A Phase 1, Single-Sequence, 3 Period Crossover, Open-Label Study to Determine the Effect of Rabeprazole or H2 Blocker Famotidine on the Pharmacokinetics of Nirogacestat in Healthy Adult Male Participants

This study will evaluate the effects of the H2 blocker famotidine or the PPI rabeprazole on the PK of nirogacestat in healthy male participants

开始日期2025-09-09

申办/合作机构

Springworks Therapeutics, Inc.

NCT07170644

/ Recruiting临床2期

A Single-arm, Open-label Phase 2 Study of Nirogacestat in Adult Japanese Patients With Progressing Desmoid Tumors/Aggressive Fibromatosis (DT/AF)

This study is being conducted to characterize the efficacy and safety of nirogacestat in Japanese adults with progressing desmoid tumors/aggressive fibromatosis.

开始日期2025-08-08

申办/合作机构

Springworks Therapeutics, Inc.

NCT05879146

/ Recruiting临床2期IIT

Evaluation of the Response and Non-response of Nirogacestat in Desmoid Tumors- Clinical Study

To learn about the safety and effects of an investigational drug called nirogacestat when given to participants with a desmoid tumor/aggressive fibromatosis

开始日期2024-11-26

申办/合作机构

The University of Texas MD Anderson Cancer Center

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100 项与硝加司他相关的临床结果

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100 项与硝加司他相关的转化医学

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100 项与硝加司他相关的专利（医药）

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项与硝加司他相关的文献（医药）

2025-08-15·CURRENT TOPICS IN MEDICINAL CHEMISTRY

UPLC-LCMS-Based Method Development, Validation, Forced Degradation, and Impurity Profiling of Nirogacestat Drug Substance

Article

作者: Krishnan, Manikandan ; S. R., Thrinath ; Mankumare, Sharad D. ; K. S., Lakshmi

Introduction::

This study aims to establish a novel, straightforward, and reliable UPLCMS method for determining the stability and impurity profile of Nirogacestat under various stress conditions, in accordance with ICH guidelines. The stability of Nirogacestat was investigated under various stress conditions, including acid/base hydrolysis, oxidation (H2O2), photolysis, reduction, and thermal degradation. This research addresses the need for a validated, stability-indicating method that performs reliably across key analytical parameters, thereby contributing to pharmaceutical quality assurance.

Materials and Methods::

Stress testing was performed by exposing Nirogacestat to various degradation conditions, including acid (0.1 and 1N HCl), base (NaOH), oxidative (30% H₂O₂), thermal (105°C), photolytic, and reductive environments. The mobile phase consisted of acetonitrile and 0.1% triethylamine/formic acid, adjusted to pH 2.5 in a 30:70 (v/v) ratio. Chromatographic separation was achieved using an Acquity UPLC BEH Shield RP-18 column (50 × 1.0 mm, 1.7 μm), with a flow rate of 0.5 mL/min and detection at 251 nm. Linearity was evaluated over a concentration range of 0.25 to 1.5 μg/mL. Validation studies assessed parameters such as selectivity, linearity, accuracy, precision, robustness, and solution stability.

Results::

The method demonstrated excellent linearity (r² = 0.999), with peak area directly proportional to concentration within the studied range. All validation parameters were within acceptable limits. Forced degradation studies revealed distinct degradation products under each stress condition. Notably, alkaline degradation resulted in the least degradation, while acid, peroxide, photolytic, thermal, and reductive conditions produced a variety of degradation products. These were effectively separated from Nirogacestat using the developed method. The relative retention times for Nirogacestat and its impurities remained consistent, and mass spectrometry confirmed the identities of the degradation products.

Discussion::

The validated UPLC-MS method exhibited high sensitivity, selectivity, and robustness in detecting Nirogacestat and its impurities. It effectively distinguishes degradation products even within complex matrices and fully complies with ICH guidelines for analytical method validation. The degradation profile of Nirogacestat under various stress conditions provides critical insights into its stability behavior, which is essential for formulation development and regulatory compliance. The successful separation and identification of degradation products further underscore the method’s applicability as a stability-indicating assay.

