Remestemcel-L-rknd

9月14日，央视新闻频道报道了一则干细胞领域的重磅新闻——国内首位接受艾米迈托赛注射液治疗的患者病情好转，已经获准出院。 据央视新闻频道报道，该患者为一名青年女性，在北京大学人民医院接受造血干细胞移植后出现了重度肠道急性移植物抗宿主病，经多项治疗均无明显获益，一度生命垂危。今年6月，医生为其开出了艾米迈托赛注射液首张处方。经过治疗，该患者病情好转，已经获准出院，后续只需定期服药和复查即可。 艾米迈托赛注射液是一款间充质干细胞药物，由铂生生物自主研发。今年年初，艾米迈托赛注射液获国家药监局附条件批准上市，用于治疗14岁以上消化道受累为主的激素治疗失败的急性移植物抗宿主病（aGVHD）。 这是国内首款获批的干细胞药物，也是目前唯一的一款。 和传统新特药在线下铺设经销网点不同，铂生生物选择和医药电商头部平台阿里健康达成战略合作，由后者全权负责艾米迈托赛在国内的商业化落地。 短短3个月，这一战略决策已经初见成效——在阿里健康的全流程助力下，艾米迈托赛注射液的药品预约服务已实现全国范围的广泛覆盖，包括北京、上海、山东、江苏、河南、山西、陕西、湖北、广东等20个省份，惠及上百位患者。 真实世界一小步 随着医学技术的不断进步，异基因移植手术早已被临床广泛应用。 但一个困扰医学界的难题是，即便进行了常规的免疫抑制预防，仍然会有50%-70%的患者在移植手术后出现移植物抗宿主病（GVHD），它的基本原理是，供体的免疫细胞无法识别受体的身体环境，进而对其发动攻击。其中aGVHD（移植手术后100天内发病）是移植后非复发死亡的主要原因之一。 今年1月，铂生生物自主研发的艾米迈托赛注射液获批上市，为激素耐药型aGVHD患者开辟了一条全新的求生通道。 文章开头所讲述的，正是艾米迈托赛获得真实世界验证的典型案例。该患者是一名重型再生障碍性贫血患者，在移植手术后的5-6天内就出现了严重的肠道急性移植物抗宿主病。该患者的母亲表示，为了救治女儿，他们几乎穷尽了所有方案，“该用的药，进口药，国产药都用上了，但效果都不理想。后来医生说这个是干细胞药，刚获批，或许会有希望。” 北京大学人民医院血液科主任莫晓冬教授全程参与了该患者的救治。他表示“艾米迈托赛注射液的上市把急性移植物抗宿主病治疗从单纯的单一靶点、单一途径，过渡到了多靶点多途径的治疗阶段。所以我们对这个药的期待值比较高，希望能为我们治疗急性移植物抗宿主病提供新的武器。” 首方患者的获益并不是孤例。 在战略合作平台阿里健康的推动下，从今年6月开出全国首张处方到今天，艾米迈托赛注射液的药品供应链已极具规模，实现了全国范围的广泛覆盖——在淘宝搜索“艾米迈托赛”进入专题页面，可以看到已有北京、上海、山东、江苏、河南、山西、陕西、湖北、广东等20个省份开通了预约服务，药品的配送范围覆盖全国超半数省份。 “目前，全国有超过20个移植中心临床开始应用艾米迈托赛注射液，受益的患者已经超过百人。”阿里健康相关业务负责人表示。 疗效扎根，前景广阔 作为国内首款、也是唯一一款获批的干细胞药品，艾米迈托赛的疗效已经在真实临床世界获得验证。但这只是一个开始，据了解，艾米迈托赛正在开展更多适应症的临床研究，包括血管衰老、急性肾损伤、糖尿病肾病等。 事实上，进入21世纪后，干细胞治疗就一直是生物制药领域的热门赛道。据调研机构Precedence Research 的分析报告，2023年全球干细胞市场规模为150亿美元，预计到2034 年这一数字将增加至近500亿美元。 相比于欧美国家的先发优势，中国的干细胞治疗还处于从基础研究向临床转化的初级阶段。今年年初艾米迈托赛的获批上市打响了中国干细胞药品研发的第一枪——据不完全统计，当前中国至少有5款干细胞药品已经进入临床三期，涉及膝骨关节炎、慢性移植物抗宿主病、克罗恩病引起的肛瘘等。此外，还有44款干细胞药物获得IND批准正等待启动。因此，2025年也被业内称为中国干细胞元年。 目前，艾米迈托赛的临床应用开展3个月，药品供应链已实现全国范围广泛覆盖，走进超20个植中心开启临床应用，惠及患者上百人。而美国同时期获批的同类药品Ryoncil，实际临床应用只有9例患者。 “美国上市的同类产品19万美金一袋，中国自研的产品大约是两万元人民币一袋，可以显著降低患者的经济负担。”北京大学人民医学血液科主任医师莫晓冬接受央视采访时这样说道。 为新特药铺路搭桥 越是高精尖的新特药，往往会因为低频高价、市场宣教不够，患者缺乏认知等原因，受困于最后一公里的落地。 铂生生物显然深谙此道。为了能够让药品以最快速度和最优价格惠及患者，铂生生物并没有遵循传统的以线下药店为核心网络的经销模式，而是选择了和新特药首发平台——阿里健康达成战略合作，由后者全权负责药品在国内的全渠道独家经销。 回溯过去3个月，为了让创新药更快惠及适用患者，双方团队夜以继日，举措频出。 首先，阿里健康通过淘宝端入口聚合精准流量形成常态化专栏，为这款潜在患者数量少，但地域分布广泛的临床急需药品奠定了市场教育基础。同时，由客服承接患者在线咨询并完成预约，在通过积累多年的医疗供应链布局完成配送。在线上重构了一条全新的药品流通路径。据估算，阿里健康的这一模式为药企大幅降低运营及渠道成本。 其次，对于艾米迈托赛这样的干细胞活体生物制剂来说，运输、储存和真伪验证的要求要远高于传统药品。为了满足艾米迈托塞注射液的安全性和有效性，阿里健康充分调用了其在冷链运输和药品追溯上的能力。 一方面，阿里健康通过自建的冷链供应体系，确保了干细胞药品全程在液氮环境下进行转运，解决了配送难题。而其赖以起家的“码上放心”平台则从生产、运输和终端注射的全链路温湿度对药品进行监控和存证，确保了细胞活性和安全性。 最后，是如何解决患者对“新特药”的用药负担。阿里健康携手众安保险，不仅将艾米迈托赛注射液纳入了旗下尊享e生、众民保系列产品的赔付范围；还推出了针对艾米迈托赛注射液的用药保障——患者可享最高5万元的核心保障权益；大幅提升了该创新药的可负担性。 而如果我们从更广义的视角来看，阿里健康和铂生生物的战略合作，确是树立了一个干细胞药品的商业化样本——一家专注于创新和研发的药企，一个擅长新特药首发的医药电商平台，双方通过战略合作突破了干细胞药品流通中的关键瓶颈，缩短了商业化的周期，短时间内不仅跑通了从开具处方到线上预约，再到冷链配送，最后扫码验真线下注射的业务流；还完成了药品供应链全国范围广泛覆盖；最终让患者更早地用上药，用得起药。 这对于那些跃跃欲试的药企和翘首以盼的患者来说，又何尝不是一种鼓舞？ 运营｜李木子 声明：健识局原创内容，未经许可请勿转载 药店关门也要查！国家医保局出大招：“退保”将倒查1-2年 两家医疗器械上市公司，卷入医院检验科腐败案 新事丨康恩贝一款药物有质量问题，被暂停采购

NEW YORK, Sept. 14, 2025 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, announced that the successful commercial launch of Ryoncil® (remestemcel-L-rknd), the only mesenchymal stromal cell (MSC) product approved by the United States Food and Drug Administration (FDA), was highlighted this past week in presentations at the Morgan Stanley and Cantor Fitzgerald Annual Global Healthcare Conferences held in New York. Mesoblast Chief Executive Silviu Itescu reiterated the strong first quarter launch of Ryoncil® and product sales in the treatment of children with steroid-refractory acute graft versus host