A Randomized Phase 2/3 Study Evaluating the Safety and Efficacy of Pivekimab Sunirine (PVEK) in Combination With Venetoclax and Azacitidine in Adult Subjects With Newly Diagnosed Acute Myeloid Leukemia (AML) Ineligible to Receive Intensive Chemotherapy

Cancer is a condition where cells in a specific part of the body grow and reproduce uncontrollably. Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow (the spongy tissue inside the bones) that affects white blood cells that helps to fight infections and also prevents normal blood cell production. This study will assess the adverse events and changes in the disease activity when Pivekimab Sunirine (PVEK) is given in combination with Venetoclax (VEN) and Azacitidene (AZA) in adult participants with AML ineligible to receive intensive chemotherapy.
Pivekimab sunirine is a drug being evaluated in the treatment of AML.This is a Phase 2/Phase 3, study of PVEK. Phase 2 is open-label and randomized. Phase 3 is double-blind, randomized. Phase 2 and Phase 3 studies test potential new treatments in patients with a condition or disease. Open-label means that both patients and study doctors know which study treatment is given to patients in Phase 2 of the study. Double-blind means that neither the patients nor the study doctors know who is given which study treatment in Phase 3 of the study. Approximately 660 adult participants will be enrolled in 180 sites worldwide.
In Phase 2 of the study, patients will be randomized to receive PVEK + VEN + AZA or standard of care treatment with VEN + AZA. In Phase 3, patