更新于：2024-11-01

Hyperparathyroidism

甲状旁腺功能亢进症

更新于：2024-11-01

基本信息

别名

Elevated blood parathyroid hormone level、HPTH - Hyperparathyroidism、HYPERPARATHYROIDISM

+ [24]

简介

A condition of abnormally elevated output of PARATHYROID HORMONE (or PTH) triggering responses that increase blood CALCIUM. It is characterized by HYPERCALCEMIA and BONE RESORPTION, eventually leading to bone diseases. PRIMARY HYPERPARATHYROIDISM is caused by parathyroid HYPERPLASIA or PARATHYROID NEOPLASMS. SECONDARY HYPERPARATHYROIDISM is increased PTH secretion in response to HYPOCALCEMIA, usually caused by chronic KIDNEY DISEASES.

关联

项与甲状旁腺功能亢进症相关的药物

Sotatercept

靶点

ACVR2A x activin receptor

作用机制

ACVR2A调节剂 [+1]

在研机构

Merck Sharp & Dohme BV [+8]

原研机构

Acceleron Pharma, Inc.

在研适应症

抑郁症 [+5]

非在研适应症

先天性单纯性红细胞再生障碍性贫血 [+11]

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2024-03-26

Upacicalcet Sodium Hydrate

靶点

CaSR

作用机制

CaSR调节剂

在研机构

天士力医药集团股份有限公司 [+4]

原研机构

Sanwa Kagaku Kenkyusho Co., Ltd.

在研适应症

继发性甲状旁腺功能亢进症 [+2]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

日本

首次获批日期2021-06-23

Evocalcet

依伏卡塞

靶点

CaSR

作用机制

CaSR激动剂

在研机构

Kyowa Kirin Korea Co., Ltd. [+1]

原研机构

Mitsubishi Tanabe Pharma Corp.

在研适应症

高钙血症 [+3]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

日本

首次获批日期2018-03-23

615

项与甲状旁腺功能亢进症相关的临床试验

NCT06647966 / Not yet recruitingN/A

Diagnostic Performance of [11C]Choline PET/CT In the Preoperative Assessment of Primary Hyperparathyroidism

Primary hyperparathyroidism (HPT) ranks as the third most common endocrine disorder, characterized by excessive secretion of parathormone (PTH), a hormone crucial for regulating calcium and phosphorus metabolism within the body. The only cure for HPT is surgically removing the hyperfunctioning parathyroid tissue, both in symptomatic and asymptomatic settings. With the advance of preoperative medical imaging techniques, a paradigm shift has taken place in the surgical approach of this condition, evolving from an inpatient bilateral cervical exploration to an outpatient minimally invasive procedure, which has proved to be superior in outcome. Due to the small size of the glands, preoperative visualisation and localisation are mandatory for the surgeon. Several imaging methods have been researched for identifying hyperfunctioning parathyroid glands, including morphological, functional and hybrid techniques, with no clear agreement on the optimal imaging approach.The preoperative localization modalities available are ultrasonography; parathyroid scintigraphy using technetium [99mTc]Tc sestamibi (MIBI), multiphase multidetector 4D computed tomography (4DCT); magnetic resonance imaging; and recently, positron-emission tomography/computed tomography (PET/CT) using 11C- and 18F-labelled radiotracers. While ultrasound and [99mTc]Tc-MIBI scintigraphy have been long used to diagnose and monitor patients with HPT, they can lead to numerous false negative results and therefore require additional examinations.Choline PET/CT is steadily replacing conventional imaging in many centres, allowing for the localization of adenomas in three quarter of patients with negative previous imaging examinations, but is still considered a relatively new method. [11C]C-Choline ([11C]CH) PET/CT is gradually gaining ground as a reliable method of localising HPT preoperatively, due to its fast uptake in hyperfunctioning parathyroid tissue, shorter acquisition time and lower radiation exposure compared to other nuclear medicine investigations and conventional methods. [11C]CH PET/CT has been suggested to be superior in diagnostic performances, compared to other imaging methods, reaching sensitivities up to 98.9%.The aim of our retrospective, single centre study is to evaluate the diagnostic performances and the inter-observer agreement of [11C]CH PET/CT for the preoperative detection of parathyroid adenomas in patients with suspected primary HPT.

开始日期2025-01-01

申办/合作机构-

NCT06562881 / RecruitingN/AIIT

Patient Navigation to Improve Surgical Access in Primary Hyperparathyroidism

The goal of this study is to address surgical health equity in historically marginalized participants with primary hyperparathyroidism (PHPT). The main questions that this study aims to answer are, how does patient navigation impact:
* The proportion of PHPT participants undergo parathyroidectomy?
* The proportion of PHPT participants who complete surgical consultation?
* Time to surgical consultation?
* Time to surgery?

开始日期2024-11-11

申办/合作机构

University of Pennsylvania

NCT06542315 / Not yet recruitingN/AIIT

Intraoperative Parathyroid Hormone Monitoring to Guide Surgery in Renal hyperparathyroIdism: A Pilot Trial

The goal of this pilot clinical is to determine the feasibility of a fully powered clinical trial to determine the effectiveness of intraoperative parathyroid hormone (IOPTH) criteria in guiding surgery for secondary and tertiary hyperparathyroidism. The main question it aims to answer is:
Is a fully powered trial investigating the role for IOPTH criteria in secondary and tertiary hyperparathyroidism feasible?
The comparison group is surgery not guided by IOPTH.
Participants will be randomized to undergo parathyroid surgery with one of four IOPTH criteria or a control arm that does not use IOPTH. All recruited patients are asked to complete quality of life and cognitive questionnaires, in addition to bloodwork during the study period.

开始日期2024-10-01

申办/合作机构

McMaster University

100 项与甲状旁腺功能亢进症相关的临床结果

登录后查看更多信息

100 项与甲状旁腺功能亢进症相关的转化医学

登录后查看更多信息

0 项与甲状旁腺功能亢进症相关的专利（医药）

登录后查看更多信息

30,127

项与甲状旁腺功能亢进症相关的文献（医药）

2025-01-01·Methods in Molecular Biology

Monitoring Calcium-Sensing Receptor (CaSR)-Induced Intracellular Calcium Flux Using an Indo-1 Flow Cytometry Assay

Article

作者: Gorvin, Caroline M ; Howles, Sarah A. ; Howles, Sarah A ; Gorvin, Caroline M.

The calcium-sensing receptor (CaSR) has a critical role in maintaining serum calcium concentrations within the normal physiological range, and mutations in the receptor, or components of its signaling and trafficking pathway, cause disorders of calcium homeostasis. Inactivating mutations cause neonatal severe hyperparathyroidism or familial hypocalciuric hypercalcemia (FHH), while gain-of-function mutations cause autosomal dominant hypocalcemia (ADH). Characterizing the functional impact of mutations of the CaSR, and components of the CaSR-signaling pathway, is clinically important to enable correct diagnoses of FHH and ADH, optimize management, and prevent inappropriate parathyroidectomy or vitamin D supplementation. CaSR signals predominantly by activating the G-alpha subunit-11 to mobilize calcium release from intracellular stores. Thus, measurement of CaSR-induced intracellular calcium (Ca²⁺_i) signaling is the gold standard method to investigate the pathogenicity of CaSR genetic variants. This protocol describes a method to assess CaSR-induced Ca²⁺_I signaling using the Indo-1 calcium indicator dye and flow cytometry. This method has been used to assess multiple genetic variants in CaSR and components of its signaling and trafficking pathway in HEK293 cells.

2024-12-31·Renal Failure

Prognosis and factors related to severe secondary hyperparathyroidism in long-term peritoneal dialysis patients

Article

作者: Chen, Menghua ; Tian, Na ; Chen, Na ; Wang, Li ; Zhang, Hongxia ; Yu, Min ; Feng, Xiaonan ; Wang, Yan ; Li, Yanmei ; Song, Shuhua

