DASSOH - Systematic use of DDAVP to prevent serum sodium overcorrection in severe hyponatremia : a multicenter open-label randomized controlled trial - APHP220676

开始日期2024-12-17

申办/合作机构

Assistance Publique Hopitaux De Paris

NCT06020495

/ Recruiting临床3期IIT

Systematic Use of DDAVP to Prevent Serum Sodium Overcorrection in Severe Hyponatremia: a Multicenter Open-label Randomized Controlled Trial

ICU patients with severe hyponatremia and a high risk of rapid SNa overcorrection.

开始日期2024-12-17

申办/合作机构

Assistance Publique des Hôpitaux de Paris SA

NCT06635629

/ Recruiting临床2期IIT

Phase II Prospective Evaluation of Desmopressin Stimulation Test Performance in ACTH-dependent Cushing s Syndrome

Background:
Cushing syndrome (CS) is a set of diseases that develop when the body produces too much adrenocorticotropic hormone (ACTH). ACTH stimulates the production of a hormone called cortisol. Excess cortisol can cause serious issues, such as diabetes, high blood pressure, weight gain, and mood changes. Diagnosing CS early can be difficult. One test used to diagnose CS, the desmopressin (Desmo) stimulation test (DesmoST), has not been studied in enough people to know how accurate it is.
Objective:
To find ways to improve the DesmoST. Researchers especially want to learn more about how well the DesmoST identifies people with specific ACTH CSs: Cushing disease (CD) and ectopic ACTH syndrome (EAS).
Eligibility:
People aged 18 to 70 years who have or may have CS, especially CD or EAS. Healthy volunteers are also needed.
Design:
Participants with CS will have 3 DesmoSTs at least 48 hours apart. The procedure for each is as follows:
They will limit their fluid intake the day before each test. They will have nothing to eat or drink for 12 hours before the test.
For 1 of the tests, they will take a pill that contains a hormone (dexamethasone). They will take it around 11 pm the day before the test.
Desmo is given through a tube attached to a needle inserted into a vein.
Blood will be drawn a total of 6 times before and after the desmo is given.
Healthy volunteers will have 4 DesmoSTs. These will be 2 to 14 days apart.
All participants will have follow-up visits 3 to 5 days after each test. These visits may be by phone.

开始日期2024-12-12

申办/合作机构

National Institute of Diabetes & Digestive & Kidney Diseases

100 项与醋酸去氨加压素相关的临床结果

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100 项与醋酸去氨加压素相关的转化医学

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100 项与醋酸去氨加压素相关的专利（医药）

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6,215

项与醋酸去氨加压素相关的文献（医药）

2025-12-01·CHILDS NERVOUS SYSTEM

Intracranial lobar germinoma presenting with refractory hypernatremia

Article

作者: Saini, Jitender ; Arivazhagan, A ; Sipani, Mahak ; Shashidhar, Abhinith ; Birua, Gyani Jail Singh ; Rao, Shilpa

Central nervous system germ cell tumours are most commonly seen in the midline structures. Eccentric germinomas are rare but can be found in the basal ganglia, lobar, brainstem, and cranial vault. We present a 14-year-old boy who presented with signs and symptoms of overt diabetes insipidus and a radiological diagnosis of left frontal high-grade glioma. Confirmative histopathology revealed germinoma. The patient had a resolution of hypernatremia post-surgery and maintained normal sodium homeostasis on a single dose of 0.1 mg of tablet desmopressin. This paper showcases a rare case of an eccentric central nervous system germinoma in a young male patient presenting with Diabetes Insipidus without any apparent sellar or suprasellar involvement.

2025-07-01·AMERICAN JOURNAL OF EMERGENCY MEDICINE

Impact of desmopressin on hematoma expansion in patients presenting to the emergency department on antiplatelet therapy: Don't expand study

Article

作者: Shogren, Sophie ; Dee, Elizabeth ; Delibert, Samantha ; Howington, Gavin T. ; George, Benjamin P. ; Guerrero, Luis ; Ray, Lance ; Treu, Cierra N. ; Woolum, Jordan A. ; Rech, Megan A. ; Aggarwal, Deep ; Gilbert, Brian W. ; Ayad, Michael ; Maynard, Kaylee ; Slocum, Giles W. ; Newell, Joshua S. ; Faine, Brett A. ; Panos, Nicholas G. ; Brown, Caitlin ; Laub, Jessica ; Naylor, Ryan M. ; Flack, Tara ; Koul, Uttara ; Sheldrake, Amy K. ; Malik, Atul ; Sarangarm, Preeyaporn ; Vantine, Paul

INTRODUCTION:

Current guidelines state the effectiveness of desmopressin to reduce hematoma expansion in antiplatelet-related intracerebral hemorrhage (ICH) is uncertain. This study sought to determine if desmopressin decreased hematoma expansion in ICH patients on antiplatelet agents.

