Ensayo clínico piloto, unicéntrico, controlado con placebo, a doble-ciego, aleatorio y cruzado con alfa-1-antitripsina humana en sujetos con fibromialgia.A single-centre, randomised, double-blind, controlled with placebo, cross-over, pilot clinical trial with human alpha-1-antitrypsin in subjects with fibromyalgia.

开始日期2006-08-28

申办/合作机构

Instituto Grifols SA

100 项与 Alpha-antitrypsin(PPL Therapeutics Plc) 相关的临床结果

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100 项与 Alpha-antitrypsin(PPL Therapeutics Plc) 相关的转化医学

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100 项与 Alpha-antitrypsin(PPL Therapeutics Plc) 相关的专利（医药）

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474

项与 Alpha-antitrypsin(PPL Therapeutics Plc) 相关的文献（医药）

2025-06-01·JOURNAL OF CONTROLLED RELEASE

Impact of administration route and PEGylation on alpha-1 antitrypsin augmentation therapy

Article

作者: Liu, Xiao ; Vanbever, Rita ; Ucakar, Bernard ; Marbaix, Etienne ; Vanvarenberg, Kevin

Patients suffering from emphysema associated with alpha-1 antitrypsin (AAT) deficiency can benefit from augmentation therapy. AAT is administered to the patient once a week via intravenous infusion by a healthcare professional. However, only 2 % of the AAT dose reaches the lungs following intravenous infusion. Inhalation of AAT might be a convenient and effective alternative to intravenous infusion. Yet, it has shown limited therapeutic efficacy in a recent clinical trial. Here, we assessed the impact of these routes of AAT administration on AAT pharmacokinetics, lung distribution and therapeutic efficacy in mice. PEGylation of the serpin was employed to improve its therapeutic value. Intravenous injection of AAT or its local administration to the lungs resulted in a similar exposure of AAT in the lung parenchyma with however an AAT dose delivered to the lungs 45 times lower than the injected dose. Conjugation of AAT to a 2-armed 40 kDa polyethylene glycol (PEG) chain prolonged its half-life in plasma and lungs by 1.6-times, decreased its penetration in the lung tissue by both routes of administration but did not markedly affect the lung exposure to AAT. The PEG moiety in PEG-AAT was cleared more slowly than the protein moiety and high PEG quantities remained in the lung tissue and alveolar macrophages several days after intratracheal instillation. Pulmonary administration and PEGylation both improved AAT efficacy to prevent lung injury and inflammation in a murine model of chronic obstructive pulmonary disease where lung inflammation was induced by delivering porcine pancreatic elastase and lipopolysaccharide locally to the airways. Anti-AAT and anti-PEG antibodies were generated by AAT and PEG-AAT administration, as expected for a foreign protein. However, anti-PEG antibodies did not significantly contribute to the overall anti-drug antibody titers against the conjugate. AAT and PEG-AAT showed good stability to jet nebulization. This study provides new insights into the impact of administration route and PEGylation on lung exposure, clearance, therapeutic efficacy, and safety of AAT. It highlights that inhalation of AAT might effectively replace its intravenous infusion in augmentation therapy.

2025-04-01·JOURNAL OF ALZHEIMERS DISEASE

Cost-effectiveness of diagnosing and treating patients with early Alzheimer's disease with anti-amyloid treatment in a clinical setting

Article

作者: Jelle Visser, Pieter ; Vromen, Ellen ; Fladby, Tormod ; Wimo, Anders ; Emersic, Andreja ; Gonzalez-Ortiz, Fernando ; Sköldunger, Anders ; Kirsebom, Bjørn-Eivind ; Gregoric Kramberger, Milica ; Speh, Andreja ; Winblad, Bengt ; Blennow, Kaj ; Selnes, Per ; Handels, Ron ; Timón-Reina, Santiago ; Bon, Jaka

Background:

The introduction of anti-amyloid treatments (AAT) for Alzheimer's disease (AD) has put the cost-effectiveness into focus.

Objective:

Estimate the potential cost-effectiveness of diagnostic pathways combined with AAT for early AD.

Methods:

Diagnostic accuracy of blood-based (BBM) and cerebrospinal fluid (CSF) biomarkers was obtained from Norwegian memory clinics using positron emission tomography (PET) as reference standard. In a health-economic model, the cost-effectiveness of three diagnostic strategies was estimated relying either on BBM (p-tau 217), CSF (Aβ 42/40 ratio), and BBM with CSF confirmatory testing and compared with standard of care (SoC) and compared with CSF-AAT. The model consisted of a decision tree reflecting the diagnostic process and a subsequent Markov cohort model starting at mild cognitive impairment due to AD. All strategies except SoC were combined with AAT including costs of treatment (assumed €5000/year), infusions and monitoring.

Results:

Compared with SoC all three strategies (CSF-AAT, BBM-AAT, and BBM-CSF-AAT) resulted in QALY gains at higher costs, with an incremental cost-effectiveness ratio (ICER) of 110k€, 141k€ and 110k€ respectively. Compared with CSF-AAT both BBM-AAT and BBM-CSF-AAT strategies resulted in QALYs lost at lower costs, with an ICER of 27k€ and 109k€ respectively. Results were particularly sensitive to the price of AAT and possible subcutaneous administration.

Conclusions:

Compared with SoC all three strategies are potentially not cost-effective as they exceeded the Swedish maximum willingness to pay threshold of €94,800 per QALY gained. BBM-CSF-AAT versus CSF-AAT is potentially cost-effective if willing to accept its QALY loss. Discussions on budget impact on different payers are needed after introducing AAT.