Secondary hyperparathyroidism (SHPT) can progress to severe SHPT (sSHPT), which affects the survival rate and quality of life of patients. This retrospective cohort study investigated risk factors for sSHPT and the association between SHPT and mortality (all-cause and infection-related) among 771 clinically stable patients (421 male patients; mean age, 51.2 years; median dialysis vintage, 28.3 months) who underwent >3 months of regular peritoneal dialysis (PD) between January 2013 and March 2021. The sSHPT and non-sSHPT groups comprised 75 (9.7%) (median progression, 35 months) and 696 patients, respectively. sSHPT was defined as a serum intact parathyroid hormone (PTH) level >800 pg/mL observed three times after active vitamin D pulse therapy. The influence of sSHPT on the prognosis of and risk factors for sSHPT progression were evaluated using logistic and Cox regression analyses. After adjusting for confounding factors, higher (each 100-pg/mL increase) baseline PTH levels (95% confidence interval (CI) 1.206-1.649, p < .001), longer (each 1-year increase) dialysis vintages (95% CI 1.013-1.060, p = .002), higher concomitant diabetes rates (95% CI 1.375-10.374, p = .010), and lower (each 1-absolute unit decrease) Kt/V values (95% CI 0.859-0.984, p = .015) were independent risk factors for progression to sSHPT in patients on PD. During follow-up, 211 deaths occurred (sSHPT group, n = 35; non-sSHPT group, n = 176). The sSHPT group had significantly higher infection-related mortality rates than the non-sSHPT group (12.0% vs. 4.3%; p < .05), and sSHPT was associated with increased infection-related mortality. In conclusion, patients with sSHPT are at higher risk for death and infection-related mortality than patients without sSHPT.

2024-12-31·Renal Failure

Clinical characteristics and treatment modalities in uremic and non uremic calciphylaxis - a dermatological single-center experience

Article

作者: Schliep, Stefan ; Renner, Regina ; Busch, Dorothee ; Erfurt-Berge, Cornelia ; Yousuf, Sabine

Calciphylaxis (CP) is a serious, potentially life-threatening disease that presents with medial calcification of small-sized vessels and painful ischemic ulcerations. Although calciphylaxis is frequently seen in patients with end-stage kidney disease on dialysis (calcific uremic arteriolopathy, CUA), there are reported cases of nonuremic calciphylaxis (NUC), which often remain undiagnosed. We conducted a retrospective chart review at our dermatological hospital and evaluated data concerning the epidemiology, comorbidities, medication, laboratory abnormalities, and therapeutic approaches of 60 patients diagnosed with calciphylaxis between 01/2012 and 12/2022. We identified 21 patients diagnosed with NUC and 39 with kidney disease. The predilection sites of skin lesions were the lower legs in 88% (n = 53), followed by the thigh and gluteal regions in 7% (n = 4). Significant differences were identified in comorbidities, such as atrial fibrillation (p < 0.001) and hyperparathyroidism (p < 0.01) accounting for CUA patients. Medication with vitamin K antagonists (p < 0.001), phosphate binders (p < 0.001), and loop diuretics (p < 0.01) was found to be associated with the onset of calciphylaxis. Hyperphosphatemia (p < 0.001), increased parathyroid hormone (p < 0.01) and triglyceride levels (p < 0.01), hypoalbuminemia (p < 0.01) and decreased hemoglobin values (p < 0.001) in the CUA cohort were significantly different from those in the NUC group. All patients with CUA received systemic medication. In contrast, only 38% (n = 8) of patients with NUC received systemic treatment. Striking discrepancies in the treatment of both cohorts were detected. In particular, NUC remains a disease pattern that is still poorly understood and differs from CUA in several important parameters.

166

项与甲状旁腺功能亢进症相关的新闻（医药）

2024-10-16

·GlobeNewswire-Health Care

Crinetics Pharmaceuticals to Report Third Quarter 2024 Financial Results on November 12, 2024