METHODS:

We conducted a multi-center, retrospective propensity-matched cohort study at 11 US emergency departments (ED) that participate in EMPHARM-NET. Adult patients ≥18 years with a primary diagnosis of spontaneous ICH on antiplatelets prior to admission from January 1, 2017 through May 1, 2021 were included. The primary endpoint was good or excellent hemostatic efficacy within the first 24 h following ICH between patients that did and did not receive desmopressin. Brain imaging was reviewed using 3D-Slicer by blinded expert physicians.

RESULTS:

Overall, 1408 patients were evaluated for inclusion. A total of 324 patients were included, of which 13.8 % (n = 45) received desmopressin and 86.1 % (n = 279) did not. After propensity matching, 35 patients receive desmopressin compared to 140 controls. Baseline hematoma volume (27.6 mL vs. 2.1 mL) and was significantly higher in the desmopressin group. The primary endpoint of good or excellent hemostatic efficacy was similar between groups (74.3 % desmopressin group vs. 85 % control group, -10.7 % [-28.1 % to 6.7 %]). There was no difference in secondary outcomes.

CONCLUSION:

In this multicenter cohort, patients receiving desmopressin had higher baseline intraparenchymal hematoma volume, and did not appear to result in improved hemostatic efficacy relative to the control group. These results suggest against routine administration of desmopressin for antiplatelet-related ICH, though future study in a randomized trial design is necessary.

2025-06-01·JOURNAL OF PHARMACEUTICAL SCIENCES

Development of polylactic acid microneedles for enhanced transdermal delivery of desmopressin peptides: A computational study

Article

作者: Fanaei, Yegane ; Salimibani, Milad ; Dashti, Amirhossein

Addressing the challenges associated with traditional injection therapies, this research marks a significant advancement in personalized and targeted therapeutic interventions, offering improved efficacy, convenience, and safety for patients undergoing peptide-based treatments. This article presents a research study on the development and assessment of polylactic acid (PLA) microneedles for enhancing the delivery of proteins and peptides, mainly focusing on the transdermal administration of desmopressin. Using simulations with COMSOL multiphysics software, it has been shown that PLA microneedles have excellent durability and controlled drug delivery capabilities, which promise efficient and patient-friendly transdermal drug delivery applications. Computational modeling results highlighted the dynamic behavior of desmopressin flow within the microneedle system, emphasizing accelerated drug transport capabilities. The utilization of dissolving microneedles in this study underscores the potential of microneedle technology as a promising solution for enhancing transdermal drug permeation, particularly for hydrophilic and macromolecular substances like proteins and peptides, thus opening new avenues for effective drug delivery systems.

131

项与醋酸去氨加压素相关的新闻（医药）

2025-04-28

·GlobeNewswire-Health Care

Eton Pharmaceuticals Announces Submission of NDA for ET-600 (Desmopressin Oral Solution)

