific antibody program as well as our commitment to delivering critical medicines to the cancer community,” said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer of Regeneron. “With a 70% overall response rate in heavily pre-treated patients, we believe Lynozyfic is poised to potentially become a new standard of care for multiple myeloma. Furthermore, given the strength of the data, we are rapidly advancing our broad clinical development program for Lynozyfic – exploring its use in earlier lines of therapy as monotherapy and in novel combinations – as we aim to meaningfully advance care for patients.” Regeneron is committed to helping patients who have been prescribed Lynozyfic access their medication. The company has launched Lynozyfic Surround™, which offers financial and educational resources to help support patients throughout their treatment journey. For more information, patients can call 1-844-RGN-HEME (1-844-746-4363). “Even though the number of treatment options for multiple myeloma has expanded in recent years, it remains an incurable disease with considerable unmet need, especially among patients who have undergone multiple lines of treatment,” said Diane Moran, R.N., M.A., Ed.M., Chief Executive Officer (Interim) and Senior Vice President of Strategic Planning at the International Myeloma Foundation. “The FDA approval of Lynozyfic is a welcome milestone. It provides appropriate multiple myeloma patients and their care teams with a novel patient-centric treatment option that includes a dosing schedule that can be adapted based on patient response. We appreciate Regeneron’s continued research to further advance treatment for this community.” About Multiple Myeloma As the second most common blood cancer, there are over 187,000 new cases of MM diagnosed globally every year, with more than 36,000 diagnosed and 12,000 deaths anticipated in the U.S. in 2025. In the U.S., there are approximately 8,000 people who have MM that has progressed after three lines of therapy, and 4,000 whose disease has progressed after four or more therapies. The disease is characterized by the proliferation of cancerous plasma cells (MM cells) that crowd out healthy blood cells in the bone marrow, infiltrate other tissues and cause potentially life-threatening organ injury. Despite treatment advances, MM is not curable and while current treatments are able to slow progression of the cancer, most patients will ultimately experience cancer progression and require additional therapies. About Lynozyfic™ (linvoseltamab-gcpt) Lynozyfic was invented using Regeneron’s VelocImmune ® technology and is a fully human BCMAxCD3 bispecific antibody designed to bridge B-cell maturation antigen (BCMA) on MM cells with CD3-expressing T cells to facilitate T-cell activation and cancer-cell killing. Linvoseltamab is administered with an initial step-up dosing regimen followed by the full 200 mg dose administered weekly. At week 14, patients transition to every two-week dosing. A response-adapted regimen further enables patients to shift to every four-week dosing if they achieve and maintain a VGPR or better after having completed at least 24 weeks of therapy. Patients should be hospitalized for 24 hours after administration of the first step-up dose and for 24 hours after administration of the second step-up dose, with the potential for additional hospitalization if patients experience certain adverse events. The generic name for Lynozyfic in its approved U.S. indication is linvoseltamab-gcpt with gcpt as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the FDA. Outside of the U.S., the generic name of Lynozyfic in its approved indications is linvoseltamab. Lynozyfic is also approved in the European Union to treat adults with R/R MM after at least three prior therapies, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody, and have demonstrated disease progression on the last therapy. For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu in due course. About the Linvoseltamab Clinical Development Program The ongoing, open-label, multicenter Phase 1/2 dose-escalation and dose-expansion LINKER-MM1 trial is investigating linvoseltamab in more than 300 enrolled patients with R/R MM. The Phase 1 intravenous dose-escalation portion of the trial – which is now complete – primarily assessed safety, tolerability and dose-limiting toxicities across nine dose levels of linvoseltamab and explored different administration regimens. A subcutaneous Phase 1 portion is ongoing. The Phase 2 intravenous dose expansion portion is ongoing and assessing the safety and anti-tumor activity of linvoseltamab, with the primary endpoint of ORR. Key secondary endpoints include DoR, progression-free survival, rate of minimum residual disease negative status and overall survival. Linvoseltamab is being investigated in a broad clinical development program exploring its use as a monotherapy as well as in combination regimens across different lines of therapy in MM, including earlier lines of treatment, as well as MM precursor and other plasma cell disorders. This includes evaluating linvoseltamab in a Phase 1b trial ( LINKER-MM2 ) in combination with other cancer treatments in R/R MM as well as a Phase 3 confirmatory trial ( LINKER-MM3 ) as a monotherapy in R/R MM. For more information on Regeneron’s clinical trials in blood cancer, visit the clinical trials website , or contact via clinicaltrials@regeneron.com or 1-844-734-6643. About Regeneron in Hematology At Regeneron, we’re applying more than three decades of biology expertise with our proprietary VelociSuite ® technologies to develop medicines for patients with diverse blood cancers and rare blood disorders. Our blood cancer research is focused on bispecific antibodies that are being investigated both as monotherapies and in various combinations and emerging therapeutic modalities. Together, they provide us with unique combinatorial flexibility to develop customized and potentially synergistic cancer treatments. Our research and collaborations to develop potential treatments for rare blood disorders include explorations in antibody medicine, gene editing and gene-knockout technologies, and investigational RNA-approaches focused on depleting abnormal proteins or blocking disease-causing cellular signaling. About Regeneron’s VelocImmune ® Technology Regeneron’s VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron’s co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create a substantial proportion of all original, FDA-approved fully human monoclonal antibodies. This includes Dupixent ® (dupilumab), Libtayo ® (cemiplimab-rwlc), Praluent ® (alirocumab), Kevzara ® (sarilumab), Evkeeza ® (evinacumab-dgnb), Inmazeb ® (atoltivimab, maftivimab and odesivimab-ebgn) and Veopoz ® (pozelimab-bbfg). In addition, REGEN-COV ® (casirivimab and imdevimab) had been authorized by the FDA during the COVID-19 pandemic until 2024. Please see full Prescribing Information , including Boxed WARNING, and Medication Guide for LYNOZYFIC. What is LYNOZYFIC? LYNOZYFIC is a prescription medicine used to treat adults with multiple myeloma who: It is not known if LYNOZYFIC is safe and effective in children. About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases. Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as VelociSuite ® , which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center ® and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases. For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn , Instagram , Facebook or X . SOURCE: Regeneron Pharmaceuticals