2025-03-01·EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY

Quantitative evaluation of the efficacy and safety of first-line systemic therapies for advanced hepatocellular carcinoma

Article

作者: Wang, Xinrui ; Cai, Ruifen ; Zheng, Qingshan ; Wu, Lijuan ; Huang, Jihan ; Li, Lujin ; Liu, Yixiao

OBJECTIVES:

This study aimed to quantitatively evaluate the efficacy and safety of first-line systemic therapies for treating advanced hepatocellular carcinoma (aHCC).

METHODS:

The study included clinical trials of first-line systemic therapies for aHCC since the approval of sorafenib in 2007. Hazard function models were used to describe changes in overall survival (OS) and progression-free survival (PFS) over time. Monte Carlo simulation was used to compare OS and PFS for different treatments, including sorafenib, antiangiogenic therapies (AATs) (except sorafenib), immune checkpoint inhibitor (ICI) monotherapy, AAT + targeted therapy, AAT + chemotherapy, AAT + ICIs, and ICIs + ICIs. Furthermore, the objective response rate (ORR) and incidence of grade ≥ 3 adverse events were analyzed.

RESULTS:

Fifty studies comprising 12,918 participants were included. AAT + ICIs demonstrated a significant benefit in median OS (mOS), median PFS (mPFS), and ORR (20.5 [95% CI 17.5-24] months, 7.5 [95% CI 6.5-8.8] months, and 24% [95% CI 17%-30%], respectively). ICIs + ICIs and ICI monotherapy ranked second and third, respectively with an mOS of 20 (95% CI 18.5-21.5) months and 14.5 (95% CI 13.5-16) months, respectively. The OS, PFS, and ORR of patients treated with AAT, AAT + targeted therapy, and AAT + chemotherapy were similar to those of patients treated with sorafenib. A higher proportion of patients with Barcelona Clinic Liver Cancer (BCLC) stage C had a shorter OS. OS was associated with publication year, and PFS was associated with the proportion of patients with BCLC stage C. The incidence of grade ≥ 3 adverse events in the ICIs and ICIs + ICIs treatment groups was low.

CONCLUSIONS:

The study results provide valuable information from which to base rational clinical drug use and serves as a reliable external control for evaluating new treatments for aHCC.

项与 Alpha-antitrypsin(PPL Therapeutics Plc) 相关的新闻（医药）

2024-10-18

·药明康德

里程碑！全球首次，RNA编辑疗法获临床概念验证

Wave Life Sciences今天宣布其进行中的临床1b/2a期RestorAATion-2研究获得积极结果。该公司旗下在研RNA编辑候选疗法WVE-006能够成功编辑α-1抗胰蛋白酶缺乏症（AATD）患者的基因，完成临床机制验证。Wave预计将在2025年分享多次给药数据。根据新闻稿，WVE-006是首个进入临床开发的RNA编辑疗法，而今天的试验结果是RNA编辑疗法在人体当中完成首次机制验证。 AATD是一种可影响肺和肝脏功能的遗传疾病，由于缺乏具有正常功能的α-1抗胰蛋白酶（AAT），新生儿可能出现肝炎综合征，成年人则可能出现肝纤维化，甚至发展为肝硬化和肝细胞癌。该疾病的主要病理特点是突变型AAT蛋白的积累，这些突变的蛋白会损害肝脏和肺部。在欧美国家和地区大约有20万名患者在两个SERPINA1等位基因上都有Z突变（即Pi*ZZ纯合子基因型）。目前AATD肺病的唯一治疗选择是通过每周静脉输注以递送AAT蛋白。AATD肝病目前尚无治疗方法，许多患者最终需要接受肝脏移植。此次公布的数据来自RestorAATion-2试验中的首个单剂量组（200毫克），包括前两名Pi*ZZ型AATD患者，从试验开始至第57天的结果。由于Pi*ZZ型AATD患者不会自然产生野生型α-1抗胰蛋白酶（M-AAT）蛋白，因此，若在患者体内检测到M-AAT蛋白，便可确认突变型Z-AAT的mRNA成功编辑。此外，若患者恢复至50%的M-AAT蛋白水平，将与“MZ”杂合子基因型相符，具较低的AATD相关肺病和肝病风险。分析显示，患者血浆中的野生型M-AAT蛋白在第15天达到平均6.9微摩尔，占总AAT蛋白的60%以上。与体内产生功能性M-AAT蛋白的结果一致，患者体内中性粒细胞弹性蛋白酶的抑制与基线相较增加。基线时未能检测到患者体内的AAT蛋白，而在试验第15天时，患者的平均总AAT蛋白水平达到10.8微摩尔，达到了监管机构批准AAT增强疗法的标准。此外，早在试验第3天，患者的总AAT和M-AAT蛋白水平即较基线增加，并持续到第57天。目前，WVE-006展现出良好的耐受性和安全性。在RestorAATion-2试验及健康志愿者参与的RestorAATion-1试验中，所有不良事件均为轻度至中度，且未报告任何严重不良事件。 WVE-006是一款通过PN化学修饰和GalNAc偶联，以皮下注射方式给药的潜在“first-in-class”RNA编辑寡核苷酸疗法，由Wave寡核苷酸化学平台开发。该平台具备将腺嘌呤编辑为肌苷（A至I）的能力（即AIMer）。WVE-006的设计旨在修复SERPINA1等位基因编码mRNA中的单碱基突变，从而恢复血液中的功能性野生型AAT蛋白，同时减少异常Z-AAT蛋白的存在，以潜在治疗AATD相关的肺病和/或肝病。Wave公司在2022年与GSK达成了一项为期四年的研发合作协议，授予GSK WVE-006的全球独家许可。在Wave完成RestorAATion-2研究后，开发和商业化责任将由GSK接手。 Wave还在通过该公司的"edit-verse"平台，针对多个关键医疗需求积极推进一系列RNA编辑疗法。该平台结合了遗传数据集和先进的深度学习模型，以识别和靶向新的RNA编辑靶点。而除了Wave，于2020年成立的Ascidian Therapeutics也专注于RNA编辑疗法的开发。该公司的RNA外显子编辑平台结合了先进计算机生物学与高通量分子生物学筛选技术，可设计出一种创新的RNA分子。该公司进展最快的研发项目是针对Stargardt病和其他ABCA4相关视网膜病的在研疗法ACDN-01，该疗法的IND申请在今年1月获得美国FDA的许可，并被授予快速通道资格。新闻稿指出，ACDN-01是首个获美国FDA许可进入临床试验的RNA外显子编辑疗法，并且是首款专门针对Stargardt病遗传起源的临床期治疗方法。该公司同时在今年6月与罗氏（Roche）达成一项总额高达约18亿美元的研究合作与许可协议，将共同发现和开发针对神经疾病的RNA外显子编辑疗法。此外，AIRNA公司也在今年7月成功完成6000万美元融资。AIRNA通过其RESTORE+平台开发药物，该平台优化寡核苷酸序列、化学和递送，以实现精确、高效和安全的RNA编辑。该公司的首个候选药物是一种潜在“best-in-class”的AATD疗法。而Amber Bio公司则在去年8月走出隐匿模式，并获得超额认购的2600万美元种子轮融资。根据公司官网，Amber Bio的基于人工智能专有平台具潜力对数千个不同突变进行RNA编辑，可以重写长达数千碱基对的RNA片段，扩大治疗疾病的范围。与现有基因编辑疗法每次仅针对单一突变进行治疗不同的是，Amber Bio的候选药物原则上可以靶向同一个基因的几千个突变，因此具治疗因多种突变所引起疾病患者的潜力。同月，ADARx Pharmaceuticals也宣布成功完成超额认购的2亿美元C轮融资。ADARx正在开发一种不受限于治疗模式的RNA靶向平台，包括用于抑制、降解和编辑寡核苷酸的技术平台，以及新型寡核苷酸递送技术。参考资料： [1] Wave Life Sciences Announces First-Ever Therapeutic RNA Editing in Humans Achieved in RestorAATion-2 Trial of WVE-006 in Alpha-1 Antitrypsin Deficiency. Retrieved October 16, 2024 from https://www.globenewswire.com/news-release/2024/10/16/2964053/0/en/Wave-Life-Sciences-Announces-First-Ever-Therapeutic-RNA-Editing-in-Humans-Achieved-in-RestorAATion-2-Trial-of-WVE-006-in-Alpha-1-Antitrypsin-Deficiency.html 内容来源于网络，如有侵权，请联系删除。