- NDA submitted for the treatment of central diabetes insipidus -- Expected 10-month review; commercial preparations underway for a potential Q1 2026 launch – DEER PARK, Ill., April 28, 2025 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for ET-600, Eton’s proprietary, patented oral solution of desmopressin under development for the treatment of central diabetes insipidus. The Company expects the application to be assigned a 10-month FDA review, allowing for potential approval and launch in the first quarter of 2026. “The NDA submission for ET-600 is another important milestone for Eton, and for the thousands of children impacted by diabetes insipidus. If approved, ET-600 will be the only FDA-approved oral liquid formulation of desmopressin, providing the small, precise, and titratable doses required to treat pediatric patients,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “Leading pediatric endocrinologists have long expressed a need for this product, and we’re pleased to be one step closer to bringing it to patients. Pre-launch commercial activities are already underway as we plan for a potential approval in the first quarter of 2026.” Eton has been issued a patent by the U.S. Patent and Trademark Office on ET-600, which expires in 2044 and has additional patent applications under review. In a bioequivalence study conducted in 75 human subjects, ET-600 demonstrated pharmacokinetic equivalence to the FDA-approved reference product of the same active ingredient. The company estimates that central diabetes insipidus impacts approximately 3,000 pediatric patients in the United States. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has seven commercial rare disease products: INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has six additional product candidates in late-stage development: ET-400, ET-600, Amglidia®, ET-700, ET-800 and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Eton to undertake certain activities and accomplish certain goals and objectives. These statements include but are not limited to statements regarding Eton’s business strategy, Eton’s plans to develop and commercialize its product candidates, the safety and efficacy of Eton’s product candidates, Eton’s plans and expected timing with respect to regulatory filings and approvals, and the size and growth potential of the markets for Eton’s product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Eton’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Eton’s development programs and financial position are described in additional detail in Eton’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Eton undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Investor Relations:Lisa M. Wilson, In-Site Communications, Inc.T: 212-452-2793E: lwilson@insitecony.com Source: Eton Pharmaceuticals, Inc.

申请上市

2025-04-28

·ir.etonpharma.com

Eton Pharmaceuticals Announces Submission of NDA for ET-600 (Desmopressin Oral Solution) | Eton Pharmaceuticals, Inc.

News Release Details « Back to news Eton Pharmaceuticals Announces Submission of NDA for ET-600 (Desmopressin Oral Solution) - NDA submitted for the treatment of central diabetes insipidus - - Expected 10-month review; commercial preparations underway for a potential Q1 2026 launch – DEER PARK, Ill., April 28, 2025 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for ET-600, Eton’s proprietary, patented oral solution of desmopressin under development for the treatment of central diabetes insipidus. The Company expects the application to be assigned a 10-month FDA review, allowing for potential approval and launch in the first quarter of 2026. “The NDA submission for ET-600 is another important milestone for Eton, and for the thousands of children impacted by diabetes insipidus. If approved, ET-600 will be the only FDA-approved oral liquid formulation of desmopressin, providing the small, precise, and titratable doses required to treat pediatric patients,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “Leading pediatric endocrinologists have long expressed a need for this product, and we’re pleased to be one step closer to bringing it to patients. Pre-launch commercial activities are already underway as we plan for a potential approval in the first quarter of 2026.” Eton has been issued a patent by the U.S. Patent and Trademark Office on ET-600, which expires in 2044 and has additional patent applications under review. In a bioequivalence study conducted in 75 human subjects, ET-600 demonstrated pharmacokinetic equivalence to the FDA-approved reference product of the same active ingredient. The company estimates that central diabetes insipidus impacts approximately 3,000 pediatric patients in the United States. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has seven commercial rare disease products: INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has six additional product candidates in late-stage development: ET-400, ET-600, Amglidia®, ET-700, ET-800 and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Eton to undertake certain activities and accomplish certain goals and objectives. These statements include but are not limited to statements regarding Eton’s business strategy, Eton’s plans to develop and commercialize its product candidates, the safety and efficacy of Eton’s product candidates, Eton’s plans and expected timing with respect to regulatory filings and approvals, and the size and growth potential of the markets for Eton’s product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Eton’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Eton’s development programs and financial position are described in additional detail in Eton’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Eton undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Investor Relations: Lisa M. Wilson, In-Site Communications, Inc. T: 212-452-2793 E: lwilson@insitecony.com Source: Eton Pharmaceuticals, Inc. Source: Eton Pharmaceuticals