Conclusion::

The developed UPLC-MS method is the first validated stability-indicating technique for Nirogacestat, offering comprehensive impurity profiling. It is precise, accurate, linear, and robust, making it highly suitable for routine quality control and regulatory submission. This method enables the reliable detection of degradation products, thereby enhancing the safety and efficacy profile of Nirogacestat in pharmaceutical preparations.

2025-05-30·Current Oncology

Evaluating Management of Extra-Abdominal Desmoid Fibromatosis: A Retrospective Analysis of Treatments, Outcomes and Recurrence Patterns.

Article

作者: Saraf, Vidhi ; Triplicane Dwarakanathan, Hariharan ; Gupta, Sanjay ; Mahendra, Ashish ; Al-Abri, Al-Muaayad ; Nixon, Ioanna ; Vaughan, Sarah

Background: Desmoid fibromatosis (DF) is a rare, locally aggressive soft tissue tumour with unpredictable clinical behaviour. Historically, treatment has involved surgery; however, contemporary guidelines, such as those from the Desmoid Tumour Working Group, advocate active surveillance. This article reviews current perspectives on DF, focusing on epidemiology, pathogenesis, treatment strategies, emerging research directions and cost effectiveness based on our experience at the West of Scotland Musculoskeletal Oncology Service, Glasgow Royal Infirmary (GRI). Methodology: We reviewed 101 patients diagnosed with desmoid fibromatosis between 2010 and 2024. A review of patient records was conducted to gather information on demographics, date of diagnosis, prior treatment, treatment initiation, intervention types, imaging intervals, follow-up duration, recurrence rate for surgery and other intervention, and discharge timelines. All data was systematically organized and analyzed to assess our outcomes. Results: Out of 101 patients with DF in the study, 66% were females. The most common site of primary tumour was lower extremity (39.6%) followed by near equal distribution in upper extremity and trunk. Out of the total cases, 72 (71.2%) were successfully managed with active surveillance involving serial imaging and clinical reviews in accordance with European guidelines. A total of 22 patients (21%) received treatment: 10 underwent surgery alone, 2 had surgery combined with radiotherapy, 8 received only radiotherapy, 1 was treated with hormonal therapy and 1 participated in a trial with Nirogacestat. Of the seven remaining patients, six had unplanned surgery outside followed by active surveillance at GRI. One patient was on alternative treatment modality, homeopathy. The average number of MRI scans per patient was 3.11, with many patients requiring significantly more imaging. MRI surveillance varies significantly in desmoid tumours due to their heterogeneous behaviour. Active or symptomatic tumours often require more frequent scans (every 3-6 months), while stable cases may need only imaging annually or just clinical monitoring. Recurrence was noted in eight patients, all of which were related to prior surgery. The total combined cost of imaging and appointments exceeds £6500 per patient in active surveillance. Conclusions: We conclude that most patients with desmoid fibromatosis in our cohort were effectively treated with active surveillance, consistent with current European guidelines. Surgical management of desmoid fibromatosis in our cohort is historic and has shown a significant recurrence risk. Our study proposes a revised follow-up protocol that significantly reduces costs without compromising on patient care. We suggest a two-year surveillance period for stable disease with patient-initiated return to reduce unnecessary clinic visits, imaging and healthcare costs.

2025-04-21·CANCER INVESTIGATION

Clinical Challenges and Evolving Treatments in Desmoid Fibromatosis: A Single Institution Experience

Article

作者: Gitelis, Steven ; Butler, Zachary ; Tasse, Jordan ; Yu, Austin ; T Blank, Alan ; Honore, Lesly ; Demetrious, Matthew ; Unson, Gabrielle

