更新于：2024-11-04

Danicopan

更新于：2024-11-04

概要

研发状态

概要

基本信息

药物类型

小分子化药

别名

(2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidine-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridine-2-yl)-4-fluoropyrrolidine-2-carboxamide、ACH-CFDIS、Danicopan (JAN/USAN/INN)

+ [5]

靶点

CFD

作用机制

CFD抑制剂(补体因子D抑制剂)

治疗领域

血液及淋巴系统疾病其他疾病遗传病与畸形+ [1]

在研适应症

阵发性睡眠性血红蛋白尿症溶血年龄相关性黄斑变性+ [1]

非在研适应症

C3肾小球病新型冠状病毒感染膜增生性肾小球肾炎+ [3]

原研机构

Alexion Pharmaceuticals, Inc.

在研机构

Alexion Pharmaceuticals, Inc.Alexion Pharma GmbHAlexion Pharma GK

+ [2]

非在研机构

Achillion Pharmaceuticals, Inc.National Institute of Allergy & Infectious Diseases

最高研发阶段批准上市

首次获批日期

日本 (2024-01-18),

阵发性睡眠性血红蛋白尿症

最高研发阶段(中国)-

特殊审评突破性疗法 (美国)、孤儿药 (美国)、孤儿药 (欧盟)、优先药物（PRIME） (欧盟)、孤儿药 (日本)、孤儿药 (韩国)、儿科研究计划 (欧盟)

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结构

分子式C26H23BrFN7O3

InChIKeyPIBARDGJJAGJAJ-NQIIRXRSSA-N

CAS号1903768-17-1

关联

项与 Danicopan 相关的临床试验

NCT06449001 / Not yet recruiting临床3期

A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis

The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).

开始日期2025-01-15

申办/合作机构

Alexion Pharmaceuticals, Inc.

NCT05708573 / Completed临床1期

A Phase 1 Study to Evaluate the Potential Drug Interaction Between ALXN2040 and Rosuvastatin in Healthy Adult Participants

This study will assess the potential drug-drug interaction (DDI) between ALXN2040 and rosuvastatin.

开始日期2023-02-01

申办/合作机构

Alexion Pharmaceuticals, Inc.

CTIS2023-504867-18-00 / Recruiting

A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated with Danicopan in an Alexion-sponsored Clinical Study. - ALXN2040-PNH-303

开始日期2023-01-05

申办/合作机构

Alexion Pharmaceuticals, Inc.

100 项与 Danicopan 相关的临床结果

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100 项与 Danicopan 相关的转化医学

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100 项与 Danicopan 相关的专利（医药）

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项与 Danicopan 相关的文献（医药）

2024-10-02·Expert Review of Clinical Pharmacology

Oral complement factor D inhibitor danicopan for paroxysmal nocturnal hemoglobinuria

Review

作者: Xu, Bo ; Zhou, Jiecan

INTRODUCTION:

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder characterized by episodic hemolysis, with additional clinical manifestations including thrombosis and bone marrow failure. The US FDA approved a complement factor D inhibitor, danicopan (Voydeya™), previously known as ACH-4471, for the treatment of extravascular hemolysis in adults with PNH on 29 March 2024. The primary purpose of this review is to examine the clinical efficacy and safety of danicopan.

AREAS COVERED:

We systematically searched for articles on PubMed, Web of Science, and three publishers Springer, Elsevier, Wiley up to 6 May 2024.

EXPERT OPINION:

Danicopan acts on the alternative pathway of the complement cascade, preferentially controlling C3 fragment-mediated extravascular hemolysis. Recommended dosage is 150 mg orally three times a day, which can be increased to 200 mg three times a day when necessary. Vaccination is required before administration to prevent infections by encapsulated bacteria. In a pivotal phase 3 trial ALPHA, danicopan significantly increased hemoglobin levels compared to placebo (p < 0.0001), 60% of patients experienced an increase in hemoglobin levels of at least 2 g/dL, compared to none in the placebo group (adjusted difference of 47%; p < 0.0001). Common adverse events during danicopan treatment include headache and upper respiratory tract infection.

