Plozasiran

和其他跨国药企一样，赛诺菲也在加快产品组合的重组步伐，这次选择将原研抗癌药泰索帝®的全球业务整体转手。 9 月 30 日，韩国保宁制药董事长兼首席执行官金正均（Jay Jung Gyun Kim）宣布了一项交易信息，表示保宁制药已和赛诺菲签署资产收购协议，收购赛诺菲经典抗肿瘤药物泰索帝（Taxotere，多西他赛注射液）的全球业务权益。 此次交易涵盖该药在全球范围内的市场、销售、分销、注册、生产及商标等相关权利，预计收购总额最高达约 1.75 亿欧元（约 14.6 亿元人民币），其中包括 1400 万欧元的潜在里程碑付款。 根据协议，在获得各国监管批准后，保宁公司将全面接手泰索帝®在包括韩国、德国、西班牙在内的 19 个国家市场，其中也包括中国市场，以及南美和中东部分国家的业务。 完成交易相关程序后，保宁公司计划在韩国礼山园区进行生产，并直接负责该原研药在全球市场的分销与销售。 保宁制药近年来在肿瘤治疗领域持续布局，这也是这家韩国药企第三次收购知名品牌抗肿瘤药物，保宁期待在本次战略收购完成后，公司能够成为一家拥有知名抗肿瘤药物产品组合的跨国药企。 多西他赛被列入世界卫生组织（WHO）基本药物清单，其原研品牌正是赛诺菲的泰索帝®。 该药于 1995 年首次获美国 FDA 批准，是一种具有里程碑意义的细胞毒性化疗药物，广泛应用于乳腺癌、前列腺癌、胃癌及头颈部肿瘤的治疗，是全球抗癌治疗的重要基石，并持续在联合用药等方案中扩展应用。 在 2010 年仿制药进入市场后，泰索帝®销售额迅速跌落，已不再是重磅炸弹级药物，赛诺菲也已将该药视为非核心资产。 不过，据赛诺菲披露，泰索帝®在 2024 年的全球销售额仍高达约 7000 万欧元（约 58 亿元人民币）。 此前，保宁制药已有收购跨国药企原研抗肿瘤药物的经验，2021 年和 2023 年，依次收购了礼来的健择®（Gemzar，吉西他滨）和力比泰®（Alimta，培美曲塞二钠）在韩国的业务，并顺利实现稳定供应和本地生产。 而本次收购泰索帝®，是保宁公司首次跨国药企原研抗癌药全球权益，如前所述，这标志着其战略扩张进入关键阶段，意图通过这项交易，为成长为全球化制药企业奠定基础。 虽然这一品种成长空间已经有限，但保宁公司表示：“尽管肿瘤治疗正向靶向治疗与免疫肿瘤治疗方向发展，但细胞毒性药物仍是抗癌治疗的基础。然而，这类临床必需药物的全球供应频繁短缺与中断已经影响了患者治疗。通过此次收购赛诺菲泰索帝®全球业务，保宁将确保建立该药物稳定的全球供应链，并拓展细胞毒性抗癌药的差异化组合。” 赛诺菲在剥离非核心资产的同时，另一方面，在自家专注的领域也在不断加码，投资布局创新疗法。 比如和中国市场相关的一项交易在今年 8 月 1 日产生，当日，赛诺菲宣布与 Arrowhead 的子公司维亚臻（Visirna Therapeutics）签署资产购买协议。维亚臻目前在大中华区开发和商业化 Arrowhead 的四种在研心血管代谢候选药物。根据协议，赛诺菲将获得在大中华区开发和商业化在研药物普乐司兰钠注射液（Plozasiran）的权利，这是 Arrowhead 同类首创的 RNA 干扰（RNAi） 治疗候选药物，旨在减少载脂蛋白 C-III（APOC3）的产生，在大中华区，它是家族性乳糜微粒血症综合征（FCS）和严重高甘油三酯血症（SHTG）的潜在治疗方案。 可以看到，近年来，跨国药企在产品组合上的不断瘦身，也给一些本土小型药企带来了机会，这些新兴力量得以通过承接经典产品快速切入市场，积累品牌与现金流。 跨国巨头通过腾挪资源，集中火力押注创新疗法与前沿技术，小型本土药企则借势起跑，加速成长——两股力量在全球各区域市场的交汇，也正在共同塑造下一阶段的医药格局。 希望大家都有美好的未来。 相关阅读： 一跨国药企宣布，退出27个市场 22名医药人被罚！全在这家药企… 《国谈药品管理专家共识》征求意见 刚刚，A司按下“升级键”，信息量很大 确认了！BMS出售合资药企，不是退出 超14亿！又一跨国药企出售中国市场业务 知名药企官宣，补税超三千万 告别GSK，齐欣执掌奥利佳 百济神州全国多部门热招中 第十一批国采《采购文件》下载 钟睒睒巨资入局，山西女首富就任总经理 超两百亿！罗氏买买买，员工也转入 重磅合作！医学界×腾讯 今天，多家药企高管大变动！ 刚刚，又一省医保局原副局长被查 9月更新版！10个重点监控药品名单 超500亿！辉瑞强势入局减肥赛道 严查异常处方！国家医保局启动百日行动 官宣了！默克更换全球首席执行官 百时美施贵宝中国为何“换挡”？ 千亿医药巨头，百万年薪副总裁辞职 绿竹生物任命副总经理，薪酬超五百万 套取销售费用，一药企被要求整改 名字撞车啦！一医药外企无辜躺枪… 费森尤斯卡比中国秋季热招中 从医药代表到CEO！GSK换帅 某重点监测清单，50种药品 新政策，利好跨国药企 掌舵十年，吴以芳告别复星医药 加入拜耳正当时！中秋热招 MRCLUB原创作者招募中 数十万精准会员每日阅读，助您快速提升个人品牌，扩大在医药行业及社会化媒体的影响力！（详情请访问网站http://wemr.club） 医药代表 伴您成长！

iStock, Grandbrothers From more than 30 target action dates in the last three months of the year, BioSpace has narrowed the list to six regulatory decisions that could have far-reaching implications for biopharma and patients. As 2025 enters its home stretch, the FDA is also nearing the end of its calendar. But that doesn’t mean that the industry is in for a quiet year-end—quite the opposite, in fact. In the last three months of 2025, the FDA is set to decide on at least 30 regulatory applications, many of which could help shift the treatment landscape for hard-to-treat diseases or push the boundaries for novel modalities. One upcoming verdict, for instance, could mark another industry first for CAR T therapies, while another could mean the comeback of a previously-pulled cancer drug. Here, BioSpace rounds up six of these most highly anticipated decisions in Q4, looking at the data backing their bids and what their approvals could mean for the industry. After Delay, Regeneron Awaits Two Eylea HD Verdicts Regeneron is looking to boost the pro high-dose Eylea with a move into macular edema after retinal vein occlusion (RVO) and a monthly dosing regimen across all its approved indications. The FDA is currently reviewing these applications and is expected to release its verdict in the fourth quarter. No specific decision date has yet been indicated. The regulator had an initial target action date of Aug. 19, but extended the review period due to issues at a third-party manufacturing site, Regeneron announced earlier that month. The production plant in contention was previously owned by the CDMO giant Catalent, which was acquired by Novo Nordisk’s parent company for $16.5 billion in February 2024 . An FDA report made public in August revealed uncontrolled and unaddressed contaminations at this site, including with bacteria and cat hair. Regeneron has nevertheless reassured investors that aside from these third-party problems, the FDA has not flagged any problems with the submission. “Based on our discussions, we believe that there’s nothing significant left to be done,” CEO Leonard Schleifer said during the company’s Q2 earnings call . “We are expecting, once the resolution of the filling issues has occurred, to receive favorable action, we hope, from the FDA.” To support its application for high-dose Eylea, Regeneron filed data from the Phase III QUASAR study, which demonstrated that the high-dose formulation was non-inferior to the currently approved standard-dose version of Eylea when used to treat patients with macular edema after RVO, according to a readout in December 2024. Adverse events were comparable between the two formulations. High-dose Eylea is currently approved for wet age-related macular degeneration, diabetic macular edema and diabetic retinopathy. GSK’s Blenrep Comeback Bid Nears Moment of Truth By Oct. 23, the FDA is expected to release its decision on GSK’s proposal to bring Blenrep back to the market, this time as a second-line treatment for relapsed/refractory multiple myeloma. Blenrep, an antibody-drug conjugate targeting the CD38 protein, was originally approved in August 2020 under the FDA’s accelerated pathway. But soon after failing its confirmatory Phase III study in November 2022, GSK withdrew the product from the market. Then, last year, GSK started setting the stage for Blenrep’s comeback with data from the Phase III DREAMM-7 trial. A readout in February 2024 demonstrated a significant 59% drop in the risk of death or disease progression in patients treated with Blenrep plus bortezomib and dexamethasone, as compared with a similar regimen using Johnson & Johnson’s Darzalex. Then, in June that same year, the pharma reported findings from another late-stage study, DREAMM-8, touting significantly better progression-free survival for Blenrep versus Takeda’s Velcade. GSK is using these two studies to support its regulatory application for Blenrep, but in July this year, the FDA’s internal reviewers flagged “high rates of ocular toxicity and dose modifications” in both trials. The DREAMM studies also had “limited dose exploration” which in turn highlight the unaddressed need for a “careful evaluation” of Blenrep’s risks in its proposed indications, according to the reviewers. An external advisory committee in July agreed with this assessment and voted 7–1 against Blenrep , finding an unfavorable benefit-risk pro the drug in its proposed indication. “The efficacy data were strong but the toxicity data were also very strong,” panelist Neil Vasan said at the time. Shortly after the vote, the FDA extended Blenrep’s