Rexlemestrocel-L

Several leading classes of therapies are exploring diverse mechanisms of action to treat CHF, including SGLT2 inhibitors, cardiac myosin activators, myeloperoxidase inhibitors, mineralocorticoid receptor antagonists, GLP-1 receptor agonists, cell therapies, and more. This growing focus is expected to drive the chronic heart failure market forward. In summary, the existing blockbuster therapies such as Entresto, recently approved SGLT2 inhibitors, and Soluble guanylate cyclase (sGC) stimulators, along with emerging cell therapies, peptides, monoclonal antibodies, and small molecules, together have the potential to drive the existing market along with securing a significant market share upon introduction of new entrants to the CHF treatment landscape. LAS VEGAS, Jan. 22, 2025 /PRNewswire/ -- DelveInsight's Chronic Heart Failure Market Insights report includes a comprehensive understanding of current treatment practices, chronic heart failure emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Chronic Heart Failure Market Report According to DelveInsight's analysis, the market size for chronic heart failure was found to be approximately USD 5.5 billion in the US in 2023. Among the 7MM, the US contributed the largest market share of the total CHF market size and will continue to experience a steep rise in the market size throughout the forecast period. As per DelveInsight's estimate, the total diagnosed prevalent cases of CHF in the 7MM were approximately 20 million in 2023. Heart failure with preserved ejection fraction (HFpEF) contributed to most of the cases. Within class-specific diagnosed prevalence of Heart failure, NYHA class II and class III contribute the maximum. Established chronic heart failure companies with approved therapies in the market are Novartis, Otsuka, Boehringer Ingelheim/Eli Lilly, Bayer/Merck, Amgen/Servier, scPharmaceuticals, and Lexicon Pharmaceuticals/Viatris. In addition, Daiichi Sankyo and American Regent are targeting adult patients with heart failure who are iron deficient. Among all the approved therapies, Novartis and Otsuka's Entresto, approved since 2015 in the US, is still the leading therapy generating more than USD 4 billion in the 7MM in 2023. Among, Eli Lilly's Jardiance and AstraZeneca's Farxiga, both SGLT2 inhibitors, Farxiga is the leading prescribed SGLT2 inhibitor globally by volume Leading chronic heart failure companies developing emerging therapies, such as Cytokinetics, Bayer, Eli Lilly, BioCardia, Mesoblast, Tenax Therapeutics, Novo Nordisk, AstraZeneca, Boehringer Ingelheim, Bristol Myers Squibb, Intra-Cellular Therapies, Cardurion Pharmaceuticals, Rivus Pharmaceuticals, and others are developing novel chronic heart failure drugs that can be available in the chronic heart failure market in the coming years. The promising chronic heart failure therapies in the pipeline include Omecamtiv Mecarbil, KERENDIA (finerenone), Tirzepatide (LY3298176), CardiAMP Cell Therapy, Ziltivekimab (NN6018), REVASCOR (rexlemestrocel-L), Levosimendan (TNX-103), Semaglutide, Balcinrenone (AZD9977) + dapagliflozin, Mitiperstat (AZD4831), Vicadrostat (BI 690517) + Empagliflozin, Cimlanod (CXL-1427/BMS-986231), Lenrispodun (ITI - 