This is a prospective, open-label drug study that will examine the effects of Zemaira (alpha-1 trypsin inhibitor) in patients with Eosinophilic Esophagitis.

开始日期2025-05-15

申办/合作机构

Children's Hospital & Medical Center

[+2]

NCT05582798

/ Completed临床4期IIT

A Proof of Concept Trial of Alpha-1 Antitrypsin Augmentation Therapy in Patients With Bronchiectasis

Double-blind, randomized, cross-over trial involving 20 participants with bronchiectasis.
This trial will make an important contribution to therapeutic development in bronchiectasis by determining whether alpha-1 antitrypsin (AAT) therapy results in reduced airway inflammation and improves neutrophil function.
Patients will be randomly assigned to receive Prolastin-C 120mg/kg (n=10 patients) by weekly intravenous infusions, Prolastin-C 180mg/kg (n=10 patients) by weekly intravenous infusions or placebo (0.9% saline) for a period of 4 weeks, followed by a 3-5 week washout period and a further 4 weeks during which patients will cross-over to receive the alternative therapy.

开始日期2022-11-01

申办/合作机构

University Of Dundee

NCT01700036

/ Completed临床2期IIT

A Pilot Study of Alpha-1-Antitrypsin (AAT) in Steroid Refractory Acute Graft vs Host Disease

This clinical trial will study the safety and efficacy of using the drug Zemaira, an Alpha 1-Antitrypsin (AAT) medication (also known as an Alpha1-Proteinase Inhibitor [Human]) for the treatment of steroid refractory GVHD.
For bone marrow transplant patients, the most common, serious complication is Graft vs Host Disease (GVHD), which at its most severe is a life-threatening, complication and a significant cause of treatment related death, following stem cell transplantation. GVHD is a major obstacle to the overall success of transplant treatment, a strategy that would otherwise provide the possibility of a cure for patients with blood cancers or severe blood disorders. GVHD primarily affects the skin, gut, and liver of the recipient, and involves the interaction of the recipient's (the host's) cells and tissues with the donor's immune system cells that see the host tissues as foreign, and attack the host's cells resulting in tissue and organ damage.
The severity of acute GvHD ranges from mild to severe, and for patients who don't respond to steroid therapy, the complication is nearly always fatal, either from organ damage or opportunistic infection as a consequence of high dose, steroid treatments.
There is currently no known effective therapy for patients with acute graft vs host disease that's refractory (nonresponsive) to steroid therapy. As stated earlier,the overwhelming majority of these patients may ultimately die from infection. The incidence of acute GvHD that requires intervention, is higher for unrelated donor transplants, the most common treatment option available, and therefore, these patients are at higher risk for treatment related complications from GVHD. Approximately 20,000 unrelated donor transplants are performed each year. The magnitude of this problem then is significant for patients who otherwise might be cured of their blood cancer or disease.

开始日期2013-07-01

申办/合作机构

Rogel Cancer Center: University of Michigan

[+2]

100 项与 Alpha-1-Proteinase Inhibitor (Human)(CSL Behring) 相关的临床结果

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100 项与 Alpha-1-Proteinase Inhibitor (Human)(CSL Behring) 相关的转化医学

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100 项与 Alpha-1-Proteinase Inhibitor (Human)(CSL Behring) 相关的专利（医药）

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项与 Alpha-1-Proteinase Inhibitor (Human)(CSL Behring) 相关的文献（医药）

2018-03-22·Blood1区 · 医学

α1-Antitrypsin infusion for treatment of steroid-resistant acute graft-versus-host disease

1区 · 医学

Article

作者: Choi, Sung Won ; Soiffer, Robert J. ; Alyea, Edwin P. ; Pawarode, Attaphol ; Magenau, John M. ; Antin, Joseph H. ; Riwes, Mary M. ; Yanik, Gregory A. ; Goldstein, Steven C. ; Koreth, John ; Braun, Thomas ; Lewis, Eli C. ; Reddy, Pavan ; Dinarello, Charles A. ; Ho, Vincent T. ; Peltier, Dan ; Kennel, Maggi ; Cutler, Corey S. ; Parkin, Brian L.

Key Points:

AAT infusion produced a high proportion of durable clinical responses in SR-aGVHD. AAT is associated with minimal toxicity and low rates of infection in patients with SR-aGVHD at significant risk for mortality.