Desmoid tumor (DT), also known as desmoid fibromatosis, is a rare, locally proliferative tumor characterized by an overgrowth of myofibroblastic cells. Due to the varied clinical presentation of DT, there are a multitude of treatment options. This study provides our institutional experience in characterizing and treating DT as well as patient outcomes. A retrospective review was performed for 49 patients diagnosed with DT. Patient demographics, tumor characteristics, treatment characteristics, and tumor recurrence were reported. We reported our institution's treatment trends over time, relative risk analysis for surgery, as well as univariate analysis for recurrence. Thirty-seven patients received surgery with an overall recurrence rate of 29.7% (11/37). In total, ten patients received medical therapy including tamoxifen/sulindac (n = 7), nirogacestat (n = 1), and sorafenib (n = 2). One patient has been followed with active surveillance. Relative risk for surgery and tumor recurrence was not significantly correlated with race, gender, location, or large tumor size > 5 cm. Four patients treated with medical therapy experienced tumor reduction and symptomatic improvement. Management of DT includes many surgical and non-surgical options. We noted a similar recurrence rate in patients who received surgical treatment to what has been reported in the literature roughly 33%. We also noted effective tumor control in patients receiving medical therapy. As such, surgery can be utilized in situations with well-demarcated DT which can be removed en bloc, while utilizing medical therapy for highly invasive tumors.