2024-08-01·ANNALS OF HEMATOLOGY

Stable responses to danicopan as add-on to ravulizumab in two patients with paroxysmal nocturnal hemoglobinuria

Article

作者: Füreder, Wolfgang ; Valent, Peter

2024-06-02·CURRENT MEDICAL RESEARCH AND OPINION

Exploring treatment strategies for paroxysmal nocturnal hemoglobinuria: an overview of registered clinical trials

Review

作者: Vieira, Mónica ; Prudêncio, Cristina ; Peixoto, Vanda P.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disease in which blood cells lack anchored proteins that regulate the complement system. The erythrocytes are then destroyed because of uncontrolled complement activity, leading to intravascular hemolysis (IVH) and a high risk of thrombosis outcome. A huge alteration in the treatment of the disease was the development of terminal complement inhibitors, with the achievement of IVH blockade, reduction or abolishment of red blood cell (RBC) transfusions, and thromboembolic events prevention. However, patients treated with these inhibitors can still present extravascular hemolysis (EVH) caused by C3 activation and residual IVH or clinically relevant levels of breakthrough hemolysis (BTH). Proximal complement inhibitors turned out to be the key to the solution of this problem by targeting components of the proximal complement pathway, avoiding intra and extravascular hemolysis. FDA approved eculizumab, ravulizumab (terminal inhibitors), pegcetacoplan, iptacopan, and danicopan (proximal inhibitors) as a treatment for PNH so far. Various clinical trials are underway to find the most effective method to treat patients with PNH. This review aimed to summarize 71 registered clinical trials in the ClinicalTrials.gov database with the various treatment drugs, possible mechanisms, and novel findings related to PNH treatment.

项与 Danicopan 相关的新闻（医药）

2024-10-25

·PMLiVE

AstraZeneca’s Voydeya recommended by NICE to treat rare blood disorder PNH

AstraZeneca’s (AZ) Voydeya (danicopan) has been recommended by the National Institute for Health and Care Excellence (NICE) as an add-on therapy for adults with the rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH). The oral factor D inhibitor has been recommended for use alongside AZ’s complement component 5 (C5) inhibitors, Ultomiris (ravulizumab) or Soliris (eculizumab) to treat PNH patients who have residual haemolytic anaemia. The health technology assessment agency also specified that those eligible for the drug will have clinically significant extravascular haemolysis (EVH) while on treatment with a C5 inhibitor and that the company must provide the drug according to the commercial arrangement. PNH is caused by an acquired genetic mutation and results in the body’s immune system attacking its red blood cells. Patients can experience a range of symptoms, including blood clots, abdominal pain, difficulty swallowing, shortness of breath, excessive fatigue and anaemia. The symptoms and complications of the condition can be reduced by blocking the C5 protein. However, a subset of PNH patients who are treated with a C5 inhibitor experience clinically significant EVH, which results in continued symptoms of anaemia and may require blood transfusions. NICE’s decision on Voydeya was supported by positive results from the phase 3 ALPHA trial, which evaluated the efficacy and safety of the drug as an add-on to Ultomiris or Soliris in patients with PNH who experienced clinically significant EVH. Results showed that Voydeya met the primary endpoint of change in haemoglobin from baseline to week 12 as well as all key secondary endpoints, including transfusion avoidance and change in FACIT-Fatigue score, which measures patients’ level of fatigue during their usual daily activities. Deborah Richards, general manager of Alexion, AZ’s rare disease unit, said: “[Voydeya], a first-in-class, oral, factor D inhibitor, is the latest innovation in decades of work in our complement inhibition research programme. We’re committed to bettering the lives of all patients with PNH and we look forward to making this treatment available to eligible patients across the country.” The positive opinion comes six months after the European Commission approved Voydeya as an add-on therapy for adult PNH patients with residual haemolytic anaemia. The therapy has also been authorised for use in the US and Japan for certain adults with PNH.

临床结果上市批准临床3期

2024-09-02

·PRNewswire

Roche's PIASKY Approval for Paroxysmal Nocturnal Hemoglobinuria Treatment Will Give Tough Competition to AstraZeneca and Other Pharma Companies | DelveInsight