2017-05-01·BIOLOGICALS

Pathogen safety and characterisation of a highly purified human alpha 1 -proteinase inhibitor preparation

Article

作者: Nowak, Thomas ; Gröner, Albrecht ; Popp, Birgit ; Schäfer, Wolfram ; Weber, David ; Roth, Nathan J ; Kee, Scott

Alpha₁-proteinase inhibitor (A₁PI) deficiency is a genetic condition predisposing to emphysema. Respreeza/Zemaira, a therapeutic preparation of A₁PI, is prepared from human plasma. This article describes the purity and stability of Respreeza/Zemaira and the capacity of virus and prion reduction steps incorporated into its manufacturing process. Purity and stability of Respreeza/Zemaira were analysed using established methods. To test pathogen clearance capacity, high levels of test viruses/prions were spiked into aliquots of production intermediates and clearance studies were performed for selected manufacturing steps, under production and robustness conditions, using validated scale-down models. Respreeza/Zemaira had a purity of 99% A₁PI and consisted of 96% monomers. It remained stable after storage for 3 years at 25 °C. Specific activity was 0.895 mg active A₁PI/mg protein. Pasteurisation inactivated enveloped viruses and the non-enveloped hepatitis A virus. 20 N/20 N virus filtration was highly effective and robust at removing all tested viruses, including parvoviruses, to below the limit of detection. Cold ethanol fractionation provided substantial reduction of prions. The manufacturing process of Respreeza/Zemaira ensures the production of a stable and pure product. Taking into consideration the donor selection process, the testing of donations, and the highly effective virus and prion reduction, Respreeza/Zemaira has a high safety margin.

2017-01-01·The Lancet. Respiratory medicine

Long-term efficacy and safety of α1 proteinase inhibitor treatment for emphysema caused by severe α1 antitrypsin deficiency: an open-label extension trial (RAPID-OLE)

Article

作者: Chorostowska-Wynimko, Joanna ; Stoicescu, Paul I ; Piitulainen, Eeva ; Thompson, Philip J ; Burdon, Jonathan ; Glanville, Allan ; Fries, Michael ; McElvaney, Noel G ; Edelman, Jonathan M ; Mäkitaro, Riitta ; Wark, Peter A B ; Sanak, Marek ; Seersholm, Niels ; Tortorici, Michael A ; Teschler, Helmut ; Wencker, Marion ; Hernandez, Paul ; Altraja, Alan ; Chapman, Kenneth R ; Vit, Oliver ; Holmes, Mark ; Chlumsky, Jan ; Ficker, Joachim H

BACKGROUND:

Purified α1 proteinase inhibitor (A1PI) slowed emphysema progression in patients with severe α1 antitrypsin deficiency in a randomised controlled trial (RAPID-RCT), which was followed by an open-label extension trial (RAPID-OLE). The aim was to investigate the prolonged treatment effect of A1PI on the progression of emphysema as assessed by the loss of lung density in relation to RAPID-RCT.

METHODS:

Patients who had received either A1PI treatment (Zemaira or Respreeza; early-start group) or placebo (delayed-start group) in the RAPID-RCT trial were included in this 2-year open-label extension trial (RAPID-OLE). Patients from 22 hospitals in 11 countries outside of the USA received 60 mg/kg per week A1PI. The primary endpoint was annual rate of adjusted 15th percentile lung density loss measured using CT in the intention-to-treat population with a mixed-effects regression model. This trial is registered with ClinicalTrials.gov, number NCT00670007.

FINDINGS:

Between March 1, 2006, and Oct 13, 2010, 140 patients from RAPID-RCT entered RAPID-OLE: 76 from the early-start group and 64 from the delayed-start group. Between day 1 and month 24 (RAPID-RCT), the rate of lung density loss in RAPID-OLE patients was lower in the early-start group (-1·51 g/L per year [SE 0·25] at total lung capacity [TLC]; -1·55 g/L per year [0·24] at TLC plus functional residual capacity [FRC]; and -1·60 g/L per year [0·26] at FRC) than in the delayed-start group (-2·26 g/L per year [0·27] at TLC; -2·16 g/L per year [0·26] at TLC plus FRC, and -2·05 g/L per year [0·28] at FRC). Between months 24 and 48, the rate of lung density loss was reduced in delayed-start patients (from -2·26 g/L per year to -1·26 g/L per year), but no significant difference was seen in the rate in early-start patients during this time period (-1·51 g/L per year to -1·63 g/L per year), thus in early-start patients the efficacy was sustained to month 48.

INTERPRETATION:

RAPID-OLE supports the continued efficacy of A1PI in slowing disease progression during 4 years of treatment. Lost lung density was never recovered, highlighting the importance of early intervention with A1PI treatment.

FUNDING:

CSL Behring.

项与 Alpha-1-Proteinase Inhibitor (Human)(CSL Behring) 相关的新闻（医药）

2025-10-31

·上药睿尔

10月速览（一）罕见病药研发之临床试验

罕见病药研发之临床试验 ☞ 渤健公布Dravet综合征小核酸疗法长期治疗数据 ☞ 驯鹿生物公布多发性硬化CAR-T疗法研究结果 ☞ 和誉医药公布腱鞘巨细胞瘤疗法III期临床长期研究数据 ☞ 赛诺菲公布AATD新药II期临床积极结果 ☞ AskBio公布罕见肌营养不良症基因疗法长期安全性数据 01 渤健公布Dravet综合征小核酸疗法长期治疗数据渤健与 Stoke Therapeutics共同在美国夏洛特举行的第54届儿童神经学会年会上公布了合作开发的在研药物Zorevunersen 在治疗Dravet综合征患者的开放标签延长研究（OLE）长期分析结果：研究结果显示，Zorevunersen具备作为Dravet综合征疾病修饰药物的潜力。三年期“临床整体变化印象量表（CGI-C）”与“照护者整体变化印象量表（CaGI-C）”数据显示：在19例可评估患者中，临床医生与照护者均报告95% 的患者整体临床状态得到改善。该结果与此前公布的“照护者报告生活质量（EQ-VAS）改善”数据形成互补验证。 I/IIa期及OLE研究的三年随访显示，在常规抗癫痫药物治疗基础上，Zorevunersen可显著且持久降低主要运动性癫痫发作频率。其中，“70mg负荷剂量+45mg维持剂量”方案效果最突出。 Zorevunersen是一种反义寡核苷酸药物，旨在通过增加脑细胞中SCN1A基因的非突变（野生型）拷贝产生的NaV1.1蛋白达到治疗Dravet综合征的目的。Zorevunersen对比常规抗癫痫药具有显著降低癫痫发作频率的效果，此外还具有改善神经发育、认知和行为的作用。 Zorevunersen已证明其疾病缓解潜力，并已获得FDA和EMA孤儿药称号。FDA还授予Zorevunersen罕见儿科疾病称号和突破性疗法称号，用于治疗Scon1A基因突变确认与功能获得无关的Dravet综合征。2025年2月份，Stoke与Biogen达成合作协议共同开发和商业化Zorevunersen。Stoke保留Zorevunersen在美国、加拿大和墨西哥的独家权利，Biogen获得世界其他地区的独家商业化权。Stoke获得1.65亿美元预付款，共同承担开发费用，并有资格获得最高3.85亿美元的里程碑付款等款项。 Dravet综合征是一种严重的发育性和癫痫性脑病（DEE），其特征是严重的反复癫痫发作以及严重的认知和行为障碍。大多数Dravet病例是由SCN1A基因的一个拷贝突变引起的，导致大脑神经元细胞中NaV1.1蛋白水平不足。