A Monocentric, Randomized, Double-blind Study to Assess the Feasibility of Conducting a Full-scale Randomized Controlled Trial (RCT) Assessing the Effect of Carbidopa/levodopa Ratio on Orthostatic Hypotension (OH) in Multiple System Atrophy - Parkinsonian Type (MSA-P) and Parkinson Disease (PD) Patients (CARBIDOH)

This study is aimed at patients with multi-system atrophy - parkinsonian type (P-MSA) or Parkinson's disease (PD) receiving dopaminergic drugs and suffering from orthostatic hypotension (OH).
OH is a drop in blood pressure when standing, which can lead to symptoms of dizziness, lightheadedness, a black veil in front of the eyes and, when severe, can lead to fainting. HO is one of the symptoms present in AMS-P and PD.
The standard treatment for parkinsonian symptoms of slowness and stiffness is the administration of antiparkinsonian drugs containing dopamine. These dopaminergic drugs always contain 1) levodopa (which is the precursor of dopamine) and 2) an enzyme inhibitor, which may be either benserazide (in the case of Madopar® and its generics) or carbidopa (in the case of Sinemet® or Stalevo® and their generics) and whose role is to potentiate the effect of levodopa.
It has long been known that dopaminergic drugs aggravate HO. Through various mechanisms, this worsening of HO is linked as much to levodopa as to the enzyme inhibitor with which it is combined. However, investigators do not know the respective effects of these two molecules on HO.
In this study, investigators examine how the ratio of Carbidopa to levodopa affects HO in the various assays of the dopaminergic drug under study.

开始日期2025-05-01

申办/合作机构

Centre Hospitalier Universitaire Vaudois

NCT06765668

/ Recruiting临床4期

Open-Label Phase 4 Study of CREXONT® (Carbidopa and Levodopa) Extended Release Capsules in Parkinson's Disease Patients

The primary purpose of this study is to evaluate efficacy and safety of CREXONT under real world conditions in participants with Parkinson disease (PD).

开始日期2025-02-12

申办/合作机构

Impax Laboratories LLC

[+1]

CTR20244711

/ CompletedN/A

卡左双多巴缓释片（ 50 mg/200 mg ）在健康参与者中的单剂量、随机、开放、两序列、四周期、完全重复交叉空腹／餐后状态下的生物等效性研究

主要研究目的：评价重庆赛维药业有限公司生产的卡左双多巴缓释片与MSD Sharp & Dohme GmbH持证的卡左双多巴缓释片（商品名：Sinemet®（息宁®））空腹和餐后单次口服给药在中国健康人群中的生物等效性。次要研究目的：评价受试制剂和参比制剂在中国健康成年参与者中的安全性。评价中国健康研究参与者单次空腹/餐后口服受试制剂和参比制剂后的安全性。

开始日期2024-12-25

申办/合作机构

重庆赛维药业有限公司

100 项与卡比多巴/左旋多巴相关的临床结果

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100 项与卡比多巴/左旋多巴相关的转化医学

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100 项与卡比多巴/左旋多巴相关的专利（医药）

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750

项与卡比多巴/左旋多巴相关的文献（医药）

2025-05-01·JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Risk of fall with device-based advanced treatments in Parkinson’s disease: a systematic review and network meta-analysis

Review

作者: Sorrento, Gianluca ; Rajalingam, Rajasumi ; Fasano, Alfonso

Background:

Deep brain stimulation (DBS) and infusion therapies are effective treatments for the motor complications of Parkinson’s disease (PD), but less established is their role in fall prevention. This systematic review and network meta-analysis (NMA) aimed to evaluate the risk of falls associated with advanced therapies in PD.

Methods:

Following PRISMA-NMA (Preferred Reporting Items for Systematic Reviews and Meta‐Analyses for Network Meta-analyses) guidelines, we searched PubMed, Medline, Embase and CINAHL up to 20 March 2024. Eligibility criteria based on PICOS (Population Intervention Control Outcome Study design) framework were used for DBS of the subthalamic nucleus (STN) or globus pallidus pars interna (GPi), or infusion therapies, compared with best medical treatment (BMT) or sham stimulation. Pairwise meta-analysis was conducted using RevMan V.5.4, and NMA using the netmeta package in R software.

Results:

Fourteen studies were included. A higher number of falls were observed in the DBS group compared with BMT, although the difference was not significant. Sensitivity analysis excluding a heterogeneity-contributing study showed a significantly higher fall risk in the DBS group (Risk Ratio (RR)=2.74, 95% CI 1.60, 4.67, p=0.0002). Subgroup analyses indicated that levodopa-carbidopa intestinal gel tended towards increased fall risk, while continuous subcutaneous infusion of (fos)levodopa (CSCI) significantly decreased risk with high certainty of evidence. NMA showed CSCI as the most effective in reducing falls, while STN DBS was associated with the highest risk.

Conclusions:

DBS, especially targeting the STN, may increase fall risk compared with other advanced non-DBS procedures. While LCIG might not alter fall risk, preliminary evidence suggests that CSCI positively affects fall prevention.

PROSPERO registration number:

CRD42023420637.

2025-03-01·PARKINSONISM & RELATED DISORDERS

Long-term, continuous, subcutaneous levodopa/carbidopa infusion with ND0612 in Parkinson's disease: 3-year outcomes from the open-label BeyoND study

Article

作者: Rudzińska, Monika ; Vasquez, Alberto ; Lopes, Nelson ; Kumar, Rajeev ; Roullet-Solignac, Isabelle ; Case, Ryan ; Muhlack, Siegfried ; Stocchi, Fabrizio ; Isaacson, Stuart ; Aldred, Jason ; Simuni, Tanya ; Taylor, Danette ; Greenberg, Jeffrey ; Azulay, Jean-Philippe ; Antonini, Angelo ; Yardeni, Tami ; Struck, Lynn ; Aralu, Cletus ; Rejdak, Konrad ; Djaldetti, Ruth ; Thomas, Karen ; Poewe, Werner ; Rizova, Yuliya ; Schwarz, Johannes ; Bonuccelli, Ubaldo ; Truong, Daniel ; Pazdera, Ladislav ; Savitt, Joseph ; Flitman, Stephen ; Patel, Neepa ; Gasparoli, Elisabetta ; Klos, Kevin ; Leehey, Maureen ; Ellenbogen, Aaron L. ; Gil, Ramon ; Hauser, Robert A. ; Cutler, Barry ; Hassin-Baer, Sharon ; Durif, Franck ; Espay, Alberto J. ; Bhatia, Perminder ; Defebvre, Luc ; Arkadir, David ; LeWitt, Peter ; Ebersbach, Georg ; Gurevich, Tanya ; Isa, Arnaldo ; Krawczyk, Malgorzata ; Sasson, Nissim ; Milo, Ron ; Kassubek, Jan-Rainer ; Biton, Victor ; Safirstein, Beth ; Klepitskaya, Olga ; Ginsberg, Paul ; Onofrj, Marco ; Yahalom, Gilad ; Danaila, Teodor

INTRODUCTION:

ND0612 is being investigated as a continuous, subcutaneous levodopa/carbidopa infusion, in combination with oral levodopa/carbidopa, for motor fluctuations in Parkinson's disease (PD). One-year data from the ongoing BeyoND study (NCT02726386) showed that the ND0612 regimen was safe and well tolerated and provided a sustained ≥2-h improvement in daily Good ON-time through 12 months of treatment.