review by three months. BMS’ Breyanzi Could Become First CAR T Therapy for Marginal Zone Lymphoma Bristol Myers Squibb is proposing its CAR T therapy Breyanzi for the treatment of marginal zone lymphoma (MZL), potentially blazing the trail for the therapeutic class in this indication. The FDA has a target decision date of Dec. 5. To support its application, BMS filed findings from the Phase II TRANSCEND FL study, a single-arm, open-label trial that enrolled more than 270 patients with relapsed or refractory follicular lymphoma or MZL. Data presented at the 2025 International Conference on Malignant Lymphoma in June demonstrated a 95.5% overall response rate in patients with MZL, including 62.1% who saw a complete response. The safety pro TRANSCEND FL was consistent with what had previously been established for Breyanzi. Rates of cytokine release syndrome and neurologic complications were “low,” according to BMS, with no grade 4 or 5 cases detected. If it wins the FDA’s blessing, Breyanzi would be the first CAR T therapy indicated for MZL, according to an August 2025 news release from the company. It would also mark the second time that Breyanzi notched a regulatory record: In March 2024, it became the first CAR T to win approval for chronic lymphocytic leukemia and small lymphocytic leukemia in relapsed/refractory patients. Breyanzi works by binding to the CD19 protein, in turn triggering the activation and action of CAR-T cells, as well as the release of pro-inflammatory signals to destroy the target tumor. It is also approved for large B-cell lymphoma, follicular lymphoma and mantle cell lymphoma. The drug’s label carries a boxed warning for cytokine release syndrome, neurological toxicities and secondary hematological malignancies. Arrowhead Makes Case for RNAi Therapy in Rare Cholesterol Disease After delivering four firsts for the rare disease space in August, the FDA by Nov. 18 could grant another—this one for Arrowhead Pharmaceuticals’ plozasiran , an RNA interference therapeutic for familial chylomicronemia syndrome (FCS). Afflicting 1 to 2 people per million , FCS is an ultra-rare, heritable disease that manifests as excessively high levels of triglyceride levels, typically reaching over 880 mg/dL, according to Arrowhead . In turn, patients with FCS suffer from cardiometabolic and other complications such as diabetes, hepatic steatosis, abdominal pain and even cognitive problems. There are currently no treatments that can adequately control FCS in the U.S., according to Arrowhead. Arrowhead’s answer to FCS is plozasiran , an investigational therapy that reduces the production of the ApoC3 protein, which is naturally produced in liver cells and serves as a component of triglyceride-rich lipoproteins. ApoC3 prevents the breakdown and clearance of these fatty molecules. By reducing ApoC3 production, plozasiran helps normalize lipid levels in FCS patients. Arrowhead is supporting its application with data from the Phase III PALISADE trial. A readout in November 2024 demonstrated that 25 mg of plozasiran—the proposed commercial dose—led to a roughly 80% reduction in triglyceride levels. In at least half of the treated patients, triglyceride levels stayed below 500 mg/dl, a threshold linked with worse acute pancreatitis risk. Plozasiran treatment also resulted in lower cholesterol levels. Arrowhead is also developing plozasiran