SAN DIEGO, Oct. 16, 2024 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced that it will report third quarter 2024 financial results on Tuesday, November 12, 2024 after the market closes. Company management will host a conference call at 4:30 p.m. ET to discuss financial results and provide a business update. Conference Call & Webcast Tuesday, November 12 @ 4:30 p.m. ET Domestic: 1-800-579-2543International: 1-785-424-1789Conference ID: CRNXQ3 Participants can use the dial-in numbers above OR access the call via live webcast on the Events section of the Crinetics website. To ensure a timely connection, it is recommended that participants dial-in or register at least 15 minutes prior to the scheduled start of the call. The webcast will be archived on the Investor Relations section of www.crinetics.com. About Crinetics Pharmaceuticals Crinetics Pharmaceuticals is a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for endocrine diseases and endocrine-related tumors. Crinetics’ lead development candidate, paltusotine, is the first investigational once-daily, oral, selectively-targeted somatostatin receptor type 2 (SST2) nonpeptide agonist that has completed Phase 3 clinical development for acromegaly and is in Phase 2 clinical development for carcinoid syndrome associated with neuroendocrine tumors. Crinetics is also developing atumelnant (CRN04894), an investigational, first-in-class, oral ACTH antagonist, that is currently completing Phase 2 clinical studies for the treatment of congenital adrenal hyperplasia and Cushing’s disease. All of the company’s drug candidates are orally delivered, small molecule new chemical entities resulting from in-house drug discovery efforts, including additional discovery programs addressing a variety of endocrine conditions such as hyperparathyroidism, polycystic kidney disease, Graves’ disease (including thyroid eye disease), diabetes, obesity and GPCR-targeted oncology indications. Investors:Gayathri DiwakarHead of Investor Relationsgdiwakar@crinetics.com (858) 345-6340 Media: Natalie BadilloHead of Corporate Communications nbadillo@crinetics.com (858) 345-6075

临床2期财报

2024-10-11

·药精通Bio