信使RNA寡核苷酸临床结果基因疗法

2023-05-01

·PRNewswire

Significant Positive Shift in the Glioma Market with 13% CAGR During the Study Period (2019-2032) Expected by Analysts at DelveInsight

The dynamics of the glioma market are anticipated to change in the coming years owing to the positive outcomes of the emerging pipeline candidates during the developmental stage by key players, such as Bayer, VBL Therapeutics, Candel Therapeutics, AstraZeneca, DNAtrix, DelMar Pharmaceuticals, Chimerix, KaryoPharma, VBI Vaccines, Kazia Therapeutics, and others LAS VEGAS, May 1, 2023 /PRNewswire/ -- DelveInsight's Glioma Market Insights report includes a comprehensive understanding of current treatment practices, glioma emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan]. Key Takeaways from the Glioma Market Report As per DelveInsight analysis, the glioma market size in the 7MM was approximately USD 1 billion in 2022. According to the assessment done by DelveInsight, the estimated total incident glioma cases in the 7MM were approximately 47K in 2022. Leading glioma companies such as Bayer, Chimerix, Aivita Biomedical, Denovo Biopharma, Northwest Therapeutics, VBL Therapeutics, Laminar Pharmaceuticals, MedImmune, DNAtrix, Immunomic Therapeutics, Imvax, MimiVax, CNS Pharmaceuticals, Epitopoietic Research Corporation (ERC), Istari Oncology, SonALAsense, Kintara Therapeutics, Bristol Myers Squibb, Medicenna Therapeutics, BioMimetix, Eisai and Merck Sharp & Dohme, Kazia Therapeutics, Oblato, Genenta Science, Enterome, Inovio Pharmaceuticals, Karyopharm Therapeutics, VBI Vaccines, TME Pharma, Day One Biopharmaceuticals, Servier, Orbus Therapeutics, TVAX Biomedical, AnHeart Therapeutics, Beigene, SpringWorks Therapeutics, Forma Therapeutics, Hoffmann-La Roche, Incyte Corporation, Eli Lilly, and others are developing novel glioma drugs that can be available in the glioma market in the coming years. The promising glioma therapies in the pipeline include Regorafenib, ONC201, AV-GBM-1, Enzastaurin (DB-102), DCVax-L, Ofranergene Obadenovec (VB-111), LAM561 (2-OHOA), MEDI4736 (durvalumab), Tasadenoturev (DNX-2401), ITI-1000 (pp65 DC Vaccine), IGV-001, SurVaxM, Berubicin, GLIOVAC (Sitoiganap), Lerapolturev + Pembrolizumab, SONALA-001 + Exablate 2.0 Device, VAL-083 (dianhydrogalactitol), Pomalidomide, MDNA55, BMX-001, Lenvatinib ± Pembrolizumab, Paxalisib (GDC-0084), OKN-007, Temferon, EO2401, INO-5401+ INO-9012+ LIBTAYO (cemiplimab), Selinexor (KPT-330), Olutasidenib (FT-2102), VBI-1901, NOX-A12 (Olaptesed Pegol), Tovorafenib (DAY101), Vorasidenib (AG-881), Eflornithine, TVI-Brain-1, AB-218, BGB-290, Mirdametinib, FT-2102 (Olutasidenib), Vinblastine + Bevacizumab, Cobimetinib, PEMAZYRE (Pemigatinib), VERZENIO (abemaciclib) , and others. In December 2022, Chimerix announced the successful launch of the ONC201 Phase III ACTION study in patients with H3 K27M-mutant glioma. In June 2022, Day One Biopharmaceuticals announced positive initial data from its pivotal Phase II (FIREFLY-1) study with tovorafenib (DAY101) in relapsed pediatric low-grade glioma (pLGG). Discover which therapies are expected to grab the major glioma market share @ Glioma Market Report Glioma Overview Glioma is the most common kind of glial cell tumor in the central nervous system (CNS). Gliomas are infiltrative tumors that affect the brain tissue surrounding them. Glioblastoma is the most hazardous type of brain tumor, whereas pilocytic astrocytomas are the least dangerous. Previously, diffuse gliomas were classified into subgroups and grades depending on histopathologies, such as diffuse astrocytomas, oligodendrogliomas, or mixed gliomas/oligoastrocytomas. The specific etiology of glioma, like other primary brain tumors, remains unknown. However, certain variables may increase the risk of getting a brain tumor. Glioma symptoms vary depending on tumor type, size, location, and growth rate. Some common glioma symptoms include headache, nausea or vomiting, memory loss, urine incontinence, speech problems, and others. A medical history and a physical examination are used for glioma diagnosis. Glioma Epidemiology Segmentation DelveInsight estimates that there were approximately 47K incident cases of glioma in the 7MM in 2022. As per our analysis, the United States contributed to the largest incident population of glioma, acquiring ~40% of the 7MM in 2022. The glioma market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into: Total glioma incident cases Glioma grade-specific cases Glioma age-specific cases Glioma type-specific cases Download the report to understand which factors are driving glioma epidemiology trends @ Glioma Epidemiological Insights Glioma Treatment Market Glioma treatment is based on the type of glioma, its size and location, and the patient's unique characteristics. Chemotherapy and radiation therapy will be administered after surgery, particularly