METHODS:

We describe 3-year safety and efficacy outcomes for participants who completed 12 months of ND0612 treatment in the core study period and entered the extension phase.

RESULTS:

Of the 214 enrolled participants, 120 completed the core 1-year period, and 114 participants continued into the extension phase. Of these, 95/114 (83.3 %) completed 2 years and 77/114 (67.5 %) completed 3 years of study treatment. Key reasons for discontinuation were treatment-emergent adverse events (TEAEs) (n = 5 and n = 11 after 2 and 3 years, respectively) and withdrawal of consent (n = 9 and n = 5, respectively). TEAEs were reported by 105/114 (92.1 %) participants in Year 1, 77/114 (67.5 %) in Year 2, and 73/95 (76.8 %) in Year 3. While most participants experienced infusion site reactions, these led to discontinuation in only five participants during this extension. At Month 36, the mean reduction in OFF-time from baseline was 2.81 h and the increase in Good ON-time was 2.79 h.

CONCLUSIONS:

Three-year results from this open-label study support the long-term safety, tolerability, and efficacy of ND0612. For participants who entered the extension phase, the high rate of retention supports a favorable benefit-risk ratio of the ND0612 regimen for patients with PD experiencing motor fluctuations.

2025-02-01·EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS

A novel levodopa-carbidopa three-layer gastroretentive tablet for improving levodopa pharmacokinetics

Article

作者: Zhou, Wenhu ; Yan, Peng ; Ding, Jinsong ; Zhao, Xiangcheng ; Zhang, Hailong

The narrow absorption window of levodopa and the significant impact of peripheral decarboxylase are the most limiting factors in maintaining prolonged and smooth plasma concentration in patients with Parkinson's disease (PD). Therefore, this study aims to design a novel gastroretentive carbidopa-levodopa three-layer tablet, which consists of an expansion layer, an immediate-release layer, and a sustained-release layer. The expansion layer rapidly expanded with sufficient structural strength and stayed in the beagle's stomach for more than 10 h, delineating excellent gastric retention effects. The immediate-release layer quickly released the drug and the sustained-release layer maintained a stable drug concentration. Importantly, pharmacokinetic data obtained under fed conditions demonstrated that the duration of efficacy of the three-layer tablets was significantly superior to that of the commercially available product Sinemet® CR, with effective levodopa blood levels remaining for up to 12 h. This is expected to offer more convenient clinical medication options for patients with PD.