2024 FDA已批准34款新药！其中Q3新增13款！

OTC2024类器官前沿应用与3D细胞培养论坛圆满落幕，点击图片可查看会后报告，咨询OTC2025类器官论坛请联系：王晨 180 1628 8769 🧴Berdazimer （Zelsuvmi），一种用于治疗局部传染性软疣的一氧化氮（NO）释放剂。💉头孢吡肟/恩美唑巴坦（Exblifep），一种包含头孢吡肟和恩美唑巴坦的固定剂量组合，用于治疗尿路感染。 💉LetibotulinumtoxinA （Letybo），一种纯化的 A 型肉毒杆菌神经毒素蛋白复合物，用于改善眉间纹的外观。💉替雷利珠单抗（Tivembra）是一种 IgG4 变体 PD-1 mAb，适用于治疗不可切除、局部晚期或转移性食管鳞状细胞癌。💊Resmetirom （Rezdiffra），一种甲状腺激素受体 β （NR1A2）激动剂用于治疗非肝硬化性非酒精性脂肪性肝炎。 💊Aprocitentan （Tryvio），一种用于治疗高血压的内皮素受体拮抗剂。💊Givinostat （Duvyzat），一种组蛋白脱乙酰酶抑制剂，用于治疗杜氏肌营养不良症。💉 Sotatercept （Winrevair），一种用于治疗肺动脉高压的激活素信号抑制剂。💊 Vadadustat （Vafseo），一种口服缺氧诱导因子脯氨酰羟化酶抑制剂，用于治疗成人与 CKD 相关的症状性贫血。💊 Danicopan （Voydeya），补体因子 D 抑制剂，用于治疗 PNH 患者的血管外溶血。 💉 Ceftobiprole medocaril （Zevtera）是一种广谱头孢菌素抗菌剂，适用于某些类型的肺炎、菌血症和皮肤感染。💉 Pegulicianine （Lumisight），一种用于乳腺癌荧光成像的光学成像剂。💉 Nogapendekin alfa inbakicept （Anktiva）是一种白细胞介素 15 受体激动剂与卡介苗（BCG）的组合，用于治疗膀胱癌。 💊 Tovorafenib （Ojemda），一种 II 型 RAF 激酶抑制剂，用于治疗具有 BRAF 基因突变的小儿低级别胶质瘤。💊 Mavorixafor （Xolremdi），一种用于治疗 WHIM 综合征的 CXCR4 拮抗剂。💉 Tarlatamab （Imdelltra）是一种靶向 DLL3 和 CD3 的双特异性单克隆抗体和 T 细胞接合器，用于治疗广泛期小细胞肺癌。💉 Imetelstat （Rytelo），一种寡核苷酸端粒酶抑制剂，用于治疗骨髓增生异常综合征伴输血依赖性贫血。 💊 Elafibranor （Iqirvo），一种双重 PPARα/δ 激动剂，用于治疗原发性胆汁性胆管炎。🧴Sofpironium （Sofdra），一种用于治疗多汗症的 M3 毒蕈碱受体抑制剂。💉 Crovalimab （Piasky），一种用于治疗阵发性睡眠性血红蛋白尿症的补体 C5 抑制性 mAb。🫁 Ensifentrine，一种用于治疗 COPD 的双重 PDE3/4 抑制剂。💉Donanemab-azbt，一样β淀粉样蛋白（Aβ）定向抗体，用于治疗阿尔兹海默病。💊Deuruxolitinib，一种JAK1和JAK2抑制剂，用于治疗严重斑秃。💊Vorasidenib，一种 IDH1 和 IDH2 抑制剂，用于治疗具有易感 IDH1 或 IDH2 突变的 2 级星形细胞瘤或少突胶质细胞瘤。💉Palopegteriparatide，一种用于治疗甲状旁腺功能亢进症的甲状旁腺激素类似物。💉Nemolizumab，一种用于治疗结节性痒疹的 IL-31 受体 α 拮抗剂。 💊Seladelpar 是一种 PPAR-δ 激动剂，用于单独治疗原发性胆汁性胆管炎或与熊去氧胆酸联合治疗。💉Axatilimab 是一种 CSF-1R mAb，用于降低慢性移植物抗宿主病患者的致病性巨噬细胞活性。💊Lazertinib，一种 EGFRm 激酶抑制剂，与 amivantamab 联合用于治疗具有 EGFR 突变的非小细胞肺癌。💉Lebrikizumab，一种抗 IL-13 的 IgG4 mAb，用于治疗中度至重度特应性皮炎。💊Arimoclomol 是一种热休克蛋白共诱导剂，用于与 miglustat 联合治疗 C 型尼曼-匹克病的神经系统症状。💊L-乙酰亮氨酸，亮氨酸的一种改良形式，用于治疗 C 型尼曼-匹克病患者的神经系统症状。💊Xanomeline 和 trospium chloride，联合双重 M 1 - 和 M 4 - 选择性毒蕈碱受体激动剂和毒蕈碱受体拮抗剂，用于治疗精神分裂症。 💉Flurpiridaz F-18，一种用于评估心肌缺血和梗死的放射性诊断药物Picture from Chris De Savi 文章来源：早研早聊参考素材：素材来源官方媒体/网络新闻 END 免责声明：本文仅作知识交流与分享及科普目的，不涉及商业宣传，不作为相关医疗指导或用药建议。文章如有侵权请联系删除。 OTC2024类器官前沿应用与3D细胞培养论坛圆满落幕，点击图片可查看会后报告，咨询OTC2025类器官论坛请联系：王晨 180 1628 8769