223

项与硝加司他相关的新闻（医药）

2025-09-02

·药事纵横

2025年8月FDA新药获批速览

本文简单盘点了2025年8月份FDA新药审批情况，看看有哪些药界新星闪亮登场，给生命健康带来了新希望：【Modeyso】公司：Jazz Pharmaceuticals 批准日期：2025年8月6日治疗：弥漫性中线胶质瘤 FDA加速批准由Jazz Pharmaceuticals开发的“first-in-class”药物Modeyso（dordaviprone）上市，用于治疗1岁及以上、携带H3 K27M突变且在既往治疗后疾病进展的弥漫性中线胶质瘤成人和儿童患者。新闻稿指出，这是FDA首次批准针对H3 K27M突变弥漫性中线胶质瘤的全身性治疗。 Dordaviprone可通过两种信号通路产生抗癌作用，作为多巴胺受体抑制剂，它可以减弱多巴胺受体介导的RAS信号通路的激活，而RAS信号通路的激活与癌细胞增殖相关。此外，它可以过度激活ClpP，导致线粒体蛋白质的选择性降解，让癌细胞因能量供应短缺而死亡。【Hernexeos】公司：勃林格殷格翰批准日期：2025年8月8日治疗：非鳞状非小细胞肺癌 (NSCLC) FDA 宣布加速批准勃林格殷格翰的宗格替尼（Zongertinib）上市，用于治疗携带 HER2 (ERBB2) 酪氨酸激酶结构域 (TKD)激活突变且已接受过系统性治疗的不可切除或转移性非鳞状非小细胞肺癌 (NSCLC) 成人患者。宗格替尼是勃林格殷格翰开发的一款不针对EGFR野生型的选择性HER2抑制剂。临床前研究显示，它对所有主要的HER2突变（包括HER2 YVMA插入等位基因）均具有强效抑制的活性。在中国，2025年8月29日，NMPA宣布通过优先审评审批程序附条件批准勃林格殷格翰申报的1类创新药宗艾替尼片，该药单药适用于治疗存在HER2（ERBB2）激活突变且既往接受过至少一种系统治疗的不可切除的局部晚期或转移性非小细胞肺癌（NSCLC）成人患者。值得一提的是，2024年4月，中国生物制药已与勃林格殷格翰达成战略合作，双方将共同在中国内地研发和商业化勃林格殷格翰的肿瘤药物管线，其中包括宗格替尼。【Brinsupri】公司：Insmed 批准日期：2025年8月12日治疗：非囊性纤维化支气管扩张症（NCFB） Insmed 公司宣布，FDA 已批准其 Brensocatib 的新药上市申请，用于治疗成人和 12 岁以上儿童的非囊性纤维化支气管扩张症（NCFB）患者。该疗法此前已获 FDA 授予的优先审评资格与突破性疗法认定。Insight 数据库显示，Brensocatib是全球首个获批的二肽基肽酶 1（DPP-1）抑制剂，也是 FDA 批准的首个用于治疗 NCFB 的药物。 Brensocatib 是由Insmed 公司研发的一款口服、可逆的 DPP-1 抑制剂，可通过特异性抑制 DPP1 的活性，阻断其对 NSPs 的激活过程，从而减轻支气管扩张症等炎症性疾病所致的损伤效应。本次批准是基于 III 期临床试验 ASPEN 和 II 期临床试验 WILLOW 的数据。在 ASPEN 试验中，与安慰剂组相比，服用 Brensocatib 10mg和25mg的患者年发作率分别降低了21.1% 和19.4%。此外，两种剂量的 Brensocatib 均达到了几个与病情加重相关的次要终点，包括显著延长首次发作时间，以及显著提高治疗期间无发作的患者比例。接受 Brensocatib 25 mg治疗的患者，在使用支气管扩张剂后，第52周时肺功能下降显著减少。【Dawnzera】公司：Ionis Pharmaceuticals，Inc. 批准日期：2025年8月21日治疗：遗传性血管性水肿（HAE） Ionis Pharmaceuticals，Inc. 宣布FDA已批准DAWNZERA™（donidalorsen）用于预防12岁及以上成人和儿童患者遗传性血管性水肿（HAE）发作的预防性药物。DAWNZERA是首款也是唯一一款被批准用于 HAE的RNA靶向药物。它是一款反义寡核苷酸配体偶联（LICA）药物，旨在精确靶向以沉默前激肽释放酶（PKK）的表达，中断导致HAE发作的信号通路。PKK在激活与HAE急性发作相关的炎症介质中起重要作用。DAWNZERA 80mg 每四周（Q4W）或八周（Q8W）通过皮下自动注射器自行给药一次。 