219

项与卡比多巴/左旋多巴相关的新闻（医药）

2025-04-14

·微信

2025 MDS 运动波动循证建议更新解读

帕金森病（PD）患者在长期使用左旋多巴治疗后，超过80%的患者最终会出现症状波动（如 “关期” 延长、“开期” 缩短、剂末现象等）1。在另一项国内的研究中，接受左旋多巴治疗的中国患者在近1年内有29.0%出现症状波动，而10年以上人群中症状波动的发生率则高达68.3%2。在一项针对帕金森患者的问卷调查中，高达66.3%的患者认为改善症状波动非常重要3。因此，对帕金森患者的症状波动现象进行更好的管理对改善患者的生活质量有很重要的意义。为了更好地协助医生们针对患者个体选择合适的治疗方案，MDS（International Parkinson and Movement Disorder Society，国际帕金森和运动障碍学会）在2002年第一次基于循证医学证据发表了选择治疗方法的建议，而且之后也在定期更新，最近一次的更新是在2018年。截至目前，针对帕金森病运动症状的治疗方案仍然在不断增加，多项临床试验结果也在陆续发表。因此在2025年，MDS基于102项随机对照试验（RCT，randomized controlled trial），采用了新的评估方法，系统评估了现有治疗手段（包括药物和手术）的有效性与安全性，为临床决策提供了重要参考（图1）4。图1在文章中，针对每一项干预措施的疗效评价都采用了两个维度：（1）结局的临床相关性（the clinical relevance of the outcome）；（2）证据质量（the quality of the evidence）。为了确定临床结局的相关性，如果有临床数据，就会使用相应量表上的最小临床重要差异（MCID，minimal clinically important differences）。对于 “开期” 时长、“关期” 时长以及无异动症的时间长度，最小临床重要差异为每天1小时5。而对于PDQ-39（帕金森病问卷 - 39 项，Parkinson's Disease Questionnaire - 39），最小临床重要差异为5分6。在证据质量方面，采用改良的GRADE方法（modified version of Grading of Recommendations, Assessment, Development, and Evaluations)，基于偏倚风险、不精确性、不一致性、间接性以及发表偏倚的可能性，对每一项干预措施有效性的证据质量进行评估（图2）。图2沙非胺（既往称沙芬酰胺，safinamide）是新一代的MAO-B（ B型单胺氧化酶，monoamine oxidase B）抑制剂，并且是所有药物中唯一的具有双重作用机制抗帕金森的药物。沙非胺除了通过抑制MAO-B对多巴胺递质的分解代谢外，还可以通过阻滞离子通道的途径来抑制过量谷氨酸的释放来改善帕金森患者的症状7。该篇文献经过筛查最终纳入了4项与沙非胺相关的RCT8-11。在4项RCT中，对比评估了沙非胺和安慰剂之间对于改善“开期”和“关期”时间的疗效。全部试验都观察到了具有临床意义的“关期”时间减少（相比安慰剂组减少约1h），并且增加了不伴有令人困扰的运动障碍的“开期”时间（相比安慰剂组增加约1h），显示了沙非胺对于症状波动者的优越疗效。在PDQ-39评分表方面，其中1项研究8显示沙非胺达到了改善日常生活质量的阈值，另一项研究9提示在沙非胺100mg组中，对于安慰剂，PDQ-39评分的改善也超过了5分的阈值均提示了其在改善帕金森病患者日常生活质量的作用。另一方面，值得注意的是MDS目前基于现有研究认为，沙非胺是唯一具有双重作用机制（MAO-B抑制和通道阻滞）的抗帕金森药物。而且在药物有效性方面，相比于前两代MAO-B阻滞剂司来吉兰和雷沙吉兰，其对于症状波动的改善有更高程度的证据支持（沙非胺为有效，司来吉兰为证据不足，雷沙吉兰为可能有效）。并且唑尼沙胺（zonisamide）不再被认为是双机制治疗药物，其对症状波动的有效性方面，MDS也基于最新证据也将其从有效调整为可能有效（图3）。图3此外，IPX 066, ER（卡比多巴 / 左旋多巴缓释剂，Levodopa Carbidopa Extended – Release，IPX 066为其研究代码）在治疗症状波动上也有令人满意的效果，而且是唯一被MDS认为有效的Levodopa/PDI（左旋多巴 / 外周脱羧酶抑制剂）类药物。文中参考了1篇发表于2013年的RCT12。该研究发现IPX 066相比对照组在减少“关期”时间上有明显优势（约1.2 h ，P < 0.0001），并且在“开期”时间上也有明确改善（不伴令人困扰的运动障碍的“开期”时间为0.9 h, P < 0.001；不伴任何运动障碍的“开期”时间为0.7 h, P < 0.05）（图4）。图4在外科治疗方面，MDS也对各种治疗方式的临床有效性做了较大的调整。当前，Bilateral STN DBS（Bilateral Subthalamic Nucleus Deep Brain Stimulation，双侧丘脑底核脑深部电刺激术）是唯一被MDS认为有效的术式。而在上一版推荐中同样被认为有效的Bilateral GPi DBS（Bilateral Globus Pallidus Internus Deep Brain Stimulation，双侧苍白球内侧部脑深部电刺激术）和Unilateral pallidotomy（单侧苍白球切开术）现已被调整为可能有效（图5）。图5时隔7年 MDS 从数据库中15485篇相关文章最终筛选了102项高质量 RCT，并且采用了新的方法来对所有纳入的文章进行治疗有效性分析和证据质量分级。随着时间推移、新药的开发和更多临床试验结果的发表，人们对于帕金森运动波动的理解和治疗观念也随之发生改变：IPX 066成为唯一达到有效级别的 Levodopa/PDI 类药物、普拉克索和罗替高汀的治疗地位仍然稳固、恩他卡朋和雷沙吉兰的有效性级别下降、沙非胺成为唯一有效的双机制药物、唑尼沙胺在有效性级别下降的同时也不再被认为具有通道阻滞的作用、 Bilateral STN DBS是当前证据最充分的针对运动波动症状的手术方案。未来随着更多新药的开发和临床试验的进行，治疗的选择仍然可能会出现变化。 MDS最新的循证医学建议确认了沙非胺对于帕金森病症状波动治疗的有效性。作为新一代的MAO-B抑制剂，沙非胺可以抑制MAO-B对于多巴胺递质的代谢，同时调节钠离子和钙离子通道抑制过量谷氨酸的释放，通过目前唯一的双重抗帕金森机制，可以明显减少“关期”时间并且增加不伴有令人困扰的运动障碍的“开期”时间，其在改善PDQ-39评分方面也有一定的优势，并且对于非运动症状也有明显的改善作用13-15。在帕金森病的治疗中，尤其是出现运动并发症的患者，沙非胺将会是很有优势的选择。参考文献1. Stocchi F, Antonini A, Barone P,et al. Early DEtection of wEaring off in Parkinson disease: the DEEP study. Parkinsonism Relat Disord. 2014 Feb;20(2):204-11. 2. Wan Y, Yuan C. Hou X, et al. Wearing-off Identification in Parkinson's Disease: The shapd-woq Study. Front Neurol. 2020 Mar 13:11:116.3. Wüllner U, Fuchs G, Reketat N, et al. Requirements for Parkinson's disease pharmacotherapy from the patients' perspective: a questionnaire-based survey. Curr Med Res Opin. 2012 Jul;28(7):1239-46. study. Parkinsonism Relat Disord. 2014 Feb;20(2):204-11. 4. de Bie RMA, Katzenschlager R, Swinnen BEKS, Peball M, Lim SY, Mestre TA, Perez Lloret S, Coelho M, Aquino C, Tan AH, Bruno V, Dijk JM, Heim B, Lin CH, Kauppila LA, Litvan I, Spijker R, Seppi K, Costa J, Sampaio C, Fox SH, Silverdale MA. Update on Treatments for Parkinson's Disease Motor Fluctuations - An International Parkinson and Movement Disorder Society Evidence-Based Medicine Review. Mov Disord. 2025 Mar 8. doi: 10.1002/mds.30162. Epub ahead of print. PMID: 40055961. 5. Hauser RA, Gordon MF, Mizuno Y, et al. Minimal clinically important difference in Parkinson’s disease as assessed in pivotal trials of pramipexole extended release. Parkinsons Dis 2014;2014: 467131. 6. Horvath K, Aschermann Z, Kovacs M, et al. Changes in quality of life in Parkinson’s disease: how large must they be to be relevant Neuroepidemiology 2017;48(1–2):1–8. 7. Calabresi p, Kulisevsky J. Safinamide as Add-on Therapy - Moving Beyond Dopamine for a Multifaceted Approach in Parkinson's Disease.European Neurological Review,2017:12(Suppl. 5):2-6. 8. Borgohain R, Szasz J, Stanzione P, et al. Randomized trial of safinamide add-on to levodopa in Parkinson’s disease with motor fluctuations. Mov Disord 2014;29(2):229–237. 9. Hattori N, Tsuboi Y, Yamamoto A, Sasagawa Y, Nomoto M. Efficacy and safety of safinamide as an add-on therapy to L-DOPA for patients with Parkinson’s disease: a randomized, double-blind, placebo- controlled, phase II/III study. Parkinsonism Relat Disord 2020;75:17–23. 10. Schapira AHV, Fox SH, Hauser RA, et al. Assessment of safety and efficacy of safinamide as a levodopa adjunct in patients with Parkinson disease and motor fluctuations: a randomized clinical trial. JAMA Neurol 2017;74(2):216–224. 11. Wei Q, Tan Y, Xu P, et al. The XINDI study: a randomized phase III clinical trial evaluating the efficacy and safety of safinamide as add-on therapy to levodopa in Chinese patients with Parkinson’s disease with motor fluctuations. CNS Drugs 2022;36:1217–1227. 12. Hauser RA, Hsu A, Kell S, et al. Extended-release carbidopa-levodopa (IPX066) compared with immediate-release carbidopa-levodopa in patients with Parkinson’s disease and motor fluctuations: a phase 3 randomised, double-blind trial. Lancet Neurol 2013;12(4): 346–356. 13. Cattaneo C, M¨uller T, Bonizzoni E, et al. Long-Term Effects of Safinamide on Mood Fluctuations in Parkinson's Disease. J Parkinsons Dis. 2017;7(4):629-634. 14. Cattaneo C, Barone P, Bonizzoni E, et al. Effects of Safinamide on Pain in Fluctuating Parkinson's Disease Patients: A Post-Hoc Analysis. J Parkinsons Dis. 2017;7(1):95-101. 15. García DS, López IC, Guerra CL,et al. Safinamide improves sleep and daytime sleepiness in Parkinson's disease: results from the SAFINONMOTOR study. Neurol Sci. 2022 Apr;43(4):2537-2544. 提示：本材料仅供医疗卫生专业人士进行医学科学交流，不用于推广目的。