发育和认知障碍通常包括智力障碍、发育迟缓、运动和平衡问题、语言和言语障碍、生长缺陷、睡眠异常、自主神经系统紊乱和情绪障碍。与一般癫痫人群相比，Dravet综合征的人因癫痫或SUDEP意外死亡的风险更高。 02 驯鹿生物公布多发性硬化CAR-T疗法研究结果驯鹿生物宣布，其自主研发的CAR-T细胞疗法伊基奥仑赛注射液在治疗进展型多发性硬化症（PMS）的研究结果已在国际学术期刊Cell上发布。研究纳入5例进展型多发性硬化症（PMS）患者，其中包括1例原发进展型MS（PPMS）患者和4例继发进展型MS（SPMS）患者。平均发病年龄38.2岁，基线平均EDSS评分为6.2分。患者接受单次伊基奥仑赛注射液输注后，全部患者的EDSS评分、九孔柱测试（9-HPT）和25英尺步行时间（T25FW）均出现显著改善；脑脊液寡克隆带（OCBs）完全消失，Kappa游离轻链（κFLC）水平显著下降；且MRI复查未发现新发或扩大的钆增强T1病灶及T2高信号病灶。80%患者仅出现短暂性1级细胞因子释放综合征（CRS），无≥2级CRS；未观察到免疫效应细胞相关神经毒性综合征（ICANS）或其他神经毒性反应。驯鹿生物成立于2017年，总部位于江苏南京江北新区生物医药谷园区，是一家专注于血液肿瘤细胞类药物和抗体药物开发的生物技术公司，目前拥有10余个开发管线产品。2023年6月30日，其自主研发的首个一类创新药伊基奥仑赛注射液获国家药监局批准上市，用于治疗多发性骨髓瘤，上市以来已惠及数百位患者。伊基奥仑赛注射液是一种针对B细胞成熟抗原（BCMA）的CAR-T细胞疗法，以慢病毒为载体转染自体T细胞，具有快速和强劲的疗效，并有突出的体内持久存续性，可使患者获得深度而持久的缓解。 03 和誉医药公布腱鞘巨细胞瘤疗法III期临床长期研究数据和誉医药在2025年欧洲肿瘤内科学会年会（ESMO Congress 2025）上公布了自主研发的 CSF-1R 抑制剂匹米替尼（pimicotinib）治疗腱鞘巨细胞瘤（TGCT）的全球III期研究长期数据：结果显示，匹米替尼在TGCT患者中展现出强劲而持久的肿瘤缓解疗效，临床结局评估（包括关节活动度、僵硬、疼痛和身体功能）表现出具有临床意义的持续改善。中位随访14.3个月时，part 1接受匹米替尼治疗的患者客观缓解率（ORR）从第25周的54%大幅提升至76.2%，且中位缓解持续时间尚未达到。part 2结束时（第49周），患者临床结局评估持续改善且安全性良好。截至73周，关节活动范围较治疗前提升23.9%。即便初始被分配至安慰剂组的患者，在后续转换为匹米替尼治疗后也收获显著获益，中位随访8.5 个月时ORR达到64.5%，患者临床结局评估改善。匹米替尼是和誉医药自主研发的一种高选择性的口服小分子CSF-1R抑制剂。2023年12月，和誉医药与默克就匹米替尼订立独家许可协议。根据该协议条款，默克最初获得匹米替尼在中国内地、中国香港、中国澳门及中国台湾所有适应症进行商业化的独家授权，并拥有全球商业化权利的独家选择权。2025年4月1日，默克根据双方于2023年12月签订的授权协议，行使匹米替尼全球商业化选择权，行权费用为8,500万美元。 04 赛诺菲公布AATD新药II期临床积极结果赛诺菲宣布，其在研新药efdoralprin alfa（SAR447537）在治疗罕见病α-1-抗胰蛋白酶缺乏症（AATD）的II期临床研究中取得了积极结果：在每三周（Q3W）/四周（Q4W）给药方案下，达成所有主要及关键次要终点。