Donidalorsen是一款反义寡核苷酸配体偶联（LICA）药物，旨在精确靶向以沉默前激肽释放酶（PKK）的表达，中断导致HAE发作的信号通路。PKK在激活与HAE急性发作相关的炎症介质中起重要作用。2024年11月，美国FDA宣布正式受理donidalorsen的NDA，用于预防12岁及以上成人和儿童患者的HAE发作。 3期OASISplus研究结果显示，从既往预防性治疗方案切换至donidalorsen的患者，在此过程中未观察到疾病发作增加。治疗16周后，患者的平均HAE发作频率相较于既往治疗进一步显著降低。分析显示，从既往预防性治疗方案切换至donidalorsen的患者，其平均每月HAE发作次数进一步减少62%。血管性水肿控制测试（AECT）评分亦显示，药物切换后报告疾病控制良好的患者比例从67%升至93%，不论使用哪种既往治疗，患者的生活质量评估也获得普遍改善。此外，84%的受访患者表示更倾向于选择donidalorsen，主要原因包括疾病控制更佳、用药更便捷、注射部位不适反应更少等。此外，还有其他一些药物获批也值得关注：【NorUDCA】公司：Shilpa Medicare 批准日期：2025年8月8日治疗：代谢功能障碍相关脂肪性肝病（MAFLD） Shilpa Medicare于2025年8月8日宣布获得了印度中央药品标准控制组织（CDSCO）对500毫克去甲熊去氧胆酸（NorUDCA）片剂的全球首个监管批准，用以治疗代谢功能障碍相关脂肪性肝病（MAFLD）。根据新闻稿，这是MAFLD领域首个获批的治疗方案。 NorUDCA具有创新的双重作用机制，兼具抗炎功效与增强的胆汁酸调节功能。这一创新疗法有望阻止MAFLD恶化为更严重的肝脏疾病，如代谢功能障碍相关脂肪性肝炎（MASH）、肝硬化和肝衰竭。大量临床试验证明NorUDCA在疗效上显著优于安慰剂，且安全性优异，未报告重大不良事件。【Wegovy】公司：Novo Nordisk 批准日期：2025年8月15日治疗：非肝硬化型代谢功能障碍相关脂肪性肝炎（MASH）诺和诺德（Novo Nordisk）宣布，FDA已加速批准其重磅疗法Wegovy（2.4 mg司美格鲁肽）的补充新药申请（sNDA），用于联合低热量饮食及增加体育活动，治疗伴有中度至重度肝纤维化（2期或3期）的非肝硬化型代谢功能障碍相关脂肪性肝炎（MASH）成人患者。根据新闻稿，Wegovy为首个获批用于治疗MASH的胰高血糖素样肽-1（GLP-1）疗法。【Tonmya】公司：Tonix Pharmaceuticals 批准日期：2025年8月18日治疗：成人纤维肌痛症 Tonix Pharmaceuticals宣布，FDA已批准其“first-in-class”疗法Tonmya（cyclobenzaprine HCl舌下片）用于治疗成人纤维肌痛症。根据新闻稿，Tonmya是15年来首个获FDA批准用于治疗纤维肌痛症的疗法。 Tonmya（TNX-102 SL）是一种非阿片类药物，每日一次睡前服用，采用独特的舌下给药设计，能够实现快速的经黏膜吸收，并因绕过首过肝代谢而减少长半衰期活性代谢产物norcyclobenzaprine的生成。Tonmya是一种多功能药物，具有对5-HT2A血清素受体、α1-肾上腺素受体、H1-组胺受体和M1-毒蕈碱受体的强效结合和拮抗作用。【Ogsiveo】公司： SpringWorks Therapeutics 批准日期：2025年8月22日治疗：进展性硬纤维瘤 SpringWorks Therapeutics宣布，EU批准Ogsiveo（nirogacestat）片剂，用于需要全身治疗的进展性硬纤维瘤成人患者。根据新闻稿，Ogsiveo是首个在欧盟被批准用于治疗硬纤维瘤患者的药物，硬纤维瘤是一种罕见的软组织肉瘤亚型。 Ogsiveo是一款口服特异性γ-分泌酶小分子抑制剂。γ-分泌酶能够切割多种跨膜蛋白复合体，其中包括Notch蛋白。而Notch蛋白被认为能够激活导致硬纤维瘤生长的信号通路。Ogsiveo曾获得美国FDA授予快速通道资格、突破性疗法认定与优先审评资格，并在2023年11月获批用以治疗进行性、不可切除复发或难治性硬纤维瘤或深部纤维瘤病成人患者。 Ref:FDA官网、各公司新闻公告。立即扫码加入药事纵横交流群