临床研究临床结果

2025-04-03

·药明康德

全球药企研发投入TOP 15榜单揭晓！

▎药明康德内容团队编辑近日，行业媒体Endpoints News发布了2024全球生物医药企业研发投入TOP 15榜单，药明康德内容团队将结合公开资料为读者解析上榜企业在2024年扩展研发管线的战略布局，旨在通过这些企业的创新动向展现整个产业的发展趋势。点击文末“阅读原文/read more”，即可访问原始榜单页面。▲2024年研发投入最多TOP 15生物医药公司（图片来源：药明康德内容团队根据参考资料[1]制表）默沙东2024年，默沙东通过“收购+合作”双轨策略，在多个治疗领域实现深度布局：年初，公司以6.8亿美元收购Harpoon Therapeutics，斩获DLL3靶向T细胞接合器HPN328；5月，斥资30亿美元收购EyeBio，囊获四价三特异性抗体Restoret；12月又拿下翰森制药的HS-10535全球权益，进一步拓展小分子药物管线，并成功进军GLP-1领域。与此同时，默沙东加速布局前沿技术：公司与Pearl Bio达成10亿美元合作，开发含非标准氨基酸的生物制品疗法；引入Variational AI的生成式AI平台优化药物发现，并与礼新医药就PD-1/VEGF双抗达成最高27亿美元里程碑合作。不久前，默沙东还与恒瑞医药就小分子心血管新药Lp（a）抑制剂达成授权合作。这一系列举措不仅加深了默沙东在实体瘤领域的研发能力，还通过新兴技术平台主动构建未来增长引擎。罗氏罗氏正加速推进其核心研发管线，聚焦肿瘤、神经退行性疾病及呼吸系统疾病等领域。在神经退行性疾病领域，罗氏近期公布了其靶向淀粉样蛋白的单抗药物trontinemab用于治疗轻度至中度阿尔茨海默病（AD）患者的1b/2a期试验积极结果。另一款药物prasinezumab也有望于年内获得支持进入3期临床的关键数据。在慢性阻塞性肺病（COPD）领域，罗氏旗下IL-33抑制剂astegolimab的两项临床试验数据预计将于今年揭晓。在RNA治疗领域，罗氏与Alnylam合作开发的zilebesiran在治疗高血压患者的2期临床试验中展示了显著疗效。免疫学管线方面，其BTK抑制剂fenebrutinib在针对多发性硬化的2期试验中表现良好，有望在今年获得关键数据；IL-6抗体vamikibart针对葡萄膜炎性黄斑水肿的3期研究也将公布结果。从战略布局来看，罗氏通过选择性并购与全球合作进一步夯实其研发管线。公司斥资超30亿美元收购Carmot Therapeutics，以强化其在代谢疾病领域的布局；以超10亿美元收购Poseida Therapeutics以获得其现货型CAR-T技术；此外，罗氏还以近8000万美元预付款与信达生物达成合作，共同开发小细胞肺癌疗法。与此同时，罗氏与Oxford BioTherapeutics达成多年合作，聚焦抗体疗法开发，延续其在肿瘤免疫领域的深度布局。阿斯利康阿斯利康持续加码研发创新，研发投入从2019年的60亿美元跃升至2024年的逾130亿美元，驱动其从肿瘤领域向其他疾病领域加速扩张。在肿瘤管线，口服选择性雌激素受体降解剂（SERD）camizestrant的3期临床积极数据，加深了该疗法在乳腺癌治疗领域的重要性；在心血管疾病领域，阿斯利康通过收购CinCor Pharma获得新型醛固酮合成酶抑制剂baxdrostat，该药物在2期临床试验可显著降低难治性高血压患者的血压水平，为临床治疗难治性高血压提供了潜在新选择。战略布局层面，阿斯利康动作频出：与Allorion Therapeutics达成独家选择权和全球许可协议，共同开发EGFR L858R突变靶向变构抑制剂，瞄准晚期EGFR突变非小细胞肺癌（NSCLC）治疗缺口；斥资10.5亿美元收购Amolyt Pharma，获得处于3期临床阶段的甲状旁腺功能减退症创新多肽疗法eneboparatide（AZP-3601）；更斥资20亿美元收购Fusion Pharmaceuticals，整合其放射偶联药物（RDC）平台及FPI-2265等管线，强化肿瘤精准治疗布局。强生强生近期在免疫学领域取得关键研究进展：从Protagonist Therapeutics引进的口服IL-23受体拮抗剂icotrokinra针对斑块状银屑病的3期临床试验达到主要终点。在管线推进方面，靶向新生儿Fc受体的抗体疗法nipocalimab迎来多项进展。该疗法针对全身性重症肌无力（gMG）适应症已向FDA递交生物制品许可申请（BLA）并获得优先审评资格，同时向EMA同步提交上市许可申请（MAA），有望成为重磅疗法。战略布局方面，强生斥资146亿美元收购Intra-Cellular Therapies，斩获每日一次口服精神疾病治疗药物Caplyta（ITI-007）和ITI-1284，同步整合其临床阶段研发管线。艾伯维2024年，艾伯维专注多元化管线的拓展。在核心产品方面，免疫领域药物Skyrizi获批溃疡性结肠炎适应症；肿瘤领域亦取得重要突破，其抗体偶联药物（ADC）Elahere获FDA完全批准；神经科学领域则推出创新产品Vyalev，该疗法为首款获FDA批准的基于左旋多巴的皮下24小时持续输注帕金森病疗法。为补充研发管线，艾伯维开展了一系列战略收购，包括以2亿美元收购Nimble获得口服肽类IL-23R抑制剂，纳入Celsius的TREM1抗体和Landos Biopharma的口服NLRX1激动剂强化炎症性肠病治疗领域，以及收购Aliada布局阿尔茨海默病临床阶段疗法ALIA-1758。在创新合作方面，艾伯维同样成果丰硕：与EvolveImmune Therapeutics达成15亿美元合作开发多特异性抗肿瘤疗法，联合Umoja Biopharma推进原位CAR-T细胞疗法研发，携手Gilgamesh Pharmaceuticals合作开发针对精神疾病的下一代疗法，与Parvus Therapeutics共同开发炎症性肠病的新疗法，以及与OSE Immunotherapeutics和Tentarix Biotherapeutics建立合作共同推进肿瘤学和免疫学等领域的新药研发。通过一系列收购和合作，艾伯维不仅加强其在免疫学和肿瘤学领域的布局，还在神经科学领域取得了重要进展。百时美施贵宝百时美施贵宝持续加大研发投入以巩固创新优势，2024年研发投入再攀新高，公司通过战略性并购与合作增强其管线建设。在神经精神疾病领域，百时美施贵宝斥资140亿美元收购Karuna Therapeutics获得突破性药物Cobenfy（KarXT），该药已获FDA批准治疗成人精神分裂症，成为近数十年来FDA批准的首个基于新机制的精神分裂症药。此外，该药物用于治疗阿尔茨海默病患者精神病发作的潜力。也引发了高度关注。在合作方面，百时美施贵宝不断迭代其管线：公司与BioArctic达成合作，共同开发靶向PyroGlu-Aβ的阿尔茨海默病抗体疗法BAN1503和BAN2803；携手Prime Medicine开发下一代T细胞疗法；与VantAI合作部署分子胶技术平台。通过这些举措，百时美施贵宝在多个关键领域实现了战略布局，并进一步增强了其创新能力。礼来2024年，礼来公司持续在减重和阿尔茨海默病治疗领域发力。在肥胖症治疗领域，其口服GLP-1受体激动剂orforglipron在2期临床试验中展现出显著的减重效果，其3期试验正在推进；同时，公司布局的多靶点GLP-1/GIP/GCG激动剂retatrutide在2期研究中使肥胖或超重成人患者在经过24周的治疗后，达成至少15%的体重下降。在神经退行性疾病管线中，靶向β淀粉样蛋白的remternetug（每季度一次注射）也已进入关键临床阶段。在心血管和肿瘤领域，礼来也在同步发力：降血脂药物lepodisiran与muvalaplin正在加速临床开发；KRAS抑制剂olomorasib展现出显著的潜力。战略布局层面，礼来通过多重手段扩展创新边界，包括以32亿美元收购Morphic Holding，获得炎症性肠病口服整合素靶向疗法MORF-057，进一步强化自身免疫学管线；与KeyBioscience合作开发胰淀素/降钙素双重激动剂；携手HAYA Therapeutics利用RNA引导基因调控平台推进代谢疾病的临床前药物研发；并与Isomorphic Labs达成合作，借助AlphaFold技术加速小分子药物发现。通过持续的研发投入与精准并购，礼来成功构建了一个覆盖代谢、神经、心血管及肿瘤领域的创新生态。辉瑞2024年，辉瑞公司通过创新疗法研发和战略合作持续强化其研发管线布局。