上市批准

2024-10-10

·BiG生物创新社

2024年FDA批准新药汇总

作者｜momo 截止目前，FDA已经批准35款新药上市，其中多款药物具有里程碑意义。 01 2024年FDA已批准药物 1.1.Zelsuvmi (berdazimer) 适应症：局部传染性软疣批准日期：2024年1月5日企业：Novan Inc. 1.2.Exblifep (cefepime and enmetazobactam) 适应症：尿路感染靶点：LACTB、PBP 批准日期：2024年2月22日企业：Orchid Chemicals & Pharmaceuticals Ltd. 1.3.Letybo (letibotulinumtoxin a-WLBG) 适应症：中度至重度眉间纹的外观改善靶点：BoNT/A 批准日期：2024年2月29日企业：Hugel Inc. 1.4.Tevimbra (tislelizumab-JSGR) 适应症：不可切除、局部晚期或转移性食管鳞状细胞癌靶点：PD-1 批准日期：2024年3月13日企业：百济神州 1.5.Rezdiffra (resmetirom) 适应症：中晚期非肝硬化性非酒精性脂肪性肝炎靶点：THR-β 批准日期：2024年3月14日企业：罗氏 1.6.Tryvio 适应症：高血压靶点：ET-A、ET-B 批准日期：2024年3月19日企业：Idorsia. 1.7.Duvyzat 适应症：杜氏肌营养不良症靶点：HDAC 批准日期：2024年3月21日企业：Italfarmaco Spa. 1.8.Winrevair (sotatercept-CSRK) 适应症：肺动脉高压靶点：INHBA 批准日期：2024年3月26日企业：Acceleron Pharma Inc. 1.9.Vafseo 适应症：成人慢性肾脏疾病引起的贫血靶点：HIF 批准日期：2024年3月27日企业：宝洁 1.10.Voydeya 适应症：阵发性夜间血红蛋白尿治疗血管外溶血靶点：CFD 批准日期：2024年3月29日企业：Achillion Pharmaceuticals Inc. 1.11.Zevtera (ceftobiprole medocaril sodium) 适应症：血液感染、细菌性皮肤和相关组织感染、社区获得性细菌性肺炎靶点：PBP 批准日期：2024年4月3日企业：Roche 1.12.Lumisight 适应症：乳腺癌荧光成像批准日期：2024年4月17日企业：Lumicell Inc., 杜克大学 1.13.Anktiva (nogapendekin alfa inbakicept-pmln) 适应症：膀胱癌靶点：IL-15R 批准日期：2024年4月22日企业：Altor Bioscience Corp. 1.14.Ojemda (tovorafenib) 适应症：复发或难治性儿童低级别胶质瘤靶点:RAF 批准日期：2024年4月23日企业：Viracta Therapeutics Inc. 1.15.Xolremdi (mavorixafor) 适应症：治疗12岁及以上患有WHIM综合征的患者靶点：CXCR4 批准日期：2024年4月26日企业：赛诺菲 1.16.Imdelltra (tarlatamab-dlle) 适应症：广泛期小细胞肺癌靶点：CD3E、DLL3 批准日期：2024年5月16日企业：Amgen Inc. 1.17.Rytelo (imetelstat) 适应症：低危骨髓增生异常综合征伴输血依赖性贫血靶点：Telomerase 批准日期：2024年6月6日企业：Geron Corp. 1.18.Iqirvo (elafibranor) 适应症：原发性胆汁性胆管炎靶点：PPARɑ/γ 批准日期：2024年6月10日企业：Genfit SA 1.19.Sofdra (sofpironium) 适应症：原发性腋窝多汗症靶点：AChR 批准日期：2024年6月18日企业：Bodor Laboratories Inc. 1.20.Piasky (crovalimab-akkz) 适应症：阵发性睡眠性血红蛋白尿症靶点：C5 批准日期：2024年6月20日企业：Sino-Foreign Pharmaceutical Co., Ltd. 1.21.Ohtuvayre（Ensifentrine）适应症：治疗慢性阻塞性肺病靶点：PDE3/4 批准日期：2024年6月26日企业：Verona Pharma. 1.22.Kisunla（Donanemab-azbt）适应症：阿尔兹海默病靶点：pGlu3Aβ 批准日期：2024年7月2日企业：lilly 1.23.Leqselvi（Deuruxolitinib）适应症：严重斑秃靶点：JAK 批准日期：2024年7月25日企业：Sun Pharmaceuticals 1.24.Voranigo（vorasidenib）适应症：IDH1或IDH2易感突变的2级星形细胞瘤或少突胶质细胞瘤。