优先审批突破性疗法加速审批上市批准临床2期

2025-08-21

·药事纵横

默克收购SpringWorks后第二项重大获批- OGSIVEO成欧盟首个硬纤维瘤治疗疗法

2025年8月18日，SpringWorks Therapeutics公司宣布EMA授予其口服γ分泌酶抑制剂OGSIVEO（nirogacestat）的上市许可，用于需要全身治疗的进展性硬纤维瘤成人患者。OGSIVEO是欧盟首个获批用于治疗硬纤维瘤的疗法。这是默克公司自完成以 39 亿美元收购美国生物技术公司SpringWorks 以来的第二次重大批准，今年7月欧盟批准口服 MEK 抑制剂 Ezmekly（mirdametinib），用于治疗1型神经纤维瘤病患者的肿瘤。这两种药物是默克公司在罕见肿瘤领域建立影响力的关键组成部分。硬纤维瘤是一种罕见的、局部侵袭性肿瘤，形成于人体的结缔组织中。在欧盟，每年约有1300至2300例新确诊的硬纤维瘤病例，其会导致剧烈疼痛、功能受限、活动能力丧失、畸形和疲劳。OGSIVEO是一种极具创新性的疗法，通过抑制γ分泌酶的活性，阻断Notch蛋白的蛋白水解激活过程，使其无法发挥促肿瘤作用，最终达到抑制硬纤维瘤生长的效果，且能显著改善硬纤维瘤症状，包括显著减轻疼痛。欧盟批准OGSIVEO是基于3期 DeFi 试验的结果，该试验纳入了142 名进展性硬纤维瘤成人患者，并达到了改善无进展生存期（PFS）的主要终点。与安慰剂相比，OGSIVEO 显示出具有统计学意义的显著改善，将疾病进展风险降低了71%。OGSIVEO在客观缓解率（ORR）方面也显示出显著改善，根据 RECIST v1.1 标准，OGSIVEO 组的确认ORR 为41%，而安慰剂组为8%；OGSIVEO组的完全缓解率为7%，安慰剂组为0%。OGSIVEO组的中位首次缓解时间为5.6个月，安慰剂组为11.1个月。此外，OGSIVEO 在患者报告结局（PROs）方面显示出早期且持续的改善，包括疼痛、硬纤维瘤特异性症状、身体/角色功能以及整体健康相关生活质量。整个研究期间OGSIVEO具有可控的安全性和耐受性。 OGSIVEO在美国和欧盟获批作为单一疗法，用于治疗需要全身治疗的进展性硬纤维瘤成年患者。FDA和EMA 已授予OGSIVEO 用于治疗硬纤维瘤的孤儿药称号，未来随着临床应用的推进，OGSIVEO有望进一步改写硬纤维瘤的治疗格局，让更多患者摆脱疾病困扰。立即扫码加入药事纵横交流群

