Resecabtagene Autoleucel

As a novel calcineurin inhibitor with proven efficacy in improving renal outcomes, LUPKYNIS addresses a high unmet medical need. With increasing awareness, supportive guidelines, and potential global expansion, it is well-positioned for steady market growth. LAS VEGAS, May 8, 2025 /PRNewswire/ -- DelveInsight's " LUPKYNIS Market Size, Forecast, and Market Insight Report" highlights the details around LUPKYNIS, the first oral lupus nephritis-specific treatment, which has expanded the therapeutic arsenal. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of LUPKYNIS. The report also highlights the historical and forecasted sales from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Aurinia Pharmaceuticals/Otsuka Pharmaceutical's LUPKYNIS (voclosporin) Overview LUPKYNIS (voclosporin) is an oral immunosuppressant classified as a calcineurin inhibitor (CNI), shown to improve both short- and long-term outcomes in adult patients with active lupus nephritis when used alongside standard immunosuppressive therapies. By targeting calcineurin, LUPKYNIS helps suppress cytokine activity, inhibits interleukin-2 (IL-2) production, and reduces T-cell driven immune responses. Although the exact mechanism by which voclosporin inhibits calcineurin is not fully understood, it is known that lymphocyte activation increases intracellular calcium, which binds to calcineurin's regulatory site. This activates the calmodulin-binding catalytic subunit, leading to dephosphorylation and activation of the transcription factor NFATc (nuclear factor of activated T-cells, cytoplasmic). The drug's immunosuppressive effect results in decreased lymphocyte proliferation, reduced cytokine production, and lower expression of T-cell activation markers. The recommended initial dose of LUPKYNIS is 23.7 mg twice daily. It should be used in conjunction with mycophenolate mofetil (MMF) and corticosteroids. Learn more about LUPKYNIS projected market size for lupus nephritis @ LUPKYNIS Market Potential Lupus nephritis is a serious and potentially life-threatening complication of systemic lupus erythematosus (SLE), affecting 40–60% of those diagnosed with the disease. It is typically characterized by symptoms such as hematuria (blood in the urine) and proteinuria (protein in the urine). This condition is more prevalent in women, particularly between the ages of 20 and 40. In 2023, the United States recorded the highest number of lupus nephritis cases among the 7MM, with approximately 211K cases, a figure projected to grow by 2034. Treatment strategies for lupus nephritis depend on the kidney biopsy classification of the disease. Standard therapy often includes corticosteroids in combination with immunosuppressive drugs like cyclophosphamide, mycophenolate mofetil, azathioprine, and calcineurin inhibitors. Rituximab, a monoclonal antibody, is widely used, especially for patients with relapsing or treatment-resistant LN, due to its ability to reduce reliance on steroids. Currently, there are only two FDA-approved medications for lupus nephritis: BENLYSTA (belimumab), available as an intravenous or subcutaneous formulation, and LUPKYNIS (voclosporin), a novel oral calcineurin inhibitor. Leading pharmaceutical companies such as Novartis, AstraZeneca, Roche, and Kezar Life Sciences, among others, are actively developing innovative therapies that could redefine the treatment paradigm for lupus nephritis. The treatment landscape is rapidly evolving, shifting away from broad immunosuppression toward more precise, targeted approaches. Recent drug approvals and a strong pipeline of candidates highlight both advancements and the ongoing challenges in creating treatments that are effective, safe, and long-lasting. Emerging therapies ranging from anti-CD20 antibodies and complement inhibitors to interferon-blocking agents and CAR-T cell therapies hold promise but must address key issues such as safety, sustained efficacy, and accessibility. As our understanding of the disease's underlying mechanisms