申请上市

2025-04-07

·ChenFJMU

重磅预防｜全球第七款siRNA药物Fitusiran 获批，用于血友病 A/B出血发作，每两月皮下注射一次！

ChenFJMU团队编辑（原创，全链条硬核解读：从结构剂型，到临床药理，到合理用药）思语湖畔QuoteYou were born to win, but to be a winner you must plan to win, prepare to win, and expect to win. — Zig Ziglar Fitusiran，01Qfitlia（Fitusiran，芬妥司兰），由赛诺菲（Sanofi）和Alnylam制药公司联合研发。于2025年3月28日获美国FDA批准，用于常规预防或减少12 岁及以上患有血友病 A 或血友病 B 的成人和儿童患者的出血发作频率，无论是否有凝血因子 VIII 或 IX 抑制剂。作为首个抗凝血酶（AT）降低寡核苷酸疗法，被证明通过抑制肝脏中抗凝血酶的生成发挥作用。在中国，CDE已于2024年5月受理了Fitusiran注射液的新药上市申请，并将其纳入《以患者为中心的罕见疾病药物研发试点工作计划》，拟用于作为常规预防治疗，用于有或无凝血因子VIII或IX抑制物的血友病A或B的成人和12岁以上患者。这款“First-in-class”创新siRNA疗法，有望在五年内成为重磅的革命性预防手段，将有望造福所有血友病患者。血友病 A/B 是罕见的遗传性终身出血疾病，因 VIII 或 IX 因子缺乏导致凝血功能受损，引发过度出血和自发性关节出血。因此，患有血友病的患者无法正常凝血，在受伤或手术后出血时间可能比正常情况更长，他们还可能出现肌肉、关节和器官自发性出血，导致关节损伤、慢性疼痛并显著影响生活质量。这些出血事件通常需要使用含有 FVIII 或 FIX 的产品或模拟因子按需、间歇性治疗或预防性治疗来控制。Qfitlia的获批不仅是血友病治疗的里程碑，也为siRNA疗法在其他疾病中的应用开辟了道路。这种精准的基因治疗技术有望在未来改变更多罕见病患者的命运。【黑框警告】Qfitlia可能会引发血栓事件（血液凝固）、急性和复发性胆囊疾病（部分患者需要切除胆囊）和肝毒性。警告患者需要在基线时进行肝血检测，并在开始使用 Qfitlia 治疗或增加剂量后至少六个月内每月监测一次。【结构与剂型处方】Fitusiran序列：正义链GGUUAACACCAUUUACUUCAA；反义链GACCAAUUGUGGUAAAUGAAGUU。采用Alnylam公司增强稳定化学ESC-GalNAc共轭技术，序列中包括硫代磷酸酯PS（6个）、2’-OMe（Am、Cm、Gm、Um）、2’-F（Af、Cf、Gf、Uf）、GalNAc靶头修饰（L96）。Qfitlia注射液中的有效成分为Fitusiran，这是一种抗凝血酶导向的双链小干扰核糖核酸（siRNA），与GalNAc部分的配体共价连接。Fitusiran钠盐的分子式为C520H636F21N175Na43O309P43S6，分子量为17193 Da。该原料药为白色至淡黄色粉末，易溶于水和磷酸盐缓冲盐溶液，在50 mM KCl中1%水溶液的pH值为5.4（4.4-7.3）。结构序列如下：Qfitlia是一种无菌、无防腐剂、透明、无色至淡黄色的溶液，用于皮下注射（SC）给药。每支50 mg单剂量预充笔在0.5 mL中输送50 mg Fitusiran（相当于53.0 mg Fitusiran-Na），还含有磷酸一氢钠0.585 mg、磷酸二氢钠0.044 mg、氯化钠2.455 mg和USP注射用水。