孤儿药临床3期上市批准临床结果并购

2025-08-20

·药明康德

首款！欧盟批准抗肿瘤小分子疗法；超10亿美元合作助力开发潜在“best-in-class”小分子

首款！欧盟批准抗肿瘤小分子疗法 SpringWorks Therapeutics日前宣布，欧盟批准Ogsiveo（nirogacestat）片剂，用于需要全身治疗的进展性硬纤维瘤成人患者。根据新闻稿，Ogsiveo是首个在欧盟被批准用于治疗硬纤维瘤患者的药物，硬纤维瘤是一种罕见的软组织肉瘤亚型。此次Ogsiveo的获批是基于3期DeFi试验此前取得的积极结果。结果显示，DeFi试验达到了改善无进展生存期（PFS）的主要终点，Ogsiveo比安慰剂治疗有统计学上显著的改善，将疾病进展风险降低71%。基于RECIST v1.1的确认客观缓解率（完全缓解+部分缓解），Ogsiveo组为41%，安慰剂组为8%；Ogsiveo组的完全缓解率为7%，安慰剂组的完全缓解率为0%。此外，Ogsiveo在患者报告的结局（PROs）方面也显示出统计学意义和临床意义的改善。 Ogsiveo是一款口服特异性γ-分泌酶小分子抑制剂。γ-分泌酶能够切割多种跨膜蛋白复合体，其中包括Notch蛋白。而Notch蛋白被认为能够激活导致硬纤维瘤生长的信号通路。Ogsiveo曾获得美国FDA授予快速通道资格、突破性疗法认定与优先审评资格，并在2023年11月获批用以治疗进行性、不可切除复发或难治性硬纤维瘤或深部纤维瘤病成人患者。超10亿美元合作助力开发潜在“best-in-class”小分子 Jazz Pharmaceuticals与Saniona今日宣布，两家公司已达成全球许可协议，后者将授予Jazz对SAN2355的全球独家开发权。SAN2355是一种具差异性的钾通道激活剂，主要用于癫痫及其他潜在适应症的开发，旨在克服非选择性Kv7靶向化合物的局限性。根据协议条款，Jazz将负责主导并资助SAN2355的进一步开发、监管申报及全球商业化活动。Saniona将获得4250万美元的前期付款，并有资格根据开发和监管里程碑获得高达1.925亿美元的款项，以及获得高达8亿美元的潜在商业里程碑付款。 SAN2355是一种选择性、潜在“best-in-class”的小分子Kv7.2/Kv7.3钾通道激活剂，其机制已被证实可抑制癫痫发作。以往的Kv7靶向药物虽显示临床疗效，但剂量受脱靶激活引发的不良事件限制。SAN2355专一性针对Kv7.2/Kv7.3亚型，即在抑制癫痫发作的Kv7亚型的同时，避免激活其他Kv7亚型。这种高度选择性使SAN2355能够在保证疗效的同时实现剂量优化。参考资料: [1] European Commission Grants Approval of OGSIVEO® (nirogacestat) for the Treatment of Adults with Desmoid Tumors. Retrieved August 20, 2025 from https://www.globenewswire.com/news-release/2025/08/18/3135068/0/en/European-Commission-Grants-Approval-of-OGSIVEO-nirogacestat-for-the-Treatment-of-Adults-with-Desmoid-Tumors.html [2] Jazz Pharmaceuticals Enters Exclusive Licensing Agreement with Saniona to Develop and Commercialize SAN2355. Retrieved August 20, 2025 from https://www.prnewswire.com/news-releases/jazz-pharmaceuticals-enters-exclusive-licensing-agreement-with-saniona-to-develop-and-commercialize-san2355-302534545.html 免责声明：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：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