continues to grow, the future of lupus nephritis treatment is expected to become increasingly personalized, offering renewed hope to patients battling this complex disorder. Discover more about the lupus nephritis market in detail @ Lupus Nephritis Market Report Emerging Competitors of LUPKYNIS The developing pipeline for lupus nephritis treatments shows a growing variety of approaches. Multiple candidates, both in early and late stages of development, are underway, including ianalumab (VAY736) (Novartis), GAZYVA (obinutuzumab) (Roche), SAPHNELO (anifrolumab) (AstraZeneca), FABHALTA (iptacopan) (Novartis), ULTOMIRIS (ravulizumab) (AstraZeneca), as well as advanced cellular therapies like CABA-201 (Cabaletta Bio) and YTB323 (Novartis). In March 2025, the FDA accepted a supplemental Biologics License Application based on data from the Phase III REGENCY trial, where obinutuzumab demonstrated a 46.4% Complete Renal Response (CRR), compared to 33.1% for placebo. Unlike previous anti-CD20 therapies such as rituximab, it provides enhanced B-cell depletion and is the first in its class to show efficacy in a randomized Phase III lupus nephritis trial. If approved, it will become the first CD20-targeted treatment specifically indicated for lupus nephritis, with anticipated sales of USD 400 million by 2034. To know more about the number of competing drugs in development, visit @ LUPKYNIS Market Positioning Compared to Other Drugs Key Milestones of LUPKYNIS In September 2024, Japan's Ministry of Health approved it for Lupus Nephritis treatment alongside mycophenolate mofetil. In September 2022, the European Commission approved LUPKYNIS for active Class III, IV, or V Lupus Nephritis. In January 2021, Aurinia Pharmaceuticals received US FDA approval for LUPKYNIS to treat adult lupus nephritis patients in combination with immunosuppressive therapy. Discover how LUPKYNIS is shaping the lupus nephritis treatment landscape @ LUPKYNIS Lupus LUPKYNIS Market Dynamics LUPKYNIS, developed by Aurinia Pharmaceuticals, is the first FDA-approved oral therapy specifically indicated for active lupus nephritis, a serious manifestation of systemic lupus erythematosus. Approved in January 2021 in the US, LUPKYNIS entered a previously underserved market where treatment was largely dependent on off-label use of immunosuppressants like mycophenolate mofetil and corticosteroids. The drug represents a significant advancement in lupus nephritis care, offering both improved efficacy and a better safety profile compared to existing options, especially in reducing proteinuria and preserving kidney function. The market dynamics for LUPKYNIS are shaped by several key factors. On the positive side, there is a strong unmet medical need, a growing prevalence of lupus nephritis, and increasing physician awareness about early diagnosis and intervention. Moreover, LUPKYNIS benefits from a relatively high pricing strategy, supported by its novel mechanism and disease-modifying potential. Aurinia has also built a dedicated commercial infrastructure and patient support programs to drive uptake and adherence. However, LUPKYNIS faces competitive and reimbursement challenges. One of the main competitors is BENLYSTA (belimumab) from GSK, which gained FDA approval for lupus nephritis in December 2020, just a month before LUPKYNIS. Benlysta has the advantage of brand recognition and a longer track record in treating systemic lupus. Additionally, payers have shown some hesitancy in approving LUPKYNIS due to its cost, requiring extensive prior authorizations and documentation of clinical need. Patient compliance also remains an issue, given the need for frequent monitoring of kidney function and drug levels. Looking ahead, the market potential for LUPKYNIS will depend on its ability to expand its label, demonstrate long-term real-world effectiveness, and potentially enter international markets. Aurinia is also exploring lifecycle management strategies, including combination therapies and expanded indications. Partnerships or