靶点：IDH1、IDH2 批准日期：2024年8月6日企业：Servier Pharmaceuticals 1.25.Yorvipath（palopegteriparatide）适应症：甲状旁腺功能亢进批准日期：2024年8月9日企业：ascendis Pharma. 1.26.Nemluvio（nemolizumab-ilto）适应症：结节性痒疹靶点:IL-31 批准日期：2024年8月12日企业：Galderma Pharma. 1.27. Livdelzi（seladelpar）适应症：原发性胆汁性胆管炎靶点：PPAR-δ 批准日期：2024年8月14日企业：Gilead Sciences 1.28.Niktimvo（axatilimab-csfr）适应症：慢性移植物抗宿主病靶点CSF-1R 批准日期：2024年8月14日企业：Incyte/Syndax制药 1.29.Lazcluze（lazertinib）适应症：非小细胞肺癌靶点：EGFRm 批准日期：2024年8月19日企业：强生 1.30.Ebglyss（lebrikizumab-lbkz）适应症：中度至重度特应性皮炎靶点：IL-13 批准日期：2024年9月13日企业：lilly 1.31.Miplyffa（arimoclomol）适应症：C型尼曼-匹克病靶点：HSP 批准日期：2024年9月20日企业：Zevra Therapeutics 1.32.Aqneursa （levacetylleucine）适应症：儿童和成人C型尼曼匹克病批准日期：2024年9月24日企业：IntraBio. 1.33.Cobenfy（xanomeline and trospium chloride）适应症：成人精神分裂症靶点：M1-和M4-选择性毒蕈碱受体批准日期：2024年9月26日企业：百时美施贵宝 1.34.Flyrcado（flurpiridaz F 18）适应症：评估心脏血流阻塞（心肌缺血）和心脏病发作（心肌梗塞）批准日期：2024年9月27日企业：GE HealthCare 1.35.Tevimbra（Tislelizumab）适应症：不可切除或转移性食管鳞状细胞癌靶点：PD-1 批准日期：2024年10月4日企业：百济神州 02 2024年FDA批准的突出药物 Voranigo（vorasidenib）这种双重IDH1/IDH2抑制剂处理导致癌细胞代谢变化的突变。它被批准用于2级星形细胞瘤或少突胶质细胞瘤，这是一种生长缓慢的脑肿瘤，具有显著的风险。这是一个突破，因为它直接针对驱动肿瘤生长的突变，提供了一种更有效的治疗选择，可以延缓疾病进展，减少对辐射等侵入性治疗的依赖。 Niktimvo（Axatilimab）是一种CSF-1R单克隆抗体，通过限制巨噬细胞活性来治疗cGVHD，巨噬细胞在免疫应答中起着至关重要的作用。这种疗法对患有cGVHD的患者至关重要，这是干细胞移植后的一种严重并发症，通常对现有治疗无效。它提供了一种更有针对性的策略来改善患者的生活质量。 Cobenfy作为Xanomeline和Trospium Chloride这种联合治疗方法，通过调节毒蕈碱受体，为治疗精神分裂症带来了新的途径。通过平衡大脑中的神经递质活动，它解决了这种疾病的阳性和阴性症状，这使得它对那些用其他药物都没有成功的患者特别有价值。它的双重机制提供了更全面的症状管理，改善了那些受这种具有挑战性的精神健康状况影响的结果。 Rezdiffra（resmetirom）是一款甲状腺激素受体（THR）-β口服选择性激动剂，THR-β在肝脏中高度表达，能够调节脂代谢，降低LDL-C、甘油三酯和致动脉粥样硬化性脂蛋白。此外，THR-β还能通过促进脂肪酸的分解和刺激线粒体的生物发生来减少脂肪毒性并改善肝功能，从而减少肝脏脂肪。Rezdiffra用于治疗患有中重度肝纤维化（F2至F3期）的非肝硬化非酒精性脂肪性肝炎（NASH）患者，成为FDA批准的首款NASH疗法，具有里程碑意义。 Anktiva是一款IL-15超级激动剂。IL-15通过影响自然杀伤（NK）细胞和免疫T细胞的发育、维持和功能，在免疫系统中发挥着至关重要的作用。Anktiva由与IL-15受体α/IgG1 Fc融合蛋白结合的IL-15突变体（IL-15N72D）组成。它与βγ T细胞受体结合，直接特异性刺激CD8阳性T细胞和NK细胞，同时避免刺激调节性T细胞（Treg）。与天然、非复合IL-15相比，Anktiva在患者体内具有更优的药代动力学特性，且能在淋巴组织中存在更长时间，表现出增强的抗肿瘤活性。Anktiva在2019年获FDA授予突破性疗法认定，用于与BCG联合治疗此前对BCG应答不佳的非肌层浸润性原位膀胱癌患者。参考资料 [1]Novel Drug Approvals for 2024 | FDA [2]百济神州官网 BiG近期会议 10月26日，第三届生物计算大会保留栏目-第二届吐槽大会即将再度来袭！本届吐槽大会将围绕投融资、行业心态、年轻人，及AI等热门话题展开策划，通过移幕换景，大咖带新秀，让更多80-00后的行业亲历者来到聚光灯下，邀与会嘉宾一同探讨AI+Biomed的融合之道！共建Biomedical创新生态圈！如何加入BiG会员？

上市批准