Following achievements in Asia, Roche's PNH treatment drug PIASKY (crovalimab) received its first FDA approval. This approval has spiked the competition between Roche and current market leader AstraZeneca. Additionally, the other pharma companies also geared up to launch their respective lead assets. LAS VEGAS, Sept. 2, 2024 /PRNewswire/ -- Paroxysmal nocturnal hemoglobinuria (PNH), a rare disorder, shows a triad of features: intravascular hemolysis, thromboembolic events, and cytopenia. Manifestations vary among patients, making classification based on typical presentations challenging due to the disease's unpredictable nature. In 2023, the Paroxysmal Nocturnal Hemoglobinuria diagnosed prevalent cases were ~12,000 cases in the 7MM, which might reach ~13,000 cases by 2034. In the 7MM, the highest number of paroxysmal nocturnal hemoglobinuria diagnosed prevalent cases were observed in the US. Until 2007, PNH was a devastating disease without treatment for hemolysis and thrombosis, the leading cause of death in patients. However, the last decade saw a revolutionary shift with the advent of the anti-C5 agent eculizumab. It significantly reduced hemolysis, decreased transfusion dependency, and, crucially, lowered the thrombosis rate. The disease-modifying therapeutic strategy for paroxysmal nocturnal hemoglobinuria includes complement inhibition therapy, with drugs like SOLIRIS, ULTOMIRIS, and EMPAVELI approved by the FDA, and considered the gold standard. SOLIRIS, the pioneering therapy for PNH, was succeeded by ULTOMIRIS, both developed by Alexion Pharmaceuticals, offering C5 inhibitors as essential therapeutic options. Recent approvals of factor B and D inhibitors like FABHALTA (iptacopan) in the US and VOYDEYA (danicopan) in Japan aim to offer improved treatment with fewer side effects. Learn more about the FDA-approved paroxysmal nocturnal hemoglobinuria drugs @ Drugs for Paroxysmal Nocturnal Hemoglobinuria Treatment The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended crovalimab for treating PNH in adults and adolescents aged 12 and above in June 2024, based on findings from the global Phase III COMMODORE 2 clinical trial. The FDA accepted a biologics license application for crovalimab in September 2023 and subsequently approved it in June 2024 for treating PNH in adults and adolescents aged 13 years and older. Genentech, a subsidiary of Roche, has made the drug available in the US. In Japan, Chugai Pharmaceutical submitted a new drug application for PiaSky to the Japan Ministry of Health, Labour, and Welfare (MHLW) in June 2023. The drug was approved in March 2024 and launched in May 2024 for patients with PNH aged 12 years and above. In February 2024, China's National Medical Products Administration (NMPA) approved crovalimab for treating PNH in adults and adolescents aged 12 years and older who have not previously received complement inhibitor treatment, following a priority review that began in August 2022. PiaSky is also undergoing review by other regulatory authorities, including those in Europe and Taiwan. To know more about paroxysmal nocturnal hemoglobinuria treatment options, visit @ New Treatment for Paroxysmal Nocturnal Hemoglobinuria The paroxysmal nocturnal hemoglobinuria market is crowded with so many companies working in the domain. Emerging PNH therapies include Pozelimab (REGN3918) + Cemdisiran, OMS906, NM8074 (ruxoprubart), and others, reflecting a dynamic evolution in treatment strategies. Discover which therapies are expected to grab major paroxysmal nocturnal hemoglobinuria market share @ Paroxysmal Nocturnal Hemoglobinuria Market Report Pozelimab is a novel, fully human monoclonal antibody currently under investigation. It aims to inhibit complement factor C5, thereby preventing the destruction of red blood cells (hemolysis) responsible for symptoms in conditions like Paroxysmal Nocturnal Haemoglobinuria (PNH) and other diseases influenced by complement pathway activity. This IgG4 antibody binds tightly to both wild-type and variant forms of human C5, effectively blocking its function. In its ongoing development, pozelimab is also being studied in combination with Alnylam's cemdisiran, a siRNA C5 inhibitor. This investigational combination therapy is aimed at treating various complement-mediated disorders, including PNH and myasthenia gravis. OMS-906 is currently being developed to treat paroxysmal nocturnal hemoglobinuria (PNH), complement 3 glomerulopathy (C3G), idiopathic immune complex-mediated glomerulonephritis (ICGN), and arthritis. It can be administered either subcutaneously or intravenously. This drug candidate is a monoclonal antibody designed to target mannan-binding lectin serine protease 3 (MASP-3) to exert its therapeutic effects. Discover more about drugs for paroxysmal nocturnal hemoglobinuria in development @ Paroxysmal Nocturnal Hemoglobinuria Clinical Trials The anticipated launch of these emerging therapies for paroxysmal nocturnal hemoglobinuria are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the paroxysmal nocturnal hemoglobinuria market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that in 2023, the total paroxysmal nocturnal hemoglobinuria market size in the 7MM was USD ~1.4 billion, which is expected to reach USD ~2.5 billion by 2034. This growth can be attributed to the advances in disease mechanisms that drive new diagnostics and therapeutics, fueling drug development. Additionally, the PNH market foresees positive growth, propelled by increasing cases and upcoming therapies in the forecast period. DelveInsight's latest published market report titled as Paroxysmal Nocturnal Hemoglobinuria Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the paroxysmal nocturnal hemoglobinuria country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The paroxysmal nocturnal hemoglobinuria market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Diagnosed Prevalent Cases of PNH Gender-specific Cases of PNH The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM paroxysmal nocturnal hemoglobinuria market. Highlights include: 11-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this paroxysmal nocturnal hemoglobinuria market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the paroxysmal nocturnal hemoglobinuria market. Also, stay abreast of the mitigating factors to improve your market position in the paroxysmal nocturnal hemoglobinuria therapeutic space. Related Reports Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted paroxysmal nocturnal hemoglobinuria epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Paroxysmal Nocturnal Hemoglobinuria Pipeline Paroxysmal Nocturnal Hemoglobinuria Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key paroxysmal nocturnal hemoglobinuria companies, including Biocad, AKARI Therapeutics, Amgen, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, among others. Aplastic Anemia Market Aplastic Anemia Market Insights, Epidemiology, and Market Forecast – 2034 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 aplastic anemia companies, including Pfizer, BioLineRx Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals, among others. Aplastic Anemia Pipeline Aplastic Anemia Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key aplastic anemia companies, including Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, among others. 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 . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP

上市批准优先审批临床3期

2024-07-08

·药时代

2024上半年，中美批准新药对比

2024年上半年，美国食品药品监督管理局（FDA）批准了21款1类新药，中国国家药品监督管理局（NMPA）批准了26款1类新药。本文将分别盘点中美获批的这些新药，并比较两国获批新药在药物类型、适应症等方面的异同。 21款新药FDA批准上市截至2024年6月底，美国食品药品监督管理局(FDA)药品评价研究中心(CDER)共批准了21种新药，包括17个化药和6个生物药，批准数量比2023年同期略低，但仍处于历史平均水平以上。图1. 美国FDA历年批准的新药数量数据来源：FDA官网、博药整理表1. 2024H1美国FDA CDER批准的新药商品名称活性成分适应症研发企业批准日期小分子 ZELSUVMI BERDAZIMER SODIUM 传染性软疣 LNHC 1/5/24 EXBLIFEP CEFEPIME;ENMETAZOBACTAM 复杂性尿路感染;肾盂肾炎 ALLECRA THERAPEUTICS SAS 2/22/24 REZDIFFRA RESMETIROM MASH MADRIGAL 3/14/24 TRYVIO APROCITENTAN 高血压 IDORSIA PHARMA LTD 3/19/24 DUVYZAT GIVINOSTAT 杜氏肌营养不良 ITALFARMACO SA 3/21/24 VAFSEO VADADUSTAT 慢性肾病引起的贫血 AKEBIA THERAPEUTICS INC 2024-03-27 VOYDEYA DANICOPAN 阵发性睡眠性血红蛋白尿症（PNH）阿斯利康 3/29/24 ZEVTERA CEFTOBIPROLE MEDOCARIL SODIUM 多种感染 BASILEA PHARM ALLSCH 4/3/24 OJEMDA TOVORAFENIB 儿童低级别胶质瘤 DAY ONE BIOPHARMACEUTICALS 4/23/24 XOLREMDI MAVORIXAFOR WHIM综合征 X4 PHARMACEUTICALS 4/26/24 IQIRVO ELAFIBRANOR 原发性胆汁性胆管炎（PBC） IPSEN BIOPHARM LTD 6/10/24 SOFDRA SOFPIRONIUM 原发性腋窝多汗症 BOTANIX SB INC. 6/18/24 OHTUVAYRE ENSIFENTRINE 慢性阻塞性肺病（COPD） Verona Pharma 6/26/24 显像剂 LUMISIGHT PEGULICIANINE 乳腺癌手术中辅助检测 LUMICELL 4/17/24 寡核苷酸 RYTELO IMETELSTAT SODIUM 骨髓增生异常综合征（MDS） GERON 6/6/24 单抗 TEVIMBRA TISLELIZUMAB替雷利珠单抗食管鳞状细胞癌百济神州 3/13/24 PIASKY CROVALIMAB-AKKZ 阵发性睡眠性血红蛋白尿症（PNH）罗氏 6/20/24 其他生物药 WINREVAIR SOTATERCEPT-CSRK 肺动脉高压默沙东 3/26/24 ANKTIVA Nogapendekin alfa inbakicept-pmln 非肌层浸润性膀胱癌 ALTOR BIOSCIENCE 4/22/24 IMDELLTRA TARLATAMAB-DLLE 非小细胞肺癌安进 5/16/24 LETYBO LETIBOTULINUMTOXINA-WLBG 成人中度至重度眉间纹 HUGEL INC 2/29/24 注：清单包括CDER批准的多种新药及部分生物制品，不包括疫苗、过敏性产品、血液和血液制品、血浆衍生物、细胞和基因治疗产品，以及生物制品评估和研究中心在2024年批准的其他产品。