每个20 mg单剂量小瓶在0.2 mL中输送20 mg Fitusiran（相当于21.2 mg Fitusiran-Na），还含有磷酸一氢钠0.234 mg、磷酸二氢钠0.018 mg、氯化钠0.982 mg和USP注射用水。可以加入磷酸和氢氧化钠调节pH至7.0。【作用机制】Fitusiran是一种抗凝血酶（AT）降低疗法，其不能替代缺失的凝血因子，而是通过降低抗凝血酶水平，促进凝血酶生成，从而实现止血平衡。它利用小干扰RNA（siRNA）技术，靶向结合肝脏中的抗凝血酶mRNA基因SERPINC1并阻断其翻译，抑制肝脏中抗凝血酶的产生，从而重新平衡止血功能。AT降低疗法示意图（Okaygoun D, et al. Journal of Biomedical Science, 2021, 28(1):100741.）Fitusiran的作用机制（Young, G et al. Research and Practice in Thrombosis and Haemostasis, 2023, 7(4):100179.）【药效学及药代动力学】药效学：在血友病患者的临床研究中，主要药效学（PD）观测指标是血浆AT的活性。该数据降低与年化出血率（ABR）减少相关，但AT活性持续低于15%可能增加血栓风险。在LTE15174研究中，AT活性平均值为24%（标准差4.64）。药代动力学：在血友病A或B患者服用后，评估Fitusiran的药代动力学（PK）特性，如下表所示。Fitusiran PD是由肝脏PK驱动而非血浆PK驱动，给药策略基于每月或每两个月给药10、20或50 mg，将血浆AT活性水平维持在15-35%之间。特殊人群用药：1、孕妇：尚无孕妇使用Fitusiran评估药物相关的重大出生缺陷、流产或其他不利的母亲或胎儿结局风险的可用数据。尚未使用Fitusiran对怀孕动物进行生殖研究。尚不清楚孕妇服用Fitusiran是否会对胎儿造成伤害或者影响生殖能力。2、哺乳期：没有关于母乳中存在Fitusiran或其代谢物、对母乳喂养的儿童的影响或对产奶量的影响的数据。尚不清楚在母乳喂养期间使用Fitusiran是否安全。3、儿童患者：在12岁及以上的儿童患者中，使用或不使用因子VIII或IX抑制剂的Fitusiran治疗血友病A或B的安全性和有效性已经得到证实，12岁以下儿童患者使用的安全性和有效性尚未确定。4、老年患者：临床研究没有纳入足够数量的65岁及以上患者，以确定他们的反应是否与年轻患者不同。5、肝功能损伤患者：已确诊肝功能损害(Child-Pugh A、B和C级)的患者应避免使用Fitusiran。6、避孕女性：使用激素避孕药的女性使用Fitusiran可能会增加血栓形成事件的风险。基于雌激素的激素避孕药是遗传性AT缺乏症女性血栓形成的既定危险因素。建议使用激素避孕药的患者在开始接受Fitusiran治疗之前和接受Fitusiran治疗期间使用替代非激素避孕药。免疫原性：在四项长达250周的Qfitlia治疗研究中，290名血友病成年人中有10名（3.4%）出现了抗药物抗体（ADA）。所有患者的ADA滴度均较低，大多数患者有短暂的ADA。没有发现ADA对Qfitlia的PK、PD、安全性或有效性有临床显著影响。【非临床毒理学】Fitusiran尚未对卵巢和雄性生殖器官影响的可逆性评估。无致癌性、致突变性、致染色体断裂性和对雄性生育力指标无不良影响。