in individuals when the tumor cannot be removed entirely because it has invaded critical parts of the brain or is inaccessible. The typical glioma treatment protocol comprises surgery, chemotherapy, and radiation. Chemotherapy may include carmustine (BCNU), lomustine (CCNU), or gleostine (generic), cisplatin, carboplatin, etoposide, and irinotecan in case of low-grade glioma whereas temozolomide (TEMODAR) is the standard chemotherapy used in case of high-grade glioma majorly, Gliadel wafer implants (biodegradable discs injected with BCNU) are usually given as adjunctive treatment after surgery, although usage of Gliadel wafers remains a point of controversy due to its questionable survival benefits. Avastin (Genentech) and Temodar/Temodal (Merck), among others, are approved medications for HGG. However, due to patent expiration, the generic version of Temodar and biosimilars of Avastin are available on the glioma market. As generics and biosimilars are more cost-effective for patients than new medicines, intense competition exists among them. Furthermore, due to a lack of knowledge concerning bevacizumab's overall survival benefit, it is not recommended in unselected patients with newly diagnosed GBM. Currently, only one medication for LGG is approved, Tafinlar (dabrafenib) plus Mekinist (trametinib). Moreover, the current therapeutic market lacks a viable method for curing glioblastoma; therefore, the survival rate of people diagnosed with glioma remains poor. Glioma is incurable, and treatment choices are limited. Furthermore, the tumor has a high recurrence rate and a poor patient prognosis. In addition, there is currently no approved therapy for the unmethylated MGMT patient pool. To know more about glioma treatment guidelines, visit @ Glioma Management Glioma Therapies and Key Companies Regorafenib: Bayer ONC201: Chimerix AV-GBM-1: Aivita Biomedical Enzastaurin (DB-102): Denovo Biopharma DCVax-L: Northwest Therapeutics Ofranergene Obadenovec (VB-111): VBL Therapeutics LAM561 (2-OHOA): Laminar Pharmaceuticals MEDI4736 (durvalumab): MedImmune Tasadenoturev (DNX-2401): DNAtrix ITI-1000 (pp65 DC Vaccine): Immunomic Therapeutics IGV-001: Imvax SurVaxM: MimiVax Berubicin: CNS Pharmaceuticals GLIOVAC (Sitoiganap): Epitopoietic Research Corporation (ERC) Lerapolturev + Pembrolizumab: Istari Oncology SONALA-001 + Exablate 2.0 Device: SonALAsense VAL-083 (dianhydrogalactitol): Kintara Therapeutics Pomalidomide: Bristol Myers Squibb MDNA55: Medicenna Therapeutics BMX-001: BioMimetix Lenvatinib ± Pembrolizumab: Eisai and Merck Sharp & Dohme Paxalisib (GDC-0084): Kazia Therapeutics OKN-007: Oblato Temferon: Genenta Science EO2401: Enterome INO-5401+ INO-9012+ LIBTAYO (cemiplimab): Inovio Pharmaceuticals Selinexor (KPT-330): Karyopharm Therapeutics VBI-1901: VBI Vaccines NOX-A12 (Olaptesed Pegol): TME Pharma Tovorafenib (DAY101): Day One Biopharmaceuticals Vorasidenib (AG-881): Servier Eflornithine: Orbus Therapeutics TVI-Brain-1: TVAX Biomedical AB-218: AnHeart Therapeutics BGB-290: Beigene Mirdametinib: SpringWorks Therapeutics FT-2102 (Olutasidenib): Forma Therapeutics Vinblastine + Bevacizumab: Hoffmann-La Roche Cobimetinib: Hoffmann-La Roche PEMAZYRE (Pemigatinib): Incyte Corporation VERZENIO (abemaciclib): Eli Lilly Learn more about the FDA-approved drugs for glioma @ Drugs for Glioma Treatment Glioma Market Dynamics Glioma market growth can be ascribed to various factors, including an aging population, evolving research, better diagnostic tests, developing surgical procedures, improved radiotherapy techniques, and novel systemic medicines. Commercially, there is a greater emphasis on high-grade glioma than low-grade glioma because patients with low-grade glioma often live longer than those with high-grade glioma. As a result, just a few therapies are in late-stage studies, while the majority are in early-stage trials. In the case of GBM, on the other hand, the pipeline is robust, with numerous potential therapies in late and mid-stage research that have yet to be marketed. The pipeline includes drugs with varied modes of action and administration routes. It is interesting to note that the emerging market of glioma includes budding gene therapy, i.e., ofranergene obadenovec (VB-111) by VBL Therapeutics, followed by four vaccine/immunotherapy candidates such as VBI-1901, AV-GBM-1 and ITI-1000 (pp65 DC Vaccine), tasadenoturev (DNX-2401) by VBI Vaccines, Aivita Biomedical, Immunomic Therapeutics, and DNAtrix, respectively, and these therapies are going to alter the glioma market dynamics in the upcoming years. The current glioma market has been categorized based on the dominant treatment pattern across the 7MM, which shows slight variances in the overall prescription pattern. The forecast model considers surgery, chemotherapy/radiation therapy, bevacizumab + radiotherapy/chemotherapy, and OPTUNE with temozolomide. The