突破性疗法优先审批引进/卖出临床3期

100 项与硝加司他相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10960 D11453	-	-	DB12005

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
侵袭性纤维瘤病	美国	Springworks Therapeutics, Inc.	2023-11-27

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
角膜疾病	临床2期	希腊	GSK Plc	2023-07-14
卵巢颗粒细胞瘤	临床2期	美国	Springworks Therapeutics, Inc.	2022-08-30
卵巢颗粒细胞瘤	临床2期	加拿大	Springworks Therapeutics, Inc.	2022-08-30
卵巢颗粒细胞瘤	临床2期	波兰	Springworks Therapeutics, Inc.	2022-08-30
多发性骨髓瘤	临床2期	美国	GSK Plc	2020-06-08
多发性骨髓瘤	临床2期	澳大利亚	GSK Plc	2020-06-08
多发性骨髓瘤	临床2期	加拿大	GSK Plc	2020-06-08
多发性骨髓瘤	临床2期	法国	GSK Plc	2020-06-08
多发性骨髓瘤	临床2期	德国	GSK Plc	2020-06-08
多发性骨髓瘤	临床2期	希腊	GSK Plc	2020-06-08

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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


ESMO_SRC2025	N/A	侵袭性纤维瘤病 familial adenomatous polyposis (FAP)	13	Nirogacestat 150 mg BID	衊廠築製鬱簾衊選網醖(顧壓選廠壓餘餘選顧觸) = 鹹網鏇願獵製壓夢簾壓積襯衊繭鑰鹽築遞夢鏇 (範觸餘壓衊淵簾築鑰憲 ) 更多	积极	2025-03-21
NCT03785964 (DeFi, ESMO_SRC2025)	临床3期	侵袭性纤维瘤病	70	Nirogacestat	壓鑰鬱獵願餘鏇簾壓築(積夢艱襯餘艱糧憲鏇願) = 艱構獵鬱膚範願鏇顧壓糧糧齋鑰憲衊鏇艱鏇鬱 (壓繭憲鑰範憲鏇醖製壓 ) 更多	积极	2025-03-20
NCT03785964 (DeFi, GlobeNewswire) 人工标引	临床3期	侵袭性纤维瘤病	142	Nirogacestat 150 mg	選夢齋鹽襯憲鹽膚鬱淵(壓鹽繭觸醖衊獵網鬱範) = 範繭鑰構鏇艱襯鹹衊鏇糧範網繭簾範鬱製醖艱 (襯餘範選襯構繭醖製蓋 ) 更多	积极	2024-11-07
				Placebo	-
NCT05556798 (ASH2024)	临床1期	多发性骨髓瘤	9	玛贝兰妥单抗+Nirogacestat	壓築顧願遞願襯壓築鹹(鹹糧襯窪鑰壓遞齋蓋廠) = 糧膚願鹽醖遞膚糧憲製選淵鏇窪醖鏇鏇糧餘膚 (壓鑰鬱觸鑰積鹽網製鏇 )	积极	2024-11-05
NCT03785964 (DeFi, ASCO2024) 人工标引	临床3期	侵袭性纤维瘤病 APC Mutation	29	NirogacestatNirogacestat 150 mg	襯範齋淵鏇獵鏇衊觸鏇(範積顧窪窪窪襯窪糧壓) = 鏇襯鏇鑰壓糧觸鹹襯廠鑰鑰襯獵築壓遞衊鏇憲 (積積簾簾壓襯衊淵糧繭 ) 更多	积极	2024-05-24
				Placebo	襯範齋淵鏇獵鏇衊觸鏇(範積顧窪窪窪襯窪糧壓) = 壓淵鹹膚選繭蓋憲製膚鑰鑰襯獵築壓遞衊鏇憲 (積積簾簾壓襯衊淵糧繭 ) 更多
NCT03785964 (DeFi, ASCO2024) 人工标引	临床3期	侵袭性纤维瘤病 CTNNB1	142	Nirogacestat 150 mg	網顧餘衊窪遞憲艱築繭(醖衊廠遞遞繭遞製壓窪) = 構襯憲鬱範餘願壓襯鑰壓鹹鬱遞夢製網齋願鏇 (憲憲鹹壓繭壓齋構築積, 0.11 ~ 0.70) 更多	积极	2024-05-24
				Nirogacestat	網顧餘衊窪遞憲艱築繭(醖衊廠遞遞繭遞製壓窪) = 窪築蓋蓋蓋淵網範築憲壓鹹鬱遞夢製網齋願鏇 (憲憲鹹壓繭壓齋構築積, 0.13 ~ 0.80) 更多
NCT03785964 (DeFi, ESMO_SRC2024) 人工标引	临床3期	纤维瘤	144	Nirogacestat (niro) 150 mg	鏇窪窪醖鑰網齋憲壓顧(積鹽範襯繭窪鹹蓋網憲) = 鏇醖構鑰襯鹹製蓋鏇積繭選鏇網艱獵膚繭網獵 (鑰壓壓簾顧築遞鑰願膚 )	积极	2024-03-15
				Placebo	鏇窪窪醖鑰網齋憲壓顧(積鹽範襯繭窪鹹蓋網憲) = 網積糧膚壓網鹹觸蓋艱繭選鏇網艱獵膚繭網獵 (鑰壓壓簾顧築遞鑰願膚 )
NCT03785964 (DeFi, FDA) 人工标引	临床3期	侵袭性纤维瘤病	142	OGSIVEO	廠壓範醖築構積繭廠範(遞鹹襯選淵簾鬱壓範願) = 襯壓鑰網繭衊鹽範鹹網鏇積夢網艱壓構艱鹽願 (鑰醖膚鬱壓鬱夢窪獵網 ) 更多	积极	2023-11-27
				Placebo	廠壓範醖築構積繭廠範(遞鹹襯選淵簾鬱壓範願) = 範鹹簾壓鹽襯願淵築構鏇積夢網艱壓構艱鹽願 (鑰醖膚鬱壓鬱夢窪獵網, 8.4 ~ NR) 更多
NCT03785964 (DeFi , Biospace) 人工标引	临床3期	侵袭性纤维瘤病	142	Nirogacestat	齋顧壓選蓋構觸鏇糧簾(鹹膚蓋鹽鑰鹹簾鑰網選) = Nirogacestat significantly improved mean physical functioning score from baseline per the GODDESS DTIS PF domain compared with placebo at the pre-specified time point. The GODDESS DTIS PF domain captures varying degrees of vigorous and moderate daily activity, including moving and reaching. 醖襯齋鏇窪願壓鹽夢糧 (膚襯範蓋淵餘築膚簾鏇 ) 更多	积极	2023-11-01
				Placebo
NCT03785964 (DT/AF, ASCO 12023 \| ASCO 22023) 人工标引	临床3期	侵袭性纤维瘤病	122	Nirogacestat	網築廠網餘鹽窪選蓋襯(構鏇鬱選醖衊鏇艱鬱簾) = 繭獵繭壓壓願鹽鑰鬱願醖鑰遞鏇範齋襯顧淵願 (憲壓淵繭壓製襯簾範顧, –100.0 ~ 517.2) 更多	积极	2023-05-31
				Placebo	網築廠網餘鹽窪選蓋襯(構鏇鬱選醖衊鏇艱鬱簾) = 網憲構膚鑰淵繭築廠繭醖鑰遞鏇範齋襯顧淵願 (憲壓淵繭壓製襯簾範顧, –88.6 ~ 441.7) 更多