214), CRD-740, HU6, and others. In October 2024, Viatris entered into an exclusive licensing agreement with Lexicon Pharmaceuticals for INPEFA (sotagliflozin) in all markets outside of the US and EU. In September 2024, Bayer presented the late-breaking data of Phase III FINE-HEART findings of KERENDIA (finerenone) during a Hot Line session at the European Society of Cardiology (ESC) Congress 2024. In May 2024, Eli Lilly and Company committed an additional USD 5.3 billion manufacturing investment in the company's newest Indiana site to boost API production for tirzepatide and pipeline medicines. AstraZeneca expects the data of the Phase III BalanceD-HF trial of balcinrenone (AZD9977) + dapagliflozin for heart failure in 2025. Discover which therapies are expected to grab the major chronic heart failure market share @ Chronic Heart Failure Market Report Chronic Heart Failure Overview Chronic heart failure is a long-term condition in which the heart's ability to pump blood efficiently is reduced, leading to inadequate circulation of oxygen and nutrients to meet the body's needs. The primary causes of CHF include coronary artery disease, high blood pressure, diabetes, cardiomyopathy, valvular heart disease, and previous heart attacks. Lifestyle factors like obesity, smoking, excessive alcohol consumption, and a sedentary lifestyle can also contribute to its development. The symptoms of CHF vary in severity and may include persistent fatigue, shortness of breath, swelling in the legs, ankles, or abdomen, rapid or irregular heartbeat, and difficulty exercising. Advanced cases can lead to severe fluid retention, wheezing, and an inability to carry out daily activities. Diagnosis typically involves a comprehensive medical history review and a physical examination, followed by tests like an ECG to assess heart rhythm, echocardiography to evaluate heart structure and function, blood tests, chest X-rays, and stress tests. Early diagnosis and management are crucial to improving quality of life and preventing complications. Chronic Heart Failure Epidemiology Segmentation The chronic heart failure epidemiology section provides insights into the historical and current chronic heart failure patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The chronic heart failure market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Diagnosed Prevalent Cases of Heart Failure Gender-specific Cases of Heart Failure Ejection Fraction-specific Cases of Heart Failure New York Heart Association (NYHA) Class-specific Cases of Heart Failure Type-specific Cases of Heart Failure Age-specific Cases of Heart Failure Chronic Heart Failure Treatment Market Current treatments for heart failure primarily rely on angiotensin-converting enzyme (ACE) inhibitors, angiotensin receptor blockers (ARBs), beta-blockers, and diuretics. Additional therapies, such as aldosterone antagonists, amiodarone, antiplatelets, anticoagulants, calcium channel blockers, and nitrates, are also utilized in managing the condition. Most treatment regimens involve a combination of therapies, with beta-blockers being among the most commonly prescribed. Among the approved treatments, Novartis' ENTRESTO has emerged as a leading drug in the