在肿瘤领域，高选择性CDK4抑制剂atirmociclib展现突出潜力，在针对经多线治疗的乳腺癌患者的1期临床试验中，atirmociclib组合疗法实现32%的客观缓解率（ORR），显著优于现有疗法（ORR<10%）且安全性良好。在代谢疾病领域，辉瑞重点推进口服小分子GLP-1受体激动剂danuglipron用于肥胖治疗的临床开发。疫苗领域也取得了重要突破，Abrysvo获FDA批准扩展适应症，用于预防18至59岁高风险个体因呼吸道合胞病毒（RSV）引起的下呼吸道疾病。辉瑞同时积极开展外部合作：与Quotient Therapeutics合作探索心血管和肾脏疾病的新疗法；通过旗下Ignite平台与Acepodia公司合作开发自身免疫疾病疗法；与Flagship Pioneering旗下两家公司Ampersand Biomedicines和Montai Therapeutics就肥胖症和非小细胞肺癌治疗开展靶向分子研发合作。诺华2024年，诺华继续推动包括Fabhalta、Leqvio和Scemblix等核心产品的商业化进程。在后期管线中，脊髓性肌萎缩（SMA）疗法OAV101有望扩大患者覆盖范围，而从PTC Therapeutics引进的亨廷顿病治疗药物votoplam（PTC518）也展现出重大潜力。此外，诺华通过一系列战略性收购与合作加速创新：以11亿美元收购Kate Therapeutics强化其在神经科学和基因治疗领域布局；30亿美元收购MorphoSys增强肿瘤及血液学领域管线；与Ratio Therapeutics合作开发放射性治疗药物；与Arvinas达成超10亿美元协议获得其PROTAC降解剂项目；12亿美元携手Voyager Therapeutics推进亨廷顿病和SMA基因疗法研发。这一系列举措展现了诺华在肿瘤、神经科学和罕见病等关键治疗领域的深度布局，为其长期发展奠定坚实基础。GSK2024年，GSK通过核心产品创新和战略收购持续强化其产业布局。在疫苗领域，GSK公布其带状疱疹疫苗Shingrix的十年保护效力积极数据。在呼吸系统疾病方面，一年两次给药的IL-5单抗depemokimab在3期临床研究中取得积极进展，并已向美国FDA提交生物制品许可申请。在抗感染领域，创新抗生素Blujepa（gepotidacin）获批上市。与此同时，GSK也正通过一系列战略性布局加速管线拓展：公司斥资14亿美元收购Aiolos Bio获得长效抗胸腺基质淋巴细胞生成素（TSLP）单抗AIO-001；以8.5亿美元收购恩沐生物（Chimagen Biosciences）的双靶点T细胞接合器CMG1A46，并与Muna Therapeutics、Vesalius Therapeutics等公司合作开发神经退行性疾病创新疗法。此外，GSK还获得了映恩生物（Duality Biologics）ADC药物DB-1324的全球权益（除大中华区），进一步丰富了其在肿瘤领域的产品组合。赛诺菲2024年，赛诺菲持续加大研发投入，并在管线推进方面取得显著进展。在呼吸疾病领域，IL-33单抗Itepekimab针对COPD的适应症已进入3期临床开发阶段，数据预计于下半年公布；同时，针对哮喘的双抗疗法lunsekimig已推进至2期临床阶段。在血液肿瘤管线中，BTK抑制剂tolebrutinib获得FDA优先审评资格，PDUFA日期定于2025年9月28日。此外，公司管线中的其他项目也在积极推进，包括蛋白降解药物KT-474和口服BTK抑制剂rilzabrutinib。与此同时，赛诺菲深化战略合作以布局未来增长点。公司与Orano Med达成3亿欧元股权投资及3.2亿欧元独家许可协议，联合开发下一代放射性配体药物，开辟肿瘤精准治疗新路径；并与Synthekine合作优化IL-10受体激动剂，旨在满足炎症性疾病的未满足需求。此外，赛诺菲还以17亿美元收购Inhibrx Biosciences，获得α-1抗胰蛋白酶缺乏症（AATD）候选药物INBRX-101。诺和诺德2024年，诺和诺德聚焦三大核心治疗领域：糖尿病/肥胖症/代谢功能障碍相关脂肪性肝炎（MASH）、心血管/慢性肾病以及罕见病。其重点产品CagriSema（cagrilintide/司美格鲁肽复方制剂）在3期临床试验中展现出积极疗效，治疗68周可实现22.7%的显著减重效果。为拓展创新管线，诺和诺德开展了一系列战略布局：斥资10.25亿欧元收购Cardior公司，获得其处于2期临床阶段的心力衰竭反义寡核苷酸疗法CDR132L；与Ascendis Pharma合作开发每月一次给药的GLP-1受体激动剂；同时与EraCal、Metaphore、Omega Therapeutics和Cellarity公司等多家企业达成合作协议，共同开发新一代减重疗法和MASH治疗药物。安进2024年，安进公司聚焦核心创新药物布局，重点推进代谢疾病和心血管领域突破性疗法。其注射型肥胖症药物MariTide在2期临床试验中展现出显著疗效，治疗52周实现约20%的平均体重减轻，并显著改善心血管代谢指标，目前已进入关键后期试验阶段。在心血管领域，siRNA疗法olpasiran在2期试验中表现优异，可使98%患有动脉粥样硬化性心血管疾病（ASCVD）患者的载脂蛋白（a）水平降至标准值以下，目前正在ASCVD患者中开展3期临床试验，预计2026年公布结果。安进同时积极推进其他重点产品：CD19抗体Uplizna今日获FDA批准，用于治疗免疫球蛋白G4相关疾病（IgG4-RD）成人患者；治疗特应性皮炎的rocatinlimab则在3期试验中达成所有主要和次要终点，展现出显著的临床改善效果。吉利德科学2024年，吉利德科学通过创新疗法突破和战略合作持续推动多元化发展。其在肿瘤治疗领域取得重要进展，与Arcellx合作开发的BCMA靶向CAR-T疗法anito-cel在治疗复发/难治性多发性骨髓瘤（RRMM）的2期临床试验中表现优异：总缓解率高达97%，12个月总生存率达到96.5%，且安全性良好，未出现迟发性神经毒性。同时，公司以43亿美元收购CymaBay获得了原发性胆汁性胆管炎（PBC）治疗seladelpar，显著增强了自身肝脏疾病产品管线，该药物已在2024年8月被FDA批准。此外，吉利德还通过多项战略合作加速创新布局：与Tubulis、Xilio Therapeutics及Merus达成总额超49亿美元的合作协议，重点开发ADC、肿瘤激活IL-12蛋白及三特异性抗体等前沿疗法；追加3.2亿美元投资Arcus Biosciences以推进联合开发项目。这些举措加强了吉利德在肿瘤学和肝脏疾病等新治疗领域的战略布局与创新实力。再生元2024年，再生元公司聚焦肿瘤、免疫和基因治疗三大领域，构建了丰富的创新药物管线。在肿瘤领域，抗LAG-3单抗fianlimab联合PD-1疗法展现出显著疗效，在非小细胞肺癌和黑色素瘤患者中达到近60%的客观缓解率；双特异性抗体linvoseltamab已向FDA和EMA提交上市申请，用于治疗多发性骨髓瘤。此外，与Intellia合作的CRISPR基因编辑疗法NTLA-2001在治疗转甲状腺素蛋白淀粉样变性（ATTR）的1期临床试验中取得积极结果。在代谢疾病和呼吸系统疾病领域，抗GDF8单抗trevogrumab和抗IL-33单抗itepekimab等创新疗法正处于后期临床开发阶段，展现了公司在多元化治疗领域的研发实力。除了研发进展，再生元在过去的一年还达成了多项合作与交易：与Mammoth Biosciences达成1亿美元协议开发体内CRISPR基因编辑疗法；通过收购2seventy bio的细胞疗法管线成立Regeneron Cell Medicines，探索细胞疗法与抗体药物的联合应用。▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料：[1] Big ambitions at Merck and AstraZeneca — along with a few giant pitfalls — reshape top ranks of the R&D 15. Retrieved April 3, 2025 from https://endpts.com/big-ambitions-at-merck-and-astrazeneca-along-with-a-few-giant-pitfalls-reshape-top-ranks-of-the-rd-15/[2] 各公司官网免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