这是一项全球、双盲、随机试验，共纳入97名患者，按2:2:1分组，分别接受efdoralprin alfa Q3W、Q4W给药，或每周一次的血浆源性增强治疗（对照药为CSL Behring的Zemaira）。第32周时，efdoralprin alfa组患者稳态下的功能性AAT（fAAT）谷浓度均值增幅显著高于对照组，统计学差异极具意义（p<0.0001）。在关键次要终点上，两种给药方案均实现fAAT平均浓度的优效性提升，且fAAT 水平维持在正常范围下限以上的天数占比更高。 efdoralprin alfa是一种重组人α-1抗胰蛋白酶（AAT）-Fc融合蛋白，能以更低给药频率实现持续正常的fAAT水平，这对以维持保护性蛋白水平为核心的 AATD 肺部管理至关重要。且相比现有疗法仅能让患者短期达标，该药物无需依赖献血，就能持续维持正常AAT水平。这款产品最初由Inhibrx发现并开发，2024年初，赛诺菲以17亿美元收购Inhibrx并获得了这款产品。 05 AskBio公布罕见肌营养不良症基因疗法长期安全性数据拜耳的全资子公司AskBio公司宣布，其在第30届世界肌肉学会年会上，公布了在研基因疗法AB-1003针对肢带型肌营养不良症2I/R9（LGMD2I/R9）的I期/II期LION-CS101临床试验第一批参与者的中期安全数据：AB-1003安全性可接受，且中期数据呈现三大积极结果：（1）无严重风险事件：52周随访期内，未报告任何剂量限制性毒性（可能导致治疗中断的严重副作用）或严重不良事件，排除了核心安全隐患；（2）常见副作用轻微：仅出现头痛、跌倒、恶心等轻度至中度治疗相关不良事件，对患者生活影响较小；（3）特殊指标可控：3名参与者出现无症状的短暂转氨酶升高（肝功能相关指标），但胆红素水平正常，且在调整皮质类固醇治疗后均恢复至基线水平，无持续影响。 LGMD2I/R9是肢带型肌营养不良症（LGMD）的一个亚型，是FKRP基因突变后导致肌肉细胞内FKRP酶活性缺失，进而引发肌肉无力、萎缩等症状。目前全球范围内尚无获批的针对性治疗药物，临床仅能通过康复训练、对症支持等方式缓解症状，无法阻止疾病进展。 AB-1003是一款静脉输注的基因疗法，通过AAV载体将正常的FKRP基因递送至肌肉细胞，恢复FKRP酶活性，从源头改善疾病进程，具有安全性高、靶向性强的特点。关于上药睿尔上药睿尔是上海医药集团股份有限公司的全资子公司，作为上海医药旗下唯一的罕见病及儿童药平台，全力研发和转化临床急需、供应短缺、疗效确切及全球创新的罕见病药物。同时承担了上海医药在产罕见病药物的运营管理，力争成为中国罕见病药品的领头企业。在已发布的2批罕见病目录中，上海医药共有51个品种被列名用于治疗罕见病，产品覆盖内分泌代谢治疗领域、神经系统治疗领域、免疫系统治疗领域、呼吸系统治疗领域和血液系统治疗领域，涉及重症肌无力、肝豆状核变性、婴儿痉挛症等67个罕见病病症，是我国国内拥有罕见病药品批文最多的企业。上药睿尔目前自主研发品种17个，用于治疗血液恶性肿瘤疾病、肺动脉高压、苯丙酮尿症/高苯丙氨酸血症、结节性硬化、肝豆状核变性等罕见病。也积极与全球各大科研院所、高校、医疗机构及制药公司在罕见病神经系统疾病、血液系统疾病和呼吸系统疾病等治疗领域开展合作研发，共建创新平台。