congestive heart failure market. Initially slow to gain traction, ENTRESTO has become one of Novartis' key growth drivers, with revenue expected to increase in the coming years. Following its approval for use in patients with preserved ejection fraction, ENTRESTO's market presence has grown significantly. However, ENTRESTO faces growing competition. The entry of new drugs, including SGLT2 inhibitors (such as FARXIGA and JARDIANCE) and VERQUVO, poses a challenge to ENTRESTO's dominance. Additionally, Novartis is set to lose ENTRESTO's exclusivity in the United States in 2025, raising the risk of generic competition. The company is actively engaged in several patent lawsuits to protect its position. SGLT2 inhibitors have also gained a foothold in the CHF market. Although FARXIGA and JARDIANCE are established drugs, they are relatively new in this therapeutic area. The outlook for SGLT2 inhibitors in heart failure is promising, but AstraZeneca and Lilly/Boehringer will have limited time to solidify their positions, as FARXIGA's US patent expires in 2025 and JARDIANCE's in 2028. The recently approved SGLT2 inhibitor sotagliflozin may face challenges in capturing market share from these established treatments. To know more about chronic heart failure treatment guidelines, visit @ Chronic Heart Failure Management Chronic Heart Failure Pipeline Therapies and Key Companies Omecamtiv Mecarbil: Cytokinetics KERENDIA (finerenone): Bayer Tirzepatide (LY3298176): Eli Lilly and Company CardiAMP Cell Therapy: BioCardia Ziltivekimab (NN6018): Novo Nordisk REVASCOR (rexlemestrocel-L): Mesoblast Levosimendan (TNX-103): Tenax Therapeutics Semaglutide: Novo Nordisk Balcinrenone (AZD9977) + dapagliflozin: AstraZeneca Mitiperstat (AZD4831): AstraZeneca Vicadrostat (BI 690517) + Empagliflozin: Boehringer Ingelheim Cimlanod (CXL-1427/BMS-986231): Bristol Myers Squibb Lenrispodun (ITI - 214): Intra-Cellular Therapies CRD-740: Cardurion Pharmaceuticals HU6: Rivus Pharmaceuticals Discover more about chronic heart failure drugs in development @ Chronic Heart Failure Clinical Trials Chronic Heart Failure Market Dynamics The chronic heart failure market dynamics are expected to change in the coming years. An increasing prevalence of cardiovascular diseases, fueled by aging populations and lifestyle-related risk factors such as obesity, hypertension, and diabetes, has significantly raised the demand for effective CHF treatments. Advances in medical technology, including innovative drug therapies, cardiac devices, and regenerative medicine, are expanding treatment options and improving patient outcomes. Growing awareness about heart health and early diagnosis, supported by government and non-government initiatives, is also boosting market expansion. Additionally, the rising focus on personalized medicine and the development of therapies targeting the underlying molecular mechanisms of CHF are creating lucrative opportunities. The increasing healthcare expenditure and the availability of reimbursement policies in developed and emerging economies further bolster the market's growth trajectory. Furthermore, potential therapies are being investigated for the treatment of chronic heart failure, and it is safe to predict