anticipated launch of new therapies and improved integration of early patient screening, medication in secondary care and other clinical settings, research on optimum implementation methods, and increased awareness may eventually facilitate the development of successful treatment alternatives. However, there are a few challenges to the timely identification and treatment of these patients, such as a lack of a reliable biomarker that can aid in diagnosis and patient classification and identifying recurrence and signaling medication response. Furthermore, the failure rate of high-phased drug trials is significant, making it impossible to predict trial success, exacerbating the need for medicines that could assist ease the patient segment with this severe kind of cancer. Scope of the Glioma Market Report Therapeutic Assessment: Glioma current marketed and emerging therapies Glioma Market Dynamics: Conjoint Analysis of Emerging Glioma Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's Views, Analyst's Views, Glioma Market Access and Reimbursement Discover more about glioma drugs in development @ Glioma Clinical Trials Table to Contents Related Reports Glioma Epidemiology Forecast Glioma Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted glioma epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Glioma Pipeline Glioma Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key glioma companies, including PTC Therapeutics, Ono Pharmaceuticals, Philogen, SpringWorks Therapeutics, Xennials Therapeutics, AnHeart Therapeutics, Istari Oncology, among others. High-Grade Glioma Pipeline High-Grade Glioma Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key high-grade glioma companies, including PTC Therapeutics, Aadi, Lee's Pharmaceutical, among others. Malignant Glioma Pipeline Malignant Glioma Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key malignant glioma companies, including Bexion Pharmaceuticals, Inc., Lee's Pharmaceutical Limited, Merck & Co, Oblato, Inc., OncoSynergy, Inc, among others. Recurrent Malignant Glioma Pipeline Recurrent Malignant Glioma Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key recurrent malignant glioma companies, including Bexion Pharmaceuticals, Inc., Merck, Oblato, OncoSynergy, Inc, among others. Glioblastoma Pipeline Glioblastoma Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key glioblastoma companies, including AstraZeneca, BioNTech, Merck, Pfizer, Roche, among others. Other Trending Reports Tay-sachs Disease Or Gm2 Gangliosidosis Market | Onycholysis Market | Diagnostic Imaging Equipment Market | Chemotherapy-induced Peripheral Neuropathy Market | Global Electrophysiology Devices Market | Anaphylaxis Market | Atherectomy Devices Market | Helicobacter Pylori Infections Market | Ophthalmic Imaging Equipment Market | Androgenetic Alopecia Market | Allergic Rhinitis Market | Chronic Inflammatory Demyelinating Polyneuropathy Market | Chronic Inflammatory Demyelinating Polyneuropathy Market | Colorectal Cancer Crc Market | Opioid Induced Constipation Market | Vertigo Market | Bone Anchored Hearing Systems Market | Wound Closure Devices Market | Hip Replacement Devices Market | Hemodynamic Monitoring Systems Market | Egfr Non-small Cell Lung Cancer Market | Helicobacter Pylori Infection Market | Hyperkalemia Market | Polycythemia Market Neurostimulation Devices Market | Carpal Tunnel Syndrome Market | Ventilator Market | Cerebral Aneurysm Market | Alpha Antitrypsin Market | Binge Eating Disorder Market | Bunion Market | Concussions Market Size | Exocrine Pancreatic Insufficiency Market | Healthcare Due Diligence Services | Minimal Residual Disease Market | Hypertrophic Scar Market | Lung Fibrosis Market | Anterior Uveitis Market | 22q11.2 deletion syndrome Market | X-Linked Retinitis Pigmentosa (XLRP) Market | Acute Radiation Syndrome Market | Alpha-1 Protease Inhibitor Deficiency Market | Androgenetic Alopecia Market | Hyperlipidemia Market | Cardiotoxicity Market | Hypertrophic Cardiomyopathy Market | Fatty Acid Oxidation Disorders (FAODs) Market | Androgen Insensitivity Syndrome Market | Emphysema Market | Canaloplasty Market | Dravet Syndrome Market | Celiac Disease Market | Chlamydia Infections Market | Syphilis Market | Renal Tubular Acidosis Market | Palmoplantar Pustulosis (PPP) Market | Aplastic Anemia Market | Bacterial Pneumonia Market | B cell Chronic Lymphocytic Leukemia Market | B cell Lymphomas Market | Behcets Disease Market Neoantigen-based Personalized Cancer therapeutic Vaccines Competitive Landscape and Market Forecast—by 2035 | Glioblastoma Market Related Healthcare Services Healthcare Consulting Healthcare Competitive Intelligence Services Healthcare Asset Prioritization Services 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 . Connect with us on LinkedIn |Facebook|Twitter Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