数据来源：FDA官网、博药整理 26款新药NMPA批准上市截至2024年6月底，中国国家药品监督管理局(NMPA)药品评价研究中心(CDE)共批准了26种新药，包括15个化药、8个生物药和3个中药，批准数量比2023年同期（24个）有一定增长。表2. 2024H1 NMPA获批上市的1类新药药品名称企业名称适应症批准日期小分子脯氨酸加格列净片惠升生物 2型糖尿病 1/16/24 富马酸泰吉利定注射液恒瑞医药腹部手术后中重度疼痛 1/30/24 妥拉美替尼胶囊科州药物黑色素瘤 3/12/24 盐酸伊普可泮胶囊诺华成人阵发性睡眠性血红蛋白尿症（PNH） 4/24/24 恩替司他片亿腾景昂 HR阳性、HER-2阴性，经内分泌治疗复发或进展的局部晚期或转移性乳腺癌 4/24/24 富马酸安奈克替尼胶囊正大天晴 ROS1阳性的局部晚期或转移性非小细胞肺癌 4/24/24 瑞普替尼胶囊 BMS ROS1阳性的局部晚期或转移性非小细胞肺癌 5/8/24 苯磺酸克利加巴林胶囊海思科糖尿病性周围神经病理性疼痛 5/15/24 甲磺酸瑞齐替尼胶囊倍而达药业既往经EGFR-TKI治疗时或治疗后出现疾病进展，并且经检测确认存在EGFR T790M 突变阳性的局部晚期或转移性非小细胞肺癌 5/15/24 枸橼酸依奉阿克胶囊正大天晴 ALK阳性的局部晚期或转移性非小细胞肺癌 6/11/24 甲磺酸瑞厄替尼片圣和药业 EGFR T790M突变阳性的局部晚期或转移性非小细胞肺癌 6/11/24 戈利昔替尼胶囊迪哲医药复发或难治的外周T细胞淋巴瘤 6/18/24 考格列汀片海思科 2型糖尿病 6/18/24 注射用舒巴坦钠/注射用度洛巴坦钠组合包装 Entasis Therapeutics/再鼎医药 18 岁及以上患者由鲍曼-醋酸钙不动杆菌复合体敏感分离株所致医院获得性细菌性肺炎（HABP）、呼吸机相关性细菌性肺炎（VABP） 5/15/24 多肽枸橼酸倍维巴肽注射液百奥泰急性冠脉综合征 6/25/24 依柯胰岛素注射液诺和诺德 2型糖尿病 6/18/24 单抗仑卡奈单抗注射液卫材/渤健阿尔茨海默病 1/5/24 可伐利单抗注射液罗氏阵发性睡眠性血红蛋白尿症 2/6/24 贝莫苏拜单抗注射液正大天晴广泛期小细胞肺癌 4/30/24 泽美洛韦玛佐瑞韦单抗注射液兴盟生物狂犬病病毒暴露者的被动免疫治疗 6/4/24 恩朗苏拜单抗注射液石药集团含铂方案化疗失败的PD-L1表达阳性的复发或转移性宫颈癌 6/25/24 双抗依沃西单抗注射液康方生物联合培美曲塞和卡铂，用于经EGFR-TKI治疗后进展的EGFR基因突变阳性的局部晚期或转移性非鳞状非小细胞肺癌 5/21/24 CAR-T 泽沃基奥仑赛注射液科济药业复发或难治性多发性骨髓瘤 2/23/24 中药儿茶上清丸齐进药业口腔溃疡 1/8/24 九味止咳口服液卓和药业急性气管-支气管炎中医辨证属风热证的咳嗽 2/20/24 秦威颗粒成都华西天然药物急性痛风性关节炎 3/12/24 数据来源：药智数据、药智头条整理中美异同适应症、药物类型对比分析从批准数量来看，今年上半年NMPA批准的新药有26个，比美国CDER批准的新药（21个）多近20%。如果剔除中国特色的中药和不属于CDER批准范畴的细胞疗法，则NMPA批准的新药数为22个，比美国CDER批准的新药多1个。从药物类型来看，无论是中国还是美国，小分子都是获批新药的主要类型，占比均超过一半。其次为发展较为成熟的抗体药物。另外，新型药物如双特异性T细胞接合器、双抗、CAR-T疗法中美各有涉猎，但数量不多。图2. 2024上半年美国CDER批准新药药物类型分布数据来源：FDA官网、博药整理图3. 2024上半年NMPA批准新药药物类型分布数据来源：药智数据、博药整理从某种程度上而言，在研药物领域的创新技术与获批上市的药品技术存在一定时间差，目前较热门ADC、CAR-T等领域在新药获批层面上还未体现，而从目前新药研发领域由小分子药向生物药转变的趋势上来看，后续生物药新药获批数量或许会越来越高。