并购临床3期临床2期免疫疗法

2025-04-03

·PRNewswire

Mitsubishi Tanabe Pharma America to Highlight Latest Advances in Neurodegenerative Research at the American Academy of Neurology 2025 Annual Meeting

Presentations will highlight findings from studies evaluating investigational ND0612 in Parkinson's disease and RADICAVA® (edaravone) formulations in ALS JERSEY CITY, N.J., April 3, 2025 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) announced 11 presentations will be shared at the American Academy of Neurology (AAN) 2025 Annual Meeting, taking place April 5-9 in San Diego, CA, and online. The presentations will discuss two posters in Parkinson's disease (PD) and nine in amyotrophic lateral sclerosis (ALS). "We are honored to join the scientific community at this year's AAN meeting, where we will discuss key insights from our clinical and real-world studies across PD and ALS," said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs, MTPA. "Our presence at the meeting, and the data presented, underscore our dedication to advancing impactful research and deepening our understanding of these progressive neurodegenerative diseases." PD data to be presented at the meeting include: Investigational ND0612: Presentations will highlight findings from the pivotal Phase 3 BouNDless trial (NCT04006210), including an oral presentation detailing the impact of 24-hour treatment with ND0612 on PD motor state transitions throughout the day, as well as an analysis of quality of life outcomes in people with PD experiencing motor fluctuations. Impact of 24-hour Subcutaneous Levodopa/Carbidopa Infusion (ND0612) on Motor Function: Efficacy Outcomes from the Phase 3 BouNDless Study (Rajesh Pahwa, M.D.; University of Kansas Medical Center) Scientific Platform Session S37 (#001): 1:00 p.m. – 1:12 p.m. PDT, April 9 Quality of Life With 24-hour Subcutaneous Levodopa/Carbidopa Infusion (ND0612): QoL Results from a Phase 3, Randomized, Active-Controlled Study (Harini Sarva, M.D., FAAN; New York Presbyterian Hospital) Poster Session P11 (#5-007): 8:00 a.m. – 9:00 a.m. PDT, April 9 ALS data to be presented at the meeting include: RADICAVA ORS Studies: A presentation will share final results from the MT-1186-A03 extension study, which evaluated the safety and tolerability of RADICAVA ORS® (edaravone) over 96 weeks in ALS patients who had previously participated in MT-1186-A01. Additionally, findings from the MT-1186-A04 extension study will be presented, assessing the continued efficacy and safety of RADICAVA ORS over an additional 48 weeks, comparing investigational daily dosing with the U.S. Food and Drug Administration (FDA)-approved on/off regimen. Lastly, an analysis will examine the impact of RADICAVA ORS on survival and function in ALS patients, leveraging data from the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database and propensity score matching to compare RADICAVA ORS-treated patients with participants from studies MT-1186-A01 through A04. Phase 3, Open-Label, Safety Extension Study of Oral Edaravone (Radicava ORS®) Administered Over 96 Weeks in Patients with ALS (MT-1186-A03) (Alejandro Salah, M.D., Ph.D., MBA, MHA; MTPA) Scientific Platform Session S27 (#007): 2:12 p.m. – 2:24 p.m. PDT, April 8 Extension Study MT-1186-A04 Evaluating Oral Edaravone (Radicava ORS®) Continued Efficacy and Safety up to an Additional 48 Weeks in Patients With ALS (Stephen Apple, M.D.; MTPA) Poster Session P9 (#2-007): 11:45 a.m. – 12:45 p.m. PDT, April 8 Long-term Function and Survival of Radicava ORS® (Oral Edaravone)-Treated Patients With ALS vs Propensity Score–Matched PRO-ACT Historical Controls (Alejandro Salah, M.D., Ph.D., MBA, MHA; MTPA) Poster Session P8 (#2-003): 8:00 a.m. – 9:00 a.m. PDT, April 8 Real-World Data: Multiple posters highlighting real-world data will be presented, including two studies evaluating at-home telespirometry in ALS. One examines changes in erect and supine slow vital capacity based on non-invasive ventilation (NIV) status, while the other assesses point-of-care quality classifications for slow and forced vital capacity measurements. Additionally, an analysis of real-world data will examine trends in ALS treatment utilization in the U.S. from 2016 to 2022, highlighting shifts in prescribing patterns, adoption of FDA-approved therapies and the decreasing proportion of untreated patients. Other presentations include an analysis of referral patterns and healthcare utilization among people with ALS enrolled in the OPTUM claims database, a secondary analysis describing demographics and healthcare resource utilization across specialty vs. non-specialty treatment centers, and the application of a clinical algorithm to electronic medical records to reduce the delay in time to diagnosis and treatment for patients with ALS. Smartphone─Mediated At-Home Telespirometry Erect and Supine Slow Vital Capacity Decline Differences Based On NIV Status in Subjects With ALS (Stephen Apple, M.D.; MTPA) Poster Session P8 (#2-004): 8:00 a.m. – 9:00 a.m. PDT, April 8 Smartphone─Mediated, At-Home Telespirometry Vital Capacity (VC) Measurements in ALS: Point-of-Care Quality Assessment for Slow VC and Forced VC (Stephen Apple, M.D.; MTPA) Poster Session P8 (#2-006): 8:00 a.m. – 9:00 a.m. PDT, April 8 Evolution of the Treatment Landscape for Patients Diagnosed with Amyotrophic Lateral Sclerosis in the United States (Malgorzata Ciepielewska, M.S.; MTPA) Poster Session P9 (#2-006): 11:45 a.m. – 12:45 a.m. PDT, April 8 Referral Patterns for Patients With Amyotrophic Lateral Sclerosis Enrolled in a US-Based Administrative Claims Database (Malgorzata Ciepielewska, M.S.; MTPA) Poster Session P8 (#2-009): 8:00 a.m. – 9:00 a.m. PDT, April 8 Treatment of Amyotrophic Lateral Sclerosis in General and Specialist US Healthcare Settings: Results From a Real-World Survey (Malgorzata Ciepielewska, M.S.; MTPA) Poster Session P8 (#2-012): 8:00 a.m. – 9:00 a.m. PDT, April 8 Application of a Clinical Algorithm on Real-World Electronic Medical Record Data to Assist With Earlier Detection of Amyotrophic Lateral Sclerosis (Amanda E. Fiander, M.D., FRCPC, CSCN (EMG); Maritime Neurology) Poster Session P8 (#2-010): 8:00 a.m. – 9:00 a.m. PDT, April 8 About