细胞疗法免疫疗法孤儿药引进/卖出基因疗法

2025-10-23

·Being科学

赛诺菲22亿豪赌成功收网！efdoralprin alfa2期临床达到关键终点

当地时间10月22日，赛诺菲宣布了其在研药efdoralprin alfa（SAR447537，前身为INBRX-101）在全球ElevAATe II期研究中取得了积极结果，在每三周（Q3W）/四周（Q4W）给药方案下，达成所有主要及关键次要终点。该药用于治疗罕见病 α-1 抗胰蛋白酶缺乏症（AATD）肺气肿。实验共纳入97名患者，按2:2:1分组，分别接受 efdoralprin alfa Q3W、Q4W 给药，或每周一次的血浆源性增强治疗（对照药为 CSL Behring 的 Zemaira）。第32周时，efdoralprin alfa 组患者稳态下的功能性 AAT（fAAT）谷浓度均值增幅显著高于对照组，统计学差异极具意义（p<0.0001）。在关键次要终点上，两种给药方案均实现 fAAT 平均浓度的优效性提升，且 fAAT 水平维持在正常范围下限以上的天数占比更高。作为重组人 AAT-Fc 融合蛋白，efdoralprin alfa的耐受性良好，不良事件谱与血浆源性治疗相似。额外的安全性随访将在 ElevAATe OLE II 期研究（NCT05897424）中开展。 efdoralprin alfa核心突破在于降低给药频率，能以更低给药频率实现持续正常的 fAAT 水平，这对以维持保护性蛋白水平为核心的 AATD 肺部管理至关重要。且相比现有疗法仅能让患者短期达标，该药物无需依赖献血，就能持续维持正常AAT水平。 efdoralprin alfa并非赛诺菲的原研，而是去年以17亿美元从 Inhibrx 收购而来，算上监管里程碑付款，该交易总价值可达22亿美元。当时研发负责人预测其2025年出数据、2027 年上市，有望成为重磅药物。 efdoralprin alfa 已获FDA快速通道与孤儿药资格，其研发旨在恢复 fAAT 至正常范围并抑制肺损伤酶——中性粒细胞弹性蛋白酶。若获批，efdoralprin alfa将进入由 CSL、Grifols、Takeda 血浆源性药物主导的市场。当前已有多家企业布局下一代疗法：Grifols 的皮下给药候选药处于 I/II 期，Takeda 的 RNAi 药物进入 III 期，Wave Life Sciences 的 RNA 编辑寡核苷酸已公布 I b/II a 期数据。 AATD 是遗传性罕见病，因肝脏合成的 AAT 蛋白不足，导致肺、肝组织渐进性损伤，患者易发展为慢阻肺（含肺气肿），重症者需肺移植。自1987年血浆源性疗法问世后，该领域再无新药获批。全球约 23.5 万患者，但 90% 可能未获诊断。本文旨在行业资讯和知识分享，无任何商业用途。文章仅代表作者观点，不代表本站立场。往期推荐 1.康方生物依沃西单抗“一通乱杀”！头对头再次打败百济神州PD1单抗！ 2.不为人知的诺奖辛酸史！今年生理或医学诺奖的发现，曾因证据不足未获得广泛认可 3.掌握再生医学密码，这波流量iPSC抓住了 4.礼来口服orforglipron头对头试验，再次击败阿斯利康Farxiga 5.艾伯维起诉泽布替尼侵权案尘埃落定！但百济神州身陷多起“起诉案” Being科学公众号目前已经有多个交流群（好学，有趣的生物医药人才在这里聚集）。进群请扫描下方二维码，备注“姓名/昵称-企业/高校-具体研究领域/专业”，此群仅为科研交流群，非诚勿扰。发现“分享”和“赞”了吗，戳我看看吧

临床2期孤儿药临床3期并购寡核苷酸

2025-10-22

·FierceBiotech

Sanofi\'s $1.7B rare disease bet beats CSL\'s Zemaira in phase 2 face-off

Sanofi is working to enter a market currently served by plasma-derived therapies from CSL, Grifols and Takeda. \n Sanofi’s $1.7 billion rare disease bet from last year has delivered a phase 2 win. In a midstage study, efdoralprin alfa beat the standard of care on primary and key secondary endpoints, positioning the drugmaker to talk to regulators about the next steps.France’s Sanofi acquired the drug candidate from Inhibrx early last year. Shortly after news of the deal broke, Houman Ashrafian, Ph.D., head of R&D at Sanofi, told investors that the candidate could deliver data in 2025, launch in 2027 and generate blockbuster sales. The blockbuster prediction was built on the belief that existing patients would switch to efdoralprin alfa and new-to-treatment patients would choose the drug if it\'s approved.Sanofi took a step toward realizing that vision Wednesday by reporting top-line data from a phase 2 trial. The study randomized people with alpha-1 antitrypsin deficiency (AATD) emphysema to take the AAT-Fc fusion protein efdoralprin alfa or CSL Behring’s Zemaira intravenously.At Week 32, investigators recorded a statistically significant greater mean increase in functional AAT levels in people on the study drug than in those who received CSL’s standard-of-care plasma-derived therapy, according to Sanofi\'s Oct. 22 release. As well as meeting its primary endpoint, the trial hit key secondary endpoints that looked at other details of AAT levels, Sanofi said.Patients on Zemaira received the drug weekly. Efdoralprin alfa was given every three or four weeks, depending on which trial arm patients joined. Both efdoralprin alfa regimens beat Zemaira. Sanofi is yet to share data to compare the two efdoralprin alfa regimens, but it said the results support use every three or four weeks.The available evidence suggests Sanofi’s drug candidate is better at ensuring patients have adequate AAT, a protein that protects the lungs, and can be given on a less-burdensome schedule. Sanofi plans to meet with regulators to discuss the next steps. Inhibrx designed the trial to generate data to support approval. If approved, efdoralprin alfa will enter a market served by plasma-derived therapies from CSL, Grifols and Takeda. Sanofi is part of a small pack of companies working on next-generation candidates. Grifols has a subcutaneous candidate in phase 1/2, while Takeda is taking an RNAi prospect through phase 3. In addition, Wave Life Sciences recently shared phase 1b/2a data on an RNA editing oligonucleotide in AATD.