从适应症来看，中美获批的新药中都是抗肿瘤新药最多，但是中国占比更高，接近50%，而美国为29%。从具体癌种来看，中国获批的12个肿瘤新药中有7个用于肺癌（6个非小细胞肺癌、1个小细胞肺癌）治疗，血液瘤2个，宫颈癌、乳腺癌、黑色素瘤各1个。而在美国获批的肿瘤新药中癌种较分散，包括食管癌、乳腺癌、胶质瘤等。这一系列中美差异点，或许正是国内新药同质化的原因之一，拥抱热门不可厚非，但强调差异化发展，如艾伯维一般进行建立核心疾病领域优势，对于国内创新药企业或许更值得学习。其次，抗感染、血液疾病、代谢性疾病、心血管疾病用药也是新药研发的热门领域。另外，罕见病作为美国新药开发和获批的重点领域，2024上半年获批的新药中有10个新药曾获得孤儿药认证，包括4个罕见肿瘤用药，2个血液罕见病用药，以及4个其他罕见病用药。而在中国，2024上半年获批上市的1类新药中，仅有2个进口1类罕见病新药获批用于治疗成人阵发性睡眠性血红蛋白尿症（PNH）患者，尚未出现国产自主研发的罕见病新药。当然，还有部分罕见病新药以5.1类新药申报并获批进口，不在本文统计范围内。这一点上，或许某种程度上也意味着中国新药“源头创新”意愿的不足，对于罕见病等临床上需求迫切的领域，反而因为市场空间、技术难度等问题而推进缓慢，这一点上或许有待加强。图4. 2024上半年美国CDER批准新药疾病领域分布数据来源：FDA官网、博药整理图5. 2024上半年NMPA批准新药疾病领域分布数据来源：药智数据、博药整理从企业来看，罗氏（Roche）开发的新一代C5循环抗体Piasky（crovalimab，可伐利单抗）同时在中美获批上市，用以治疗阵发性睡眠性血红蛋白尿症（PNH）患者。美国FDA批准的新药少部分来自跨国大药企，如默沙东、阿斯利康、安进等，多数来自美国的生物技术公司，例如MADRIGAL、AKEBIA、ALTOR BIOSCIENCE等。 NMPA批准的新药同样少部分来自跨国大药企，如BMS、诺华等，多数来自中国的药企和生物技术公司，例如恒瑞医药、石药集团、正大天晴、倍而达药业、科济药业、康方生物等。从审评方式来看，2024上半年美国有10个新药通过快速审批方式获得批准，占所有获批新药的48%；中国有9个新药通过优先审批获得批准，占所有获批新药的35%，可见快速审批、优先审批已是新药获得批准的重要途径。小结近年来，在特别审批、突破性治疗药物、附条件批准、优先审评审批等药品加快上市注册程序下，NMPA批准新药数量快速增长。2022年NMPA批准了23个1类新药，2023年这一数字增长到40个，而今年上半年就已批准了26个1类新药，超过美国FDA批准的1类新药数量，今年全年批准新药数量有望再创新高。但是，在适应症方面，国内新药主要集中在抗肿瘤领域，尤其是肺癌领域。而FDA批准的新药适应症罕见病和罕见肿瘤占据很大比例。这与国内外市场环境差异、创新药发展历程长短有关。目前而言，国外市场更鼓励去探索一些更新的靶点和适应症，对于比较新的靶点，药企也偏向于出海去寻找机会。未来，相信随着政策的支持、支付环境的改善，国内也有望迎来更多创新疗法。资料来源：美国FDA官网 NMPA官网药智数据各企业公开资料封面图来源：pixabay 版权声明/免责声明本文为授权转载文章。本文仅作信息交流之目的，不提供任何商用、医用、投资用建议。文中图片、视频、字体、音乐等素材或为药时代购买的授权正版作品，或来自微信公共图片库，或取自公司官网/网络，部分素材根据CC0协议使用，版权归拥有者，药时代尽力注明来源。如有任何问题，请与我们联系。衷心感谢! 药时代官方网站:www.drugtimes.cn 联系方式: 电话:13651980212 微信:27674131 邮箱:contact@drugtimes.cn 罗氏TIGIT再败肺癌后，决定提前终止这一研究一家CNS领域 biotech 的红与黑 mRNA CAR-T二期临床试验成功，公司股价却暴跌35%！点击这里，发现价值信息