RADICAVA ORS® (edaravone) The U.S. Food and Drug Administration (FDA) approved RADICAVA ORS® (edaravone) on May 12, 2022, for the treatment of amyotrophic lateral sclerosis (ALS). In 2024, the FDA granted RADICAVA ORS Orphan Drug Exclusivity based on its major contribution to patient care by providing an oral suspension route of administration that avoids the burdens of IV administration. RADICAVA ORS is taken daily for 14 consecutive days followed by a 14-day drug-free period for the initial treatment cycle. For subsequent treatment cycles, RADICAVA ORS is taken for 10 days within a 14-day period followed by a 14-day drug-free period. Each 105 mg (5mL) dose of RADICAVA ORS should be taken in the morning after overnight fasting. Patients should not eat or drink (except water) within one hour after taking RADICAVA ORS.1 Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and commercialized in the U.S. by Mitsubishi Tanabe Pharma America, Inc. (MTPA). The MTPC group companies began researching ALS in 2001 through an iterative clinical platform over a 13-year period. In 2015, edaravone was approved as RADICUT® for the treatment of ALS in Japan and South Korea. Marketing authorizations were subsequently granted in Canada (October 2018), Switzerland (January 2019), Indonesia (July 2020), Thailand (April 2021), Malaysia (December 2021) and Brazil (February 2024). Marketing authorization for RADICAVA® Oral Suspension was granted in Canada (November 2022) and Switzerland (May 2023), and RADICUT® Oral Suspension 2.1% was granted regulatory approval in Japan in December 2022. To date, in the U.S., RADICAVA ORS, along with the previously available IV RADICAVA® (edaravone), have been used to treat over 16,000 people with ALS, with over 2.0-million days of therapy, and have been prescribed by over 2,400 HCPs.2-4 INDICATION RADICAVA ORS® (edaravone) is indicated for the treatment of amyotrophic lateral sclerosis (ALS). IMPORTANT SAFETY INFORMATION Hypersensitivity Reactions RADICAVA ORS® (edaravone) is contraindicated in patients with a history of hypersensitivity to edaravone or any of the inactive ingredients of this product. Hypersensitivity reactions (redness, wheals, and erythema multiforme) and cases of anaphylaxis (urticaria, decreased blood pressure, and dyspnea) have occurred. Patients should be monitored carefully for hypersensitivity reactions. If hypersensitivity reactions occur, discontinue RADICAVA ORS, treat per standard of care, and monitor until the condition resolves. Sulfite Allergic Reactions RADICAVA ORS contain sodium bisulfite, a sulfite that may cause allergic-type reactions, including anaphylactic symptoms and life-threatening or less severe asthmatic episodes in susceptible people. The overall prevalence of sulfite sensitivity in the general population is unknown but occurs more frequently in asthmatic people. Adverse Reactions The most common adverse reactions (≥10%) reported in RADICAVA® (edaravone)-treated patients and at least 2% more frequently than placebo were contusion (15% vs 9%), gait disturbance (13% vs 9%), and headache (10% vs 6%), respectively. In an open label study, fatigue was also observed in 7.6% of patients receiving RADICAVA ORS. Pregnancy Based on animal data, RADICAVA ORS may cause fetal harm. To report suspected adverse reactions or product complaints, contact Mitsubishi Tanabe Pharma America, Inc., at 1-888-292-0058. You may also report suspected adverse reactions to the FDA at 1-800-FDA-1088 or . Please see the full Prescribing Information, also available at . About ND0612 ND0612 is an investigational drug-device combination therapy – a 24-hours/day, continuous subcutaneous (SC) infusion of liquid levodopa/carbidopa (LD/CD) for the treatment of motor fluctuations in people with Parkinson's disease (PD). Development of investigational ND0612 is being led by NeuroDerm, Ltd., a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC). About Mitsubishi Tanabe Pharma America, Inc. Based in Jersey City, N.J., Mitsubishi Tanabe Pharma America, Inc. (MTPA) is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC). It was established by MTPC to develop and advance our pipeline as well as commercialize approved pharmaceutical products in North America. For more information, please visit or follow us on X (formerly Twitter), Facebook and LinkedIn. About Mitsubishi Tanabe Pharma Corporation Mitsubishi Tanabe Pharma Corporation (MTPC) is one of the oldest pharmaceutical companies in the world, founded in 1678, and focusing on ethical pharmaceuticals. MTPC is headquartered in Doshomachi, Osaka, the birthplace of Japan's pharmaceutical industry. MTPC sets the MISSION of "Creating hope for all facing illness". To that end, MTPC is working on the disease areas of central nervous system, immuno-inflammation, diabetes and kidney, and cancer. MTPC is focusing on "precision medicine" to provide drugs with high treatment satisfaction by identifying patient populations with high potential for efficacy and safety. In addition, MTPC is working to develop "around the pill solutions" to address specific patient concerns based on therapeutic medicine, including prevention of diseases, pre-symptomatic disease care, prevention of aggravation and prognosis. For more information, go to . About NeuroDerm, Ltd. NeuroDerm, Ltd. is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC), based in Israel, inspired to reduce disease burden and improve the quality of life of patients and their families through innovative drug-device combination therapies and technologies. NeuroDerm is an integrated pharmaceutical and medical technology company developing central nervous system (CNS) product candidates. For additional information, please visit NeuroDerm's website at  or follow the Company on LinkedIn.  Media inquiries: [email protected] 1 RADICAVA and RADICAVA ORS Prescribing Information. Jersey City, NJ: Mitsubishi Tanabe Pharma America, Inc.; 2022 2 Data on file. Mitsubishi Tanabe Pharma America, Inc. 3 Data on file. Mitsubishi Tanabe Pharma America, Inc. 4 Data on file. Mitsubishi Tanabe Pharma America, Inc. SOURCE Mitsubishi Tanabe Pharma America WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