临床3期临床2期疫苗免疫疗法

2023-04-27

·PRNewswire

Chronic Kidney Disease Market to Observe Impressive Growth at a CAGR of 8.7% by 2032, Estimates DelveInsight

The expected launch of therapies by several pharma companies such as ProKidney, Reata Pharmaceuticals, Inc., Novo Nordisk A/S, Boehringer Ingelheim, Eli Lilly and Company, AstraZeneca, and others will lead to change in the chronic kidney disease market dynamic during the forecast period (2023-2032) LAS VEGAS, April 27, 2023 /PRNewswire/ -- DelveInsight's Chronic Kidney Disease Market Insights report includes a comprehensive understanding of current treatment practices, chronic kidney disease emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan]. Key Takeaways from the Chronic Kidney Disease Market Report As per DelveInsight analysis, the chronic kidney disease market size in the 7MM was approximately USD 5 billion in 2022. According to the assessment done by DelveInsight, the estimated total diagnosed prevalent chronic kidney disease cases in the 7MM were approximately 16 million in 2022. Leading chronic kidney disease companies such as ProKidney, Reata Pharmaceuticals, Inc., Novo Nordisk A/S, Boehringer Ingelheim, Eli Lilly and Company, KBP Biosciences, Kibow Pharma, Cincor Pharma, AstraZeneca, Allena Pharmaceuticals, DiaMedica Therapeutics Inc, Lexicon Pharmaceuticals, Sanofi, and others are developing novel chronic kidney disease drugs that can be available in the chronic kidney disease market in the coming years. The promising chronic kidney disease therapies in the pipeline include Renal Autologous Cell Therapy (REACT), Bardoxolone methyl, Ziltivekimab, Semaglutide, JARDIANCE (empagliflozin), KBP-5074, KT-301, CIN-107 (Baxdrostat), Zibotentan, ALLN-346, DM199, Sotagliflozin , and others. Discover which therapies are expected to grab the major chronic kidney disease market share @ Chronic Kidney Disease Market Report Chronic Kidney Disease Overview Chronic kidney disease occurs when the kidneys get damaged and cannot filter blood as efficiently as they should. Chronic kidney disease is defined as abnormalities in kidney structure or function that have been present for more than three months and have a negative impact on health. Chronic kidney disease is categorized into five phases based on the extent of kidney damage and function. In stage 1, there is modest kidney disease, and in stage 5, the kidneys have stopped working. The severity of chronic kidney disease varies. In the early stages of kidney disease, there are usually no chronic kidney disease symptoms. It can only be diagnosed if a blood or urine test is performed for another reason and the results show a kidney issue. Tiredness, swollen ankles, feet, or hands, shortness of breath, unwellness, and blood in the urine indicate a more advanced stage. Chronic kidney disease can be detected using blood and urine tests. The eGFR blood test measures the efficiency with which the kidneys function. Ultrasound, CT scan, X-ray, and MRI imaging examinations are also used for chronic kidney disease diagnosis. Chronic Kidney Disease Epidemiology Segmentation DelveInsight estimates that there were approximately 16 million diagnosed prevalent cases of chronic kidney disease in the 7MM in 2022. As per our analysis, chronic kidney disease has been identified as a female-dominant disease, with more females suffering than males. In 2022, 52% of cases of CKD were of females, while 48% of cases were of males in the 7MM. The chronic kidney disease market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into: Chronic Kidney Disease Prevalent Cases Chronic Kidney Disease Diagnosed Prevalent Cases Chronic Kidney Disease Gender-specific Diagnosed Prevalent Cases Chronic Kidney Disease Age-specific Diagnosed Prevalent cases Chronic Kidney Disease Complication-specific Diagnosed Prevalent cases Chronic Kidney Disease Stage-specific Diagnosed Prevalent Cases Chronic Kidney Disease Etiology-specific Diagnosed Prevalent Cases Download the report to understand which factors are driving chronic kidney disease epidemiology trends @ Chronic Kidney Disease Epidemiological Insights Chronic Kidney Disease Treatment Market There are various types of drugs in the chronic kidney disease market. Although available medications cannot cure CKD, they can treat problems and halt future kidney damage. DelveInsight's chronic kidney disease market projection focuses on pharmacological therapy (including all presently utilized off-label medications) recommended for chronic kidney disease management and excludes revenue produced by devices and surgical procedures, including dialysis. The chronic kidney disease drugs market is classified for the study based on drug class, such as erythropoietin stimulating agents (ESA), angiotensin-converting enzyme inhibitors, and angiotensin receptor blockers (ACE-I and ARBs), antidiabetics, secondary hyperparathyroidism treatment (SHPT), and urate-lowering therapies. There are also approved therapies for chronic kidney disease treatment, such as KERENDIA, INVOKANA, JYNARQUE, and FARXIGA. According to DelveInsight's analysis, antidiabetics and antihypertensives are the most widely utilized among the top prescribed pharmaceuticals. Given the health risks associated with renal dysfunction, DelveInsight analysts developed a comprehensive understanding of chronic kidney disease and its etiologies, defining the breadth of the therapeutic paradigm. The chronic kidney disease treatment market is classified into three segments: diabetic kidney disease (DKD), polycystic kidney disease (PKD), and hypertension induced kidney disease. To know more about chronic kidney disease treatment guidelines, visit @ Chronic Kidney Disease Management Chronic Kidney Disease Therapies and Key Companies Renal Autologous Cell Therapy (REACT): ProKidney Bardoxolone methyl: Reata Pharmaceuticals, Inc. Ziltivekimab: Novo Nordisk A/S Semaglutide: Novo Nordisk A/S JARDIANCE (empagliflozin): Boehringer Ingelheim/Eli Lilly and Company KBP-5074: KBP Biosciences KT-301: Kibow Pharma CIN-107 (Baxdrostat): Cincor Pharma Zibotentan: AstraZeneca ALLN-346: Allena Pharmaceuticals DM199: DiaMedica Therapeutics Inc Sotagliflozin: Lexicon Pharmaceuticals/Sanofi Learn more about the FDA-approved drugs for chronic kidney disease @ Drugs for Chronic Kidney Disease Treatment Chronic Kidney Disease Market Dynamics The dynamics of the chronic kidney disease market are expected to change in the coming years. The emerging treatment