acquisitions could further boost market access and penetration. Overall, while LUPKYNIS is a pioneering therapy with strong clinical value, its commercial success will be defined by navigating payer landscapes, physician adoption, and sustained differentiation from competing therapies. Dive deeper to get more insight into LUPKYNIS's strengths & weaknesses relative to competitors @ LUPKYNIS Market Drug Report Table of Contents Related Reports Lupus Nephritis Market Lupus Nephritis Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key lupus nephritis companies, including Hoffmann-La Roche, Chugai Pharmaceutical, Novartis Pharmaceuticals, among others. Lupus Nephritis Pipeline Lupus Nephritis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key lupus nephritis companies, including Hoffmann-La Roche, Qilu Pharmaceutical, Vera Therapeutics, Kyverna Therapeutics, Cabaletta Bio, Takeda, Nkarta Therapeutics, Annexon, Century Therapeutics, Lepton Pharmaceuticals, Transcenta Holding, Inflection Biosciences, among others. Systemic Lupus Erythematosus Market Systemic Lupus Erythematosus Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key SLE companies, including Biogen, Novartis, MorphoSys, Idorsia Pharmaceuticals, Viatris, RemeGen, UCB Pharma, Genentech, Bristol Myers Squibb, AbbVie, among others. Systemic Lupus Erythematosus Pipeline Systemic Lupus Erythematosus Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key myasthenia gravis companies, including Yake Biotechnology, UCB, Sorrento Therapeutics, SinoMab Bioscience Ltd, Shanghai Junshi Biosciences, Sareum, Sanofi, Roche, Rheos Medicine, Resolve, Provention Bio, Pfizer, Novartis, Neovacs, Merck, Medsenic, Landos Biopharma, Kezar Life Sciences, Kangpu Biopharmaceuticals, Janssen Research & Development, Janssen, InnoCare, ImmuPharma, I-MAB Biopharma, ILTOO, Idorsia Pharmaceuticals, Horizon Therapeutics, Genovax, Exinda Thearapeutics, Equillium, Eli Lilly and Company, Eisai, Daiichi Sankyo Company, Corestem, Corbus Pharmaceuticals, Citryll BV, Chipscreen Biosciences, Carna Bioscience, Bristol-Myers Squibb, Brickell Biotech, Boston Pharmaceuticals, Biogen, Athos Therapeutics, Asahi Kasei Pharma, Aria Pharmaceuticals, Antengene Therapeutics, Amgen, Alpine Immune Sciences, Akeso Biopharma, AbbVie, 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 . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

还是Cabaletta Bio，还是自免CAR-T CABA-201，还是神经毒性。本周一，Cabaletta Bio发布了2024年全年收益报告，再次披露了其主要核心管线CABA-201在临床中发生了一起3级免疫效应细胞相关神经毒性综合征 （ICANS）的剂量限制性毒性。这是继2024年8月报告4级ICANS后，CABA-201发生的又一起严重神经毒性事件。神经毒性频发的自免CAR-TCABA-201（resecabtagene autoleucel，Rese-cel）是一款全人源CD19 CAR-T细胞，其使用4-1BB作为共刺激结构域，增强其持久性，以便在一次性输注后耗竭CD19阳性B细胞重置免疫系统，为B细胞驱动的自免疾病患者提供长期缓解。Cabaletta公司正在推进4项CABA-201的1/2期临床研究，包括系统性红斑狼疮（SLE）、肌炎、系统性硬化症和全身性重症肌无力等适应症。此外，Cabaletta公司还将寻常型天疱疮（PV）纳入CABA-201的研究范围，正在进行一项1期研究。CABA-201先后经历了2次严重神经毒性：此前一次是，在针对SLE的1/2期试验RESET-SLE研究中，一名难治性狼疮性肾炎 （LN） 患者报告的4级免疫效应细胞相关神经毒性综合征 （ICANS）。延伸阅读：搞出4级神经毒性，股价大跌34%最近报告的是，在针对系统性硬化症（SSc）的1/2期试验RESET-SSc中，一名患者在接受CABA-201输注前3天报告发热(按照方案要求，对于任何在CABA-201输注前两周出现发热或感染的患者，研究者应该及时与试验的医疗监督员讨论输注的适当性和时间)，在为患者进行CABA-201输注之前，研究者并未通知Cabaletta医疗监控。Cabaletta公司将其描述为“严重的方案偏差”。CABA-201输注后第9天，患者在短暂意识模糊期间经历了3级ICANS的剂量限制性毒性。没有与ICANS事件相关的脑水肿、癫痫发作或运动功能障碍，患者在整个过程中都是可唤醒的。ICANS在地塞米松治疗后迅速消退，患者出院无进一步症状。经过数据审查，独立数据监测委员会建议以目前的剂量进行试验，不延误，并批准Cabaletta的建议，要求研究人员以书面形式向公司肯定确认患者在注射CABA-201前两周内没有发烧或感染的证据。影响似乎不大尽管因为CABA-201再一次报告了3级的ICANS，导致该公司股价于消息发布当日下跌10%，且在后续几日呈持续下跌趋势。但分析师对此并不担心。William Blair的分析师表示，虽然有关ICANS病例的消息令人失望，但这也凸显了自免疾病患者的围输液期管理对于最大限度降低严重安全事件的可能性至关重要。一份分析师的报告表示，如果该患者的发烧报告符合方案，进行全面检查，并确认输液应该推迟至炎症标志物恢复正常，我们认为这一事件是可以预防的。同时基于独立数据监测委员会对于此事件的决定，并未延迟试验，而是支持继续研究。使得CABA-201再发神经毒性这一不良信号，并未给CABA-201试验带来更多波折。总而言之，基于CABA-201在多种适应症上显示出的早期疗效，以及所有患者都停用了免疫抑制剂，部分患者处于或趋向无药物疾病缓解状态。CABA-201还是被看好具有较为可观的前景潜力。延伸阅读：CAR-T的自免临床进展参考资料：1.https://www.cabalettabio.com/news-media/press-releases/detail/127/cabaletta-bio-reports-fourth-quarter-and-full-year-20242.https://www.fiercebiotech.com/biotech/patient-suffers-side-effect-cabaletta-autoimmune-car-t-trial-analysts-unphased