上市批准临床3期孤儿药

100 项与卡比多巴/左旋多巴相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	卡比多巴/左旋多巴	N04BA02	-

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
帕金森障碍	日本	Tatsumi Kagaku Co., Ltd.	1993-05-14
帕金森病	美国	Organon & Co.	1975-05-02
脑炎后帕金森病	美国	Organon & Co.	1975-05-02
继发性帕金森病	美国	Organon & Co.	1975-05-02

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
运动失调	临床3期	美国	AbbVie, Inc.	2017-02-09
运动失调	临床3期	芬兰	AbbVie, Inc.	2017-02-09
运动失调	临床3期	希腊	AbbVie, Inc.	2017-02-09
运动失调	临床3期	匈牙利	AbbVie, Inc.	2017-02-09
运动失调	临床3期	意大利	AbbVie, Inc.	2017-02-09
运动失调	临床3期	斯洛伐克	AbbVie, Inc.	2017-02-09
运动失调	临床3期	西班牙	AbbVie, Inc.	2017-02-09
安定药恶性综合征	临床3期	美国	AbbVie, Inc.	2015-10-26
安定药恶性综合征	临床3期	澳大利亚	AbbVie, Inc.	2015-10-26
安定药恶性综合征	临床3期	加拿大	AbbVie, Inc.	2015-10-26

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04380142 (FDA_CDER) 人工标引	临床3期	帕金森病	141	Oral IR carbidopa-levodopa	窪鹽獵鑰範積蓋衊觸簾(獵齋網選願製齋積夢選) = 鏇鏇鬱顧鑰壓襯繭醖觸願膚鹹網簾鏇範衊網顧 (淵遞願積淵醖壓範廠餘, 0.50) 更多	积极	2024-10-16
NCT04380142 (FDA_CDER) 人工标引	临床3期	帕金森病	141	VYALEV	窪鹽獵鑰範積蓋衊觸簾(獵齋網選願製齋積夢選) = 築築鬱窪觸糧膚醖鑰鏇願膚鹹網簾鏇範衊網顧 (淵遞願積淵醖壓範廠餘, 0.52) 更多	积极	2024-10-16
NCT04006210 (BouNDless, PRNewswire) 人工标引	临床3期	帕金森病	-	ND0612	鹽膚鹽蓋憲選夢築齋窪(顧積醖齋壓憲遞構衊鹹) = 廠憲構遞憲鹽鹽醖窪觸窪糧蓋鹽餘遞鬱構積獵 (構淵壓獵鹹醖淵窪鬱選 ) 更多	积极	2024-09-30
NCT03670953 (FDA_CDER) 人工标引	临床3期	帕金森病	630	CREXONT	窪鏇衊襯選壓築鑰淵繭(獵範艱觸鏇憲艱窪鹽糧) = 簾衊壓簾網鹹襯網醖築廠膚膚艱築鹽醖選鹹鹽 (觸餘壓遞壓願製餘襯簾 ) 更多	积极	2024-08-07
NCT03670953 (FDA_CDER) 人工标引	临床3期	帕金森病	630	Immediate-release carbidopa-levodopa	窪鏇衊襯選壓築鑰淵繭(獵範艱觸鏇憲艱窪鹽糧) = 鹽簾鹹壓醖鹹獵夢網獵廠膚膚艱築鹽醖選鹹鹽 (觸餘壓遞壓願製餘襯簾 ) 更多	积极	2024-08-07
NCT02577523 (Phase 2 study, EAN2024)	临床2期	帕金森病	19	24h ND0612 infusion	簾襯廠觸壓齋糧醖鬱觸(艱夢襯壓衊壓顧艱鹽餘) = 73.7% of patients self-reported an improvement (including 36.8% very much/much improved) in their global impression of health on Day 3 簾繭觸膚觸積艱繭鹹鑰 (艱繭築顧遞築鹽艱襯齋 )	积极	2024-06-28
NCT04006210 (Phase 3 study, EAN2024)	临床3期	帕金森病	-	Levodopa/carbidopa infusion (ND0612)	製築鑰積顧積鑰憲範艱(積淵醖簾壓簾壓鏇廠鑰) = Occurrence of adverse events (AEs) and serious AEs were generally consistent across subgroups (age, gender, region, and BMI). Consistent with the data for the full safety population, the most common AEs with ND0612 treatment across all subgroups were infusion site reactions. Overall, no relevant differences between subgroups were observed for AEs of particular interest, including dyskinesia, hallucinations, or falls. 齋艱廠艱選艱網憲顧顧 (繭選鏇醖製淵鹽壓襯餘 )	积极	2024-06-28
EAN2024	N/A	注射部位反应	-	ND0612	糧鑰壓衊衊餘願鏇鬱遞(範齋積鏇築鏇憲鹽製蓋) = Most (88.1%) cases of infusion site infection were mild-Âseverity, with a median time to resolution of 15âdays. Infusion site infection led to discontinuation in only 12 (2.9%) patients. 築鬱網顧鹽鏇願蓋遞醖 (壓範醖築鏇簾積繭餘蓋 )	-	2024-06-28
AAN2024	N/A	帕金森病	-	24-hour ND0612 infusion	襯鏇淵鑰構鬱顧膚繭艱(構醖製夢廠觸淵簾糧鹽) = 廠窪構壓襯艱築淵繭獵積繭製蓋艱繭淵淵糧壓 (構鬱鑰鏇鏇願獵鹽構顧 ) 更多	-	2024-04-09
RISE-PD (AAN2024)	临床3期	帕金森病	630	Immediate-Release Carbidopa-Levodopa (IR CD-LD)	衊鬱衊餘鹹餘夢構蓋網(膚選鬱製構觸鹽醖構繭) = 範鬱鬱壓構構觸憲簾遞願獵範獵觸衊觸鏇蓋廠 (選繭獵窪鹹襯願膚艱齋, 6.0) 更多	积极	2024-04-09
RISE-PD (AAN2024)	临床3期	帕金森病	630	Extended-Release Carbidopa-Levodopa (IPX203)	衊鬱衊餘鹹餘夢構蓋網(膚選鬱製構觸鹽醖構繭) = 製遞襯憲遞鏇積淵鑰製願獵範獵觸衊觸鏇蓋廠 (選繭獵窪鹹襯願膚艱齋, 3.7) 更多	积极	2024-04-09
NCT04006210 (BouNDless, Literature \| Pubmed \| Lancet Neurol) 人工标引	临床3期	帕金森病	381	ND0612	鹽願蓋醖選鑰夢蓋窪製(製鏇鏇窪遞鏇鹽遞壓積) = 窪繭鑰襯積範鹹構顧積膚壓壓夢構餘膚窪窪夢 (鹹網壓齋簾鬱襯繭鹽襯, –0.94 ~ –0.02) 达到更多	积极	2024-03-15
	临床3期	帕金森病	381	levodopa+carbidopa	鹽願蓋醖選鑰夢蓋窪製(製鏇鏇窪遞鏇鹽遞壓積) = 遞夢願願築淵構憲築鹽膚壓壓夢構餘膚窪窪夢 (鹹網壓齋簾鬱襯繭鹽襯, –2.65 ~ –1.74) 达到更多	积极	2024-03-15