that the treatment space will significantly impact the chronic heart failure market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the chronic heart failure market in the 7MM. However several factors may impede the growth of the chronic heart failure market. A key challenge is the underdiagnosis and delayed recognition of CHF due to overlapping symptoms with other conditions and the lack of widespread access to advanced diagnostic tools in many regions. Economic constraints, particularly in low- and middle-income countries, limit patient access to costly therapies, including advanced drugs and devices. Additionally, adherence to treatment regimens remains low due to the complexity of management protocols and the long-term nature of care, leading to suboptimal outcomes. Regulatory hurdles and lengthy approval processes for novel therapies further impede the introduction of innovative treatments. Finally, a limited understanding among general practitioners about newer guidelines and therapies reduces the referral rates to specialists, creating gaps in patient care. Addressing these barriers requires concerted efforts in education, affordability, and healthcare infrastructure. Scope of the Chronic Heart Failure Market Report Therapeutic Assessment: Chronic Heart Failure current marketed and emerging therapies Chronic Heart Failure Market Dynamics: Key Market Forecast Assumptions of Emerging Chronic Heart Failure Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Chronic Heart Failure Market Access and Reimbursement Download the report to understand which factors are driving chronic heart failure market trends @ Chronic Heart Failure Market Trends Table of Contents Related Reports Chronic Heart Failure Epidemiology Forecast Chronic Heart Failure Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology as well as the chronic heart failure epidemiology trends. Chronic Heart Failure Pipeline Chronic Heart Failure Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key CHF companies, including Zensun (Shanghai) Sci & Tech, Cytokinetics, Mesoblast, Shanghai Hongyitang Biopharmaceutical Technology, Tasly Pharmaceuticals, Ionis Pharmaceuticals, Berlin Cures, CardioCell, Help Therapeutics, Eli Lilly and Company, Chong Kun Dang Pharmaceutical, Antlia Biosciences, Cardiol Therapeutics, among others. Acute Coronary Syndrome Market Acute Coronary Syndrome 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 ACS companies, including Novartis, Boehringer Idorsia Pharmaceuticals, Viatris, Recardio, AstraZeneca, Faraday Pharmaceuticals, DalCor Pharmaceuticals, Roche, Jiangsu Vcare PharmaTech, CeleCor Therapeutics, Novo Nordisk, Bristol Myers Squibb, Johnson & Johnson Innovative Medicine, CellProthera, BioCardia, Kancera, Amgen, Arrowhead Pharmaceuticals, Abcentra , among others. Acute Coronary Syndrome Pipeline Acute Coronary Syndrome Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key acute coronary syndrome companies, including Janssen Research & Development, LLC, DalCor Pharmaceuticals, Hyloris Pharmaceuticals, 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 . 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经历漫长的努力后，Mesoblast终于将Ryoncil推向美国市场。 