【临床研究】在两项3期研究中评估了Qfitlia对A型血友病或B型血友病的12岁及以上儿科及成人患者的疗效和安全性：研究ATLAS-INH（NCT03417102），针对具有抑制性抗体的A型或B型血友病患者；研究ATLAS-A/B（NCT03417245），针对不具有抑制性抗体的A型或B型血友病患者。研究受试者被随机分配接受每月一次的Qfitlia预防治疗或或根据需要接受常规按需治疗（旁路药物或凝血因子浓缩物），持续9个月。完成这两项研究的患者转入长期扩展研究ATLAS-OLE（NCT03754790），该研究中患者接受了基于抗凝血酶活性定期测量的可调剂量的Qfitlia。通过将长期扩展研究期间使用Qfitlia给药方案的患者与来自两项随机临床试验的按需对照数据进行比较，证实了Qfitlia的疗效。Qfitlia疗效的主要衡量指标是治疗出血的估计年化出血率（ABR）。在扩展研究中，接受基于抗凝血酶的Qfitlia给药方案的具有抑制物的受试者，与接受旁路药物按需治疗的受试者相比，估计年化出血率降低了73%（估计均值：5.1vs.19.1，p=0.0006）。在接受基于抗凝血酶的Qfitlia给药方案的无抑制物受试者中，与接受凝血因子浓缩物按需治疗的受试者相比，估计年化出血率降低了71%（估计均值：9.0vs.31.4，p<0.0001）。表5：在大于12岁使用抑制剂的患者中，Qfitlia预防治疗出血与按需旁路药物（BPA）治疗出血的年化出血率表6：在年龄大于12岁且无抑制剂的患者中，Qfitlia预防治疗出血与按需凝血因子浓缩物（CFC）治疗出血的年化出血率【用药指南】《使用手册》A. Qfitlia™（Fitusiran） injection（50 mg/0.5 mL）B.Qfitlia™（Fitusiran） injection（20 mg/0.2 mL）血友病治疗及Fitusiran展望（ChenFJMU）作为首款AT降低siRNA疗法，与传统的凝血因子替代疗法（如用于治疗A型血友病的VIII因子DDAVP®、Stimate®和Obizur®等、用于治疗B型血友病的IX因子Alprolix®和Nonacog alfa等）不同，Qfitlia通过一种新颖的机制发挥作用——降低AT水平以重新平衡止血。该药物是一种AT定向双链siRNA，通过皮下注射给药，每两个月一次，旨在通过RNA干扰导致AT mRNA降解，从而降低血浆抗凝血酶水平。与传统的凝血因子注射相比，Qfitlia的优势显而易见。它每年只需注射六次，极大地降低了患者的治疗负担，为血友病患者提供了一种低频给药、广泛适用、治疗效果显著的新选择，推动血友病治疗从“替代”向“根治”迈进。虽然基因治疗能够使血友病A/B患者恢复产生凝血因子，即只需要通过单次给药就可获得长期、稳定的治疗效果。但其昂贵的价格并非普通患者能够承担，据悉每剂Roctavian（valoctocogene roxaparvovec，用于治疗血友病A）定价为150万美元，而用于治疗血友病B的Hemgenix（etranacogene dezaparvovec）定价350万美元，刷新“全球最昂贵药物”的纪录。因此，Qfitlia这款“First-in-class”创新siRNA疗法，有望在五年内成为重磅的革命性预防手段，造福所有血友病患者。此外，单抗药物，如诺和诺德的Alhemo和辉瑞的Hympavzi都能在很大程度上造福血友病患者（详见公众号往期推文，2024年10月和12月FDA批准药物）。往期回顾重试重试编辑：廖源榕排版：周纯情补充和审核：陈昌买推送：ChenFJMU（欢迎个人转发分享，企业及其他公众号需授权后转载，并注明出处！）