for severe hypertriglyceridemia and dyslipidemia. Ascendis Eyes Approval in Rare Bone Growth Disorder Also awaiting a rare disease decision is Ascendis Pharma, which is advancing TransCon CNP, also known as navepegritide, for the treatment of children with achondroplasia. The FDA is set to release its verdict by Nov. 30. Achondroplasia is a rare, genetic disease characterized by dwarfism due to faulty bone development. Ascendis is targeting a specific disease type caused by mutations in the FGFR3 gene, resulting in manifestations in different tissues across the body. Aside from bone problems, patients with this specific type of achondroplasia also suffer from muscular, neurological and cardiorespiratory problems. Around 250,000 people worldwide are affected by this disease, according to Ascendis. TransCon CNP works by providing sustained exposure to the C-type natriuretic peptide, a molecule that helps stimulate bone growth and counter the inhibiting effects of FGFR3 mutations. Through this mechanism, Ascendis contends that its drug candidate could help ease the burden of symptoms and complications in achondroplasia. Ascendis tested the TransCon CNP in ACcomplisH, a randomized, double-blinded and placebo-controlled Phase II study that enrolled 42 patients who received 6 μg, 20 μg or 50 μg of the drug. Results, published in The Lancet in November 2023, showed that patients on TransCon CNP saw significant improvements in annualized growth velocity, alongside other key benefits, such as a stabilized quality of life and better physical functioning. In a June 2025 statement , Ascendis Chief Medical Officer Aimee Shu said the biotech’s clinical development program for TransCon CNP is the “first ever to demonstrate improvements beyond linear growth at 52 weeks compared to placebo.” Cytokinetics Could Challenge BMS in Cardiomyopathy Market Closing out this list is Cytokinetics and its cardio candidate aficamten, which is currently under FDA review for obstructive hypertrophic cardiomyopathy (oHCM). The FDA’s target decision date is Dec. 26. Aficamten blocks the cardiac myosin protein, which plays a role in heart contraction, allowing the oral drug candidate to address a key symptom of oHCM. Data from the Phase III MAPLE-HCM study, presented in August at the 2025 European Society of Cardiology Congress, demonstrated a significant improvement in exercise capacity over metoprolol, a beta-blocker used as standard-of-care in the disease. At the time, analysts at Truist Securities said aficamten’s results position it to be “potentially best-in-class” in oHCM. They called the MAPLE-HCM data “robust,” noting that outperforming metoprolol could help aficamten distinguish itself in the oHCM market. Cytokinetics filed for aficamten’s approval in late 2024, at which time the FDA provided an initial target action date of Sept. 26, 2025 . In May, however, the regulator extended its review by three months to review an additional risk mitigation strategy submission. If approved, aficamten will compete with BMS’s Camzyos, also a cardiac myosin inhibitor that won approval for oHCM in 2022 . The pharma is testing the drug in a non-obstructive form of the heart disease, but a readout in April this year was disappointing : Camzyos was unable to significantly outperform placebo in terms of exercise capacity outcomes, nor did it lead to better physical function.