disease (SR-aGvHD) as recently reported in the Company’s full year earnings and discussed the continued drive to onboard new sites and increase product usage. Dr. Itescu additionally highlighted plans for expanding Ryoncil® into additional indications including adult SR-aGvHD and inflammatory bowel disease (IBD), as well as key Phase 3 milestones for its second-generation product Rexlemestrocel-L. About Mesoblast Mesoblast (the Company) is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The therapies from the Company’s proprietary mesenchymal lineage cell therapy technology platform respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process. Mesoblast’s Ryoncil® (remestemcel-L-rknd) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months and older is the first FDA-approved mesenchymal stromal cell (MSC) therapy. Please see the full Prescribing Information at www.ryoncil.com. Mesoblast is committed to developing additional cell therapies for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. Ryoncil® is being developed for additional inflammatory diseases including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease. Rexlemestrocel-L is being developed for heart failure and chronic low back pain. The Company has established commercial partnerships in Japan, Europe and China. About Mesoblast intellectual property: Mesoblast has a strong and extensive global intellectual property portfolio, with over 1,000 granted patents or patent applications covering mesenchymal stromal cell compositions of matter, methods of manufacturing and indications. These granted patents and patent applications are expected to provide commercial protection extending through to at least 2041 in major markets. About Mesoblast manufacturing: The Company’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide. Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast Forward-Looking StatementsThis press release includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about: the initiation, timing, progress and results of Mesoblast’s preclinical and clinical studies, and Mesoblast’s research and development programs; Mesoblast’s ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblast’s ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals, manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast’s Ryoncil® for pediatric SR-aGVHD and any other product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblast’s product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblast’s ability to enter into and maintain established strategic collaborations; Mesoblast’s ability to establish and maintain intellectual property on its product candidates and Mesoblast’s ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblast’s expenses, future revenues, capital requirements and its needs for additional financing; Mesoblast’s financial performance; developments relating to Mesoblast’s competitors and industry; and the pricing and reimbursement of Mesoblast’s product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast’s actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise. Release authorized by the Chief Executive. For more information, please contact: Corporate Communications / InvestorsPaul HughesT: +61 3 9639 6036 Media – Global Allison WorldwideEmma NealT: +1 603 545 4843E: emma.neal@allisonworldwide.com Media – AustraliaBlueDot MediaSteve DabkowskiT: +61 419 880 486E: steve@bluedot.net.au