siRNA寡核苷酸

100 项与醋酸去氨加压素相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D02235	醋酸去氨加压素	H01BA02	DB00035

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
夜尿	美国	Acerus Pharmaceuticals Corp.	2017-03-03
尿崩症	中国	Ferring Pte Ltd.	2001-06-29
神经性尿崩症	美国	Ferring Pharmaceuticals, Inc.	1984-03-30
夜间遗尿症	美国	Ferring Pharmaceuticals, Inc.	1978-02-21

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
多尿	临床3期	日本	Ferring Pharmaceuticals A/S	2016-09-01
原发性夜尿症	临床3期	加拿大	Ferring Pharmaceuticals A/S	2004-07-01
结直肠癌	临床2期	阿根廷	Laboratorio Elea Phoenix SA	2013-04-01
消化道出血	临床2期	阿根廷	Laboratorio Elea Phoenix SA	2013-04-01
膀胱过度活动症	临床2期	美国	Ferring Pharmaceuticals A/S	2013-01-01
乳腺癌	临床2期	阿根廷	Laboratorio Elea Phoenix SA	2011-11-01

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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


Company_Website 人工标引	N/A	神经性尿崩症	75	ET-600	齋糧襯簾衊夢憲膚糧鹹(廠衊觸壓醖醖壓糧繭廠) = positive results from its bioequivalence study of ET-600. 鬱艱遞構淵鏇網齋憲襯 (夢艱襯積糧衊衊選醖選 )	积极	2025-03-14
DDW2024	N/A	消化道出血	-	DDAVP	衊製鏇繭獵鹹醖壓襯鹹(壓夢蓋製淵餘壓網窪膚) = 觸齋願鹽齋壓蓋鑰簾鏇壓繭築願鑰簾醖憲築衊 (範觸鹽簾鹽夢網壓艱鏇 ) 更多	-	2024-05-21
				No DDAVP	衊製鏇繭獵鹹醖壓襯鹹(壓夢蓋製淵餘壓網窪膚) = 簾艱壓顧醖範願鏇窪積壓繭築願鑰簾醖憲築衊 (範觸鹽簾鹽夢網壓艱鏇 ) 更多
NCT04420585 (CTgov)	临床4期	夜间遗尿症 \| 半规管裂综合征 \| 镰状细胞血症	8	Desmopressin	積齋製網糧築構膚齋憲(鬱蓋憲遞觸獵糧憲築願) = 範範憲鹹簾選積廠艱衊顧醖醖夢鹽鹽獵糧夢襯 (夢膚艱簾齋積壓築鏇齋, 26.2) 更多	-	2024-05-07
SAT321 (ENDO2023)	N/A	大细胞神经内分泌肺癌	-	Desmopressin	鏇鹹簾壓願醖選廠繭鹽(糧齋艱簾窪淵構獵顧夢) = 觸餘製鏇夢餘餘鹽鹽淵構鏇願鏇網繭餘構鹹齋 (觸壓齋壓膚簾網醖獵獵 ) 更多	-	2023-10-05
FRI187 (ENDO2023)	N/A	库欣综合征	30	Desmopressin	製網製顧鏇膚獵觸糧範(顧窪淵廠鹽簾齋製築鹹) = 鏇築鹽觸選糧觸製願淵鹽夢選鬱鬱觸鑰糧襯糧 (範壓蓋構簾餘鑰積廠繭 )	-	2023-10-05
FRI248 (ENDO2023)	N/A	垂体ACTH分泌过多	1	Desmopressin	鬱齋範簾齋醖夢鏇壓鹽(積鬱製鏇願淵鬱築觸艱) = 窪廠鹽簾淵範壓廠鹽餘觸製憲鹽遞膚齋鹽鹹鏇 (獵範構艱鹹蓋積範淵積 ) 更多	-	2023-10-05
				Cabergoline	鬱齋範簾齋醖夢鏇壓鹽(積鬱製鏇願淵鬱築觸艱) = 築繭獵鹹範顧糧餘鹹鬱觸製憲鹽遞膚齋鹽鹹鏇 (獵範構艱鹹蓋積範淵積 ) 更多
ESPE2023	N/A	神经性尿崩症 ADH levels	12	Nasogastric administration of oral DDAVP lyophilised formulation	鑰願網糧鹽齋憲築鏇膚(蓋醖築衊醖醖醖顧範醖) = No episode of hyponatremia was observed 觸鹽顧獵遞鹹鏇艱夢衊 (憲範願齋夢遞顧艱願積 ) 更多	积极	2023-09-21
NCT01982760 (CTgov)	临床2期	瘀斑	14	DDAVP (DDAVP)	選觸築蓋觸憲觸簾鹹願(構鬱鏇餘蓋願網鬱齋衊) = 鬱觸網鑰餘齋積夢夢廠積觸觸網鏇廠淵衊鑰艱 (遞選蓋鹽鬱淵鬱餘繭蓋, 夢觸夢襯鑰鹽願憲夢鬱 ~ 構獵繭網憲繭淵鏇鬱鏇) 更多	-	2023-08-16
				DDAVP (No DDAVP)	選觸築蓋觸憲觸簾鹹願(構鬱鏇餘蓋願網鬱齋衊) = 蓋願窪廠選製觸鏇壓鏇積觸觸網鏇廠淵衊鑰艱 (遞選蓋鹽鬱淵鬱餘繭蓋, 齋鏇範鏇願鏇壓淵鹽壓 ~ 鏇選憲鬱蓋觸觸艱鑰夢) 更多
ISRCTN67038373 (DASH, Pubmed)	临床2期	脑出血 antiplatelet drugs	54	Desmopressin	膚繭範鬱壓醖鹹夢餘鏇(壓築醖顧膚衊願積廠夢) = 鬱築願範鏇繭艱衊築齋範醖醖獵艱繭簾蓋餘遞 (餘簾糧獵鹹淵獵醖襯積 ) 更多	-	2023-07-01
				Placebo	膚繭範鬱壓醖鹹夢餘鏇(壓築醖顧膚衊願積廠夢) = 膚繭簾顧鏇艱糧壓構夢範醖醖獵艱繭簾蓋餘遞 (餘簾糧獵鹹淵獵醖襯積 ) 更多
ISTH2023	N/A	1型血管性血友病	-	Capped DDAVP dose (20-30 mcg)	夢鹹廠願廠齋鏇鬱鏇憲(顧鏇鏇壓夢遞鬱鏇積網) = One adverse effect, hyponatremia in the non-capped group, was recorded 艱繭淵觸憲鹽範築網構 (築淵憲壓蓋鹽簾鏇蓋鏇 )	-	2023-06-24
				Non-capped DDAVP dose