$Sanofi\'s $1.7B rare disease bet beats CSL\'s Zemaira in phase 2 face-off$

临床2期临床3期临床结果并购寡核苷酸

100 项与 Alpha-1-Proteinase Inhibitor (Human)(CSL Behring) 相关的药物交易

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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
气肿	澳大利亚	CSL Behring (Australia) Pty Ltd.	2017-02-13
α1-抗胰蛋白酶缺乏症	美国	CSL Behring LLC	2003-07-08
肺气肿	美国	CSL Behring LLC	2003-07-08

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
HIV感染	临床3期	美国	CSL Behring LLC	2006-12-01
嗜酸粒细胞性食管炎	临床2期	美国	CSL Behring LLC	2025-05-15
类固醇难治性移植物抗宿主病	临床2期	美国	CSL Behring LLC	2013-07-01
肺炎	临床2期	美国	CSL Behring LLC	2012-07-01

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临床结果

适应症

分期

评价

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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


NCT01370018 (CTgov)	临床2/3期	HIV感染	4	Zemaira (Alpha-1-Proteinase Inhibitor in HIV)	範構顧簾遞糧膚淵醖製(簾範艱簾獵製衊糧夢糧) = 廠鹽夢壓壓憲壓鑰艱衊淵構鹹壓衊壓鏇觸夢窪 (襯艱餘範壓衊壓廠鬱憲, 0.30)	-	2021-09-28
NCT01370018 (CTgov)	临床2/3期	HIV感染	4	Zemaira (Alpha-1-Proteinase Inhibitor)	艱夢衊範鏇選鹽膚憲網(衊淵獵壓廠顧選窪鬱艱) = 餘鏇顧製衊廠糧蓋窪膚淵範簾觸淵積簾窪憲鹽 (糧構淵壓遞顧衊範範蓋, 廠糧觸醖膚繭鹹範醖蓋 ~ 積齋鏇鹹範簾膚衊願選) 更多	-	2021-09-28
NCT01700036 (Pubmed \| Circulation) 人工标引	临床2期	急性移植物抗宿主病	40	α 1 -Antitrypsin	膚齋糧艱範選簾餘鬱鏇(築廠遞艱鹽繭鑰鹹艱糧) = 顧齋獵鹽壓鹹構願憲淵築壓餘網淵鏇窪鹽繭蓋 (顧艱鏇膚餘鹹醖網廠願 ) 更多	积极	2018-03-22
NCT01700036 (CTgov)	临床2期	类固醇难治性移植物抗宿主病	40	Alpha-1-Antitrypsin+AAT	鹽鬱鹹糧夢獵製繭蓋鏇 = 網壓壓製鏇獵鹹醖簾鹽顧醖艱鏇構廠糧顧築觸 (蓋憲淵網壓襯獵糧壓餘, 廠鏇艱獵艱衊構蓋鏇艱 ~ 選糧憲構獵艱憲蓋齋淵) 更多	-	2018-01-05
NCT00261833 (RAPID, CTgov)	临床4期	气肿 \| α1-抗胰蛋白酶缺乏症 \| 肺气肿	180	Alpha1-proteinase inhibitor (Zemaira®)	遞觸積網淵遞艱淵艱築(築艱夢糧網衊願齋選積) = 膚膚鹽衊齋夢鏇簾襯齋窪選鏇鹹遞憲餘網網齋 (顧繭廠觸蓋壓醖蓋觸壓, 0.22) 更多	-	2015-01-19
NCT00261833 (RAPID, CTgov)	临床4期	气肿 \| α1-抗胰蛋白酶缺乏症 \| 肺气肿	180	Placebo (Placebo)	遞觸積網淵遞艱淵艱築(築艱夢糧網衊願齋選積) = 製積願範觸遞鏇齋糧窪窪選鏇鹹遞憲餘網網齋 (顧繭廠觸蓋壓醖蓋觸壓, 0.24) 更多	-	2015-01-19