12月18日，根据公告，FDA已批准Ryoncil（Remestemcel-L）作为美国首个间充质基质细胞（MSC）疗法，针对2个月及以上儿童患者的类固醇难治性急性移植物抗宿主病（SR-aGVHD）。 Ryoncil基于同种异体骨髓生成，通过抑制T细胞增殖，下调促炎细胞因子和干扰素的产生，从而调节T细胞介导的炎症反应。 各种标签叠加，令Mesoblast在取得监管突破后，当日股价大涨54.04%，重回四年前水平。 01 首款疗法 虽然Mesoblast已经成立20年，并一直专注开发同种异体（所谓的“现货型”）干细胞疗法，但Ryoncil却并非完全出自其收笔，而要追溯到另一家公司Osiris Therapeutics身上。 1992年，Osiris开始研究骨髓MSC的应用——公司的名称，正来源于埃及神话中的冥王奥西里斯（Osiris），象征着干细胞对生命的重要价值。随后，Osiris推出了由健康青年捐献者骨髓MSC制备而成的Prochymal。 FDA曾在2010年5月，以孤儿药方式核准Prochymal用于I型糖尿病的治疗，但Prochymal的真正商业化从2012年开始。 彼时，Prochymal在加拿大和新西兰成功上市，用于GVHD儿童患者，成为全球首个获批的异体骨髓MSC疗法。次年，Mesoblast从Osiris收购Prochymal，并将其更名为Remestemcel-L，由此开启了新的市场开发。 在Mesoblast手上，Remestemcel-L于2016年2月登陆日本，商品名为Temcell，Mesoblast合作伙伴JCR Pharmaceutics负责销售。 根据日本政府国民健康保险规定，Temcell每袋报销额约合7700美元，而普通成年患者预计至少接受16袋或最多24袋治疗，基于此，Mesoblast曾预计，日本成年患者接受Temcell治疗的报销额在12.3万美元到18.5万美元之间。 然而，Remestemcel-L进军日本市场的成绩似乎并不非常理想。例如在2023初披露的财报中，Mesoblast称，其获得的特许权使用费收入为190万美元，按照固定汇率，也比上一年度的230万美元少了20万美元。 截至2024年6月30日，过去一年来自Temcell的特许权使用费收入为550万美元，同比减少了160万美元，仅有40万美元是由于日元对美元贬值造成的汇率变动。 不过，中源协和CSO、同写意CGT俱乐部理事长张宇认为，这并不能直接说明Temcell有什么实质问题。 “跟美国市场相比，日本市场还是比较小的，所以具体判断它最后的销售潜力，要看在美国上市以后的定价以及后续表现。”张宇补充说。 02 几经周折 的确，无数案例表明，高定价的美国才更像“奶与蜜之地”。但Remestemcel-L赴美之路同样不好走。 2019年5月，Mesoblast首次向FDA提出上市申请，当时的临床资料显示，Remestemcel-L治疗反应率和患者存活率都很高。2020年8月，Mesoblast还获得FDA肿瘤药物咨询委员会的背书，后者以9:1的投票结果赞成批准该药。 然而，2020年10月，FDA却出人意料地拒绝了该申请，并要求至少再进行一次随机对照研究，以便为Remestemcel-L的疗效提供更多证据。 到了2023年3月，Mesoblast再度提交BLA ，数据中纳入至少4年的长期存活结果，以及高危患者的疗效资料。此前，FDA还完成对Mesoblast生产设施的许可前检查，并无任何问题。 可5个月后，2023年8月，FDA仍向Mesoblast发出CRL。在信中，FDA并未提出任何安全性问题，却认为在重新考虑该药物的申请前，该公司应提供更多成年患者的资料。随后，Mesoblast股价暴跌。 Mesoblast首席执行官Silviu Itescu回忆称，收到FDA的CRL次月，他们派人前往沟通，向FDA专家重新展示了Remestemcel-L的各项数据和产品的科学原理。今年1月，Mesoblast跟FDA联系，希望得知还有哪些研究需要补齐，此时FDA的答复是，资料已经足够。 最终，在12月，Remestemcel-L以Ryoncil的商品名获得批准，用于治疗SR-aGVHD。 业界的一个期待是，Ryoncil提供了替代类固醇疗法的选择——GVHD通常需要高剂量类固醇治疗，但对类固醇治疗无反应的患者预后不佳——并且填补针对12岁以下患有GVHD的儿童疗法的空白。 Mesoblast指出，美国每年约有1万名患者接受同种异体造血干细胞移植（allo-HSCT），其中包括约1500名儿童。约有50%的患者会出现GVHD，其中近一半对类固醇没有反应。 相比之下，54名接受allo-HSCT后患有SR-aGVHD的儿童患者参与的Ryoncil研究显示，16名参与者（30%）在接受Ryoncil治疗28天后达到完全缓解，而22名参与者（41%）达到部分缓解。 03 一鼓作气 “随着Ryoncil获得FDA批准，Mesoblast已证明有能力将首款MSC产品推向市场。”Itescu对外表示。根据FDA的批准，Ryoncil将在美国移植中心和医院上市。 9月，Mesoblast曾对外透露，Ryoncil的库存已经准备完毕，并且“已建立供应链，以确保冷冻保存的产品在获得批准后可立即交付，满足每个站点的需求，并有能力在未来根据需要扩大规模”。 Mesoblast聘请了有经验的专业人士，开展针对性的商业战略，“因为50%的儿科移植手术仅在15家中心进行”。所以，Ryoncil的分发将首先面向那些经验最丰富、用量最多的中心，“然后再分阶段推出”。 目前，Ryoncil的美国定价暂未放出。但无论是Mesoblast，抑或其他细胞疗法市场玩家，都对此次获批抱有更多希望。 “Ryoncil是有划时代意义的。从既往FDA批准的细胞疗法看，无论是CAR-T、TCR-T还是TIL，主要还是集中自体产品。”张宇评价说，“FDA的批准代表了一个趋势，未来市场上，同种异体的细胞疗法可能会越来越多。” “我们将继续与FDA密切合作，以获得我们其他后期产品的批准，包括用于心血管疾病的Revascor和用于炎症疼痛适应症的Remestemcel-L，以及扩大Ryoncil在患有炎症的儿童和成人中的适应症。”Mesoblast在公告中写道。 Revascor（Rexlemestrocel-L）同样是一款“现货型”细胞疗法，近期对外发表的III期临床结果显示，使用Revascor可提高缺血性心力衰竭（HF）和炎症高风险患者的生存率，并降低主要发病率。 2018年7月，Mesoblast还与国内的天士力达成合作，在中国开发、生产和商业化Revascor（用于治疗慢性HF）和MPC-25-IC（用于治疗急性心肌梗死）。天士力认购了前者2000万美元的普通股。 从机制上看，T细胞是免疫系统的一部分，由骨髓中的干细胞发育而成。Ryoncil这类MSC疗法的免疫调节作用（包括抑制T细胞活化和促炎细胞因子分泌），使得它们有可能用于治疗过度炎症疾病的其他适应症。 大量数据表明，MSC疗法具备不错的安全性，张宇相信，加上“现货型”的设计，可以令普遍高昂的细胞疗法随着适应症扩大而进一步降低成本。 而Ryoncil，某种程度上就是这场考验的第一位交卷考生。成绩如何，且看来日。 友情推荐：医药行业深度技术内容，点击“博药”查看详情~ 来源 | 同写意（药智网获取授权转载） 撰稿 | 写意君 责任编辑 | 八角 声明：本文系药智网转载内容，图片、文字版权归原作者所有，转载目的在于传递更多信息，并不代表本平台观点。如涉及作品内容、版权和其它问题，请在本平台留言，我们将在第一时间删除。 合作、投稿 | 马老师 18323856316（同微信）  阅读原文，是受欢迎的文章哦