上市批准寡核苷酸疫苗核酸药物临床研究

100 项与 Danicopan 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11641	-	-	DB15401

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
阵发性睡眠性血红蛋白尿症	日本	Alexion Pharmaceuticals, Inc.	2024-01-18
阵发性睡眠性血红蛋白尿症	日本	Alexion Pharma GK	2024-01-18

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
溶血	临床3期	-	Alexion Pharmaceuticals, Inc.	2025-01-15
年龄相关性黄斑变性	临床2期	美国	Alexion Pharmaceuticals, Inc.	2021-08-23
年龄相关性黄斑变性	临床2期	日本	Alexion Pharmaceuticals, Inc.	2021-08-23
年龄相关性黄斑变性	临床2期	澳大利亚	Alexion Pharmaceuticals, Inc.	2021-08-23
年龄相关性黄斑变性	临床2期	捷克	Alexion Pharmaceuticals, Inc.	2021-08-23
年龄相关性黄斑变性	临床2期	法国	Alexion Pharmaceuticals, Inc.	2021-08-23
年龄相关性黄斑变性	临床2期	德国	Alexion Pharmaceuticals, Inc.	2021-08-23
年龄相关性黄斑变性	临床2期	匈牙利	Alexion Pharmaceuticals, Inc.	2021-08-23
年龄相关性黄斑变性	临床2期	意大利	Alexion Pharmaceuticals, Inc.	2021-08-23
年龄相关性黄斑变性	临床2期	拉脱维亚	Alexion Pharmaceuticals, Inc.	2021-08-23

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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