pipeline is robustly humongous, attributing to novel MoAs. Moreover, the rising data on chronic kidney disease and its risk factors is expected to add significantly to the patient pool. Furthermore, the current therapy paradigm includes FARXIGA (SGLT2 inhibitor), which is utilized for diabetic and non-diabetic chronic kidney disease patients. In the US, FARXIGA had a market size of USD 111 million in 2021 and is anticipated to reach USD 559 million as its peak revenue. Top SGLT2 players have been competing for lucrative approvals in recent years. The chronic kidney disease market may see another approval with Empagliflozin from Boehringer Ingelheim Pharmaceuticals, Inc by 2023. Empagliflozin has achieved landmark results in chronic kidney disease studies and would be a contender to FARXIGA because the findings could pave the road for a catch-up approval. Farxiga's sales have increased considerably as the label has expanded, but Jardiance is not far behind. According to the model, the therapy will produce a chronic kidney disease market size of USD 9 million by its launch year (2023), and this chronic kidney disease market size is predicted to grow to USD 772 million by 2032. However, several factors may impede the growth of the chronic kidney disease market, such as the undiagnosed, unreported cases, and unawareness about the disease may also impact future chronic kidney disease market growth. On the contrary, there are significant factors that may contribute to the growth in the market, as chronic kidney disease is associated with major complications, including anemia, hyperparathyroidism, and metabolic acidosis. Moreover, there is a scope for branded pharmacologic treatment with good safety and efficacy data and broader range coverage like impulse control. Scope of the Chronic Kidney Disease Market Report Therapeutic Assessment: Chronic Kidney Disease current marketed and emerging therapies Chronic Kidney Disease Market Dynamics: Attribute Analysis of Emerging Chronic Kidney Disease Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's Views, Analyst's Views, Chronic Kidney Disease Market Access and Reimbursement Discover more about chronic kidney disease drugs in development @ Chronic Kidney Disease Clinical Trials Table of Contents Related Reports Chronic Kidney Disease Epidemiology Forecast Chronic Kidney Disease Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted chronic kidney disease epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Chronic Kidney Disease Pipeline Chronic Kidney Disease Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key chronic kidney disease companies, including KBP Biosciences, Eli Lilly and Company, Novo Nordisk, Prokidney, Boryung Pharmaceutical, among others. Moderate and Severe Chronic Kidney Disease Market Moderate and Severe Chronic Kidney Disease Market Insights, Epidemiology, and Market Forecast – 2032 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 moderate and severe chronic kidney disease companies, including Kibow Pharma, Bayer, Roche, Caladrius Biosciences, UnicoCell Biomed, Pharmicell, Scohia Pharma, Unicycive Therapeutics, among others. Moderate and Severe Chronic Kidney Disease Pipeline Moderate and Severe Chronic Kidney Disease Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key moderate and severe chronic kidney disease companies, including Kibow Pharma, Bayer, Roche, Caladrius Biosciences, UnicoCell Biomed, Pharmicell, Scohia Pharma, Unicycive Therapeutics, among others. Anemia In Chronic Kidney Disease Market Anemia In Chronic Kidney Disease Market Insights, Epidemiology, and Market Forecast – 2032 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 anemia in chronic kidney disease companies, including Biocad, Xenetic Biosciences, Chiasma, Liminal BioSciences, among others. Anemia In Chronic Kidney Disease Pipeline Anemia In Chronic Kidney Disease Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key anemia in chronic kidney disease companies, including Biocad, Xenetic Biosciences, Chiasma, Liminal BioSciences, among others. Other Trending Reports Tay-sachs Disease Or Gm2 Gangliosidosis Market | Onycholysis Market | Diagnostic Imaging Equipment Market | Chemotherapy-induced Peripheral Neuropathy Market | Global Electrophysiology Devices Market | Anaphylaxis Market | Atherectomy Devices Market | Helicobacter Pylori Infections Market | Ophthalmic Imaging Equipment Market | Androgenetic Alopecia Market | Allergic Rhinitis Market | Chronic Inflammatory Demyelinating Polyneuropathy Market | Chronic Inflammatory Demyelinating Polyneuropathy Market | Colorectal Cancer Crc Market | Opioid Induced Constipation Market | Vertigo Market | Bone Anchored Hearing Systems Market | Wound Closure Devices Market | Hip Replacement Devices Market | Hemodynamic Monitoring Systems Market | Egfr Non-small Cell Lung Cancer Market | Helicobacter Pylori Infection Market | Hyperkalemia Market | Polycythemia Market Neurostimulation Devices Market | Carpal Tunnel Syndrome Market | Ventilator Market | Cerebral Aneurysm Market | Alpha Antitrypsin Market | Binge Eating Disorder Market | Bunion Market | Concussions Market Size | Exocrine Pancreatic Insufficiency Market | Healthcare Due Diligence Services | Minimal Residual Disease Market | Hypertrophic Scar Market | Lung Fibrosis Market | Anterior Uveitis Market | 22q11.2 deletion syndrome Market | X-Linked Retinitis Pigmentosa (XLRP) Market | Acute Radiation Syndrome Market | Alpha-1 Protease Inhibitor Deficiency Market | Androgenetic Alopecia Market | Hyperlipidemia Market | Cardiotoxicity Market | Hypertrophic Cardiomyopathy Market | Fatty Acid Oxidation Disorders (FAODs) Market | Androgen Insensitivity Syndrome Market | Emphysema Market | Canaloplasty Market | Dravet Syndrome Market | Celiac Disease Market | Chlamydia Infections Market | Syphilis Market | Renal Tubular Acidosis Market | Palmoplantar Pustulosis (PPP) Market | Aplastic Anemia Market | Bacterial Pneumonia Market | B cell Chronic Lymphocytic Leukemia Market | B cell Lymphomas Market | Behcets Disease Market Neoantigen-based Personalized Cancer therapeutic Vaccines Competitive Landscape and Market Forecast—by 2035 | Glioblastoma Market Related Healthcare Services Healthcare Consulting Healthcare Competitive Intelligence Services Healthcare Asset Prioritization Services 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 . Connect with us on LinkedIn |Facebook|Twitter Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

上市批准

100 项与 Alpha-antitrypsin(PPL Therapeutics Plc) 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB05481

研发状态

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
α1-抗胰蛋白酶缺乏症	临床2期	-	PPL Therapeutics Plc	-
α1-抗胰蛋白酶缺乏症	临床2期	-	Bayer AG	-
囊性纤维化	临床2期	-	Bayer AG	-
囊性纤维化	临床2期	-	PPL Therapeutics Plc	-
成人呼吸窘迫综合征	临床2期	-	Bayer AG	-
成人呼吸窘迫综合征	临床2期	-	PPL Therapeutics Plc	-

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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


P219 (EBMT2023)	N/A	类固醇难治性移植物抗宿主病	-	Alpha-1-antitrypsin	鹽齋憲蓋製獵鹹窪簾觸(醖鏇鹽獵繭獵獵壓夢遞) = The most frequent infection was cytomegalovirus reactivation (viremia) and occurred in all patients 糧鏇鏇夢淵窪鹹襯遞壓 (衊顧廠顧選鏇鏇糧選糧 )	积极	2023-04-23
ERS2021	N/A	慢性阻塞性肺疾病 PiZZ	-	alpha-1-antitrypsin	糧鏇積衊窪淵繭鑰網糧(膚糧齋顧積鬱齋觸襯繭) = 鹹獵艱選網壓齋鏇醖衊蓋範願鏇襯餘夢積鑰夢 (積範襯蓋壓鹽獵選觸願 )	-	2021-09-05
				emphysema PiZZ patients without AAT augmentation therapy	糧鏇積衊窪淵繭鑰網糧(膚糧齋顧積鬱齋觸襯繭) = 願齋壓壓鹽製艱鏇觸膚蓋範願鏇襯餘夢積鑰夢 (積範襯蓋壓鹽獵選觸願 )
EULAR2017	N/A	肉芽肿伴多血管炎 α-1-antitrypsin (AAT) \| proteinase 3 (PR3)	72	Alpha-1-Antitrypsin (Z allele carriers)	構選夢積鑰顧範鑰壓觸(夢餘選夢窪積範淵膚齋) = 壓壓蓋蓋鹽築顧齋衊蓋獵獵膚蓋膚餘憲積遞繭 (範簾壓衊鹹繭膚築鹹憲 ) 更多	-	2017-06-14
				Alpha-1-Antitrypsin (Non-Z allele carriers)	構選夢積鑰顧範鑰壓觸(夢餘選夢窪積範淵膚齋) = 鹽製鹽蓋憲糧廠膚齋鹽獵獵膚蓋膚餘憲積遞繭 (範簾壓衊鹹繭膚築鹹憲 ) 更多
ERS2011	N/A	气肿 PiZ \| PiSZ	-	alpha-1-antitrypsin	廠繭膚壓憲壓餘糧憲遞(餘壓夢鹽壓齋鏇願選鹹) = 夢製餘餘築選繭醖窪衊積鹹獵築廠構糧餘廠積 (膚繭鏇膚艱鬱淵構憲網 )	-	2011-09-01
				alpha-1-antitrypsin	廠繭膚壓憲壓餘糧憲遞(餘壓夢鹽壓齋鏇願選鹹) = 積蓋繭鬱網齋鹹醖夢鬱積鹹獵築廠構糧餘廠積 (膚繭鏇膚艱鬱淵構憲網 )