NCT04469465 (ALPHA, EHA2024) 人工标引	临床3期	阵发性睡眠性血红蛋白尿症	86	Danicopan + Ravulizumab/Eculizumab	選製範製糧夢蓋積鹽築(繭觸築膚鏇廠網範遞築) = The proportion of pts with increased Hb (≥2 g/dL) without transfusion was maintained in the Dan–Dan arm and improved in the Pbo–Dan arm from wk 12 to 24 (Dan–Dan, 54.4% to 41.8%; Pbo–Dan, 0% to 33.3%). At wks 24 and 48, mean Hb levels and ARC were maintained in the Dan–Dan arm andimproved from wk 12 in the Pbo–Dan arm 壓觸顧醖淵廠襯製築醖 (構蓋願鬱積網廠糧積蓋 ) 更多	积极	2024-05-14
NCT04469465 (ALXN2040-PNH-301, FDA_CDER) 人工标引	临床3期	阵发性睡眠性血红蛋白尿症	86	VOYDEYA + ravulizumab or eculizumab	構製膚鏇憲憲鑰範壓淵(選壓窪觸糧製顧鏇廠衊) = 簾齋鑰顧鬱醖製壓襯齋壓鏇膚襯憲鑰願膚獵願 (膚廠壓顧願構願鑰積鏇 ) 更多	积极	2024-03-30
				Placebo + ravulizumab or eculizumab	構製膚鏇憲憲鑰範壓淵(選壓窪觸糧製顧鏇廠衊) = 壓鹽壓齋構網鹹鬱網壓壓鏇膚襯憲鑰願膚獵願 (膚廠壓顧願構願鑰積鏇 ) 更多
ALPHA (BusinessWire) 人工标引	临床3期	阵发性睡眠性血红蛋白尿症	86	Danicopan + ULTOMIRIS or SOLIRIS (Danicopan-Danicopan)	窪鹹遞遞鹽構夢夢蓋構(遞齋築繭觸襯憲鏇糧鏇) = 鏇襯構醖艱遞淵繭選糧顧廠鬱網襯獵範獵遞夢 (壓選膚願衊簾願鬱積獵, 0.21) 更多	积极	2023-12-10
				Placebo danicopanULTOMIRIS or SOLIRIS (placebo-danicopan)	窪鹹遞遞鹽構夢夢蓋構(遞齋築繭觸襯憲鏇糧鏇) = 憲鑰選淵顧獵簾蓋衊齋顧廠鬱網襯獵範獵遞夢 (壓選膚願衊簾願鬱積獵, 0.31) 更多
NCT04988035 (CTgov)	临床2期	新型冠状病毒感染	201	Danicopan+Remdesivir (Remdesivir + Danicopan)	窪鹽鏇鹹範糧鹽憲壓鑰(壓壓鏇構糧積衊衊繭網): Odds Ratio (OR) = 0.64 (95% CI, 0.38 ~ 1.07), P-Value = 0.090 更多	-	2023-06-08
				Placebo+Remdesivir (Remdesivir + Placebo)
NCT03181633 (CTgov)	临床2期	阵发性睡眠性血红蛋白尿症	8	danicopan	願淵繭網製獵觸鹽衊膚(醖醖窪遞壓膚餘製簾鏇) = 鑰網觸積夢製鹹遞襯齋齋積獵構夢夢鏇製構鹹 (壓鹽觸獵壓齋觸鑰廠繭, 繭夢鹽廠廠糧淵構鏇製 ~ 鬱窪餘廠餘壓製鏇願顧) 更多	-	2023-03-14
NCT03459443 (CTgov)	临床2期	膜增生性肾小球肾炎 \| C3肾小球病	22	Danicopan	繭顧齋顧醖顧選糧鑰製(遞憲顧願構鏇選淵淵衊) = 鹹憲鹹蓋網鏇憲鬱觸繭窪鹽蓋廠艱襯餘網遞夢 (襯醖鬱鬱餘獵鬱製網淵, 餘餘簾糧積獵淵憲艱觸 ~ 遞鹽餘窪獵艱繭簾淵襯) 更多	-	2022-08-11
NCT03472885 (Pubmed) 人工标引	临床2期	阵发性睡眠性血红蛋白尿症	12	Danicopan	襯繭範製獵觸憲醖衊餘(憲膚糧鹹襯夢觸鑰鏇網) = 鹹築構膚獵膚淵窪選餘餘鏇獵製鬱鑰獵簾窪糧 (鏇鬱餘鬱鑰繭積簾築憲 ) 更多	积极	2021-11-18
NCT03369236 (CTgov)	临床2期	膜增生性肾小球肾炎 \| C3肾小球病	13	Placebo+Danicopan (Placebo (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period))	齋構艱願夢衊選願蓋鏇(衊壓壓襯壓糧窪鬱艱鏇) = 觸廠鑰範醖衊憲膚顧醖觸衊衊餘觸製簾簾範壓 (製積廠淵襯構積製選顧, 廠築鬱構膚廠築繭鹹觸 ~ 築窪觸繭衊膚糧鹽鏇窪) 更多	-	2021-11-04
				Danicopan (Danicopan (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period))	鬱簾觸繭鑰齋糧製壓齋(願夢範鹽構餘築壓鏇齋) = 構願憲憲簾鏇淵淵艱糧觸網構鬱窪願糧顧顧夢 (繭構願網觸膚構襯選齋, 蓋廠鹹窪鏇顧鏇網鬱遞 ~ 觸鏇簾鹽齋觸顧壓襯艱)
NCT03124368 (CTgov)	临床2期	膜增生性肾小球肾炎 \| C3肾小球病	6	Danicopan (Group 1: Danicopan 100 mg TID (Sentinel))	觸選醖遞觸顧積網範築(願膚選蓋築夢膚鹹壓鹽) = 範壓願願齋範願壓膚網淵糧鬱鬱廠襯艱醖遞餘 (願製觸醖繭顧繭鹽簾簾, 範簾築蓋糧膚壓鹽鬱鑰 ~ 餘廠範襯構鏇餘蓋齋簾) 更多	-	2021-07-13
				Danicopan (Group 2: Danicopan up to 200 mg TID)	觸選醖遞觸顧積網範築(願膚選蓋築夢膚鹹壓鹽) = 繭鹹壓構衊簾築範齋鹹淵糧鬱鬱廠襯艱醖遞餘 (願製觸醖繭顧繭鹽簾簾, 襯鏇齋構壓襯壓範製衊 ~ 膚醖選鏇鹹醖獵淵鏇簾) 更多
NCT03472885 (PNH, CTgov)	临床2期	阵发性睡眠性血红蛋白尿症	12	Danicopan+Eculizumab (Danicopan + Eculizumab)	鹽鏇齋鬱襯鬱艱鬱網艱(遞繭製廠餘鹹繭繭醖網) = 窪鏇獵網鏇蓋鹽膚艱觸網顧壓獵觸憲齋鹽鹽艱 (願觸鑰願衊醖鑰簾醖鏇, 簾網觸壓壓積艱襯醖構 ~ 範範範遞願繭憲範艱鹽) 更多	-	2021-06-25
				Danicopan+Eculizumab (24-Week Treatment Period: Danicopan + Eculizumab)	蓋夢蓋窪襯憲淵觸衊廠(鹹遞糧觸鬱衊鑰膚顧衊) = 膚積繭壓鏇醖糧鑰憲蓋齋襯壓齋糧膚壓壓鹹遞 (遞艱築齋築鬱醖觸顧構, 糧齋夢襯遞願積壓積壓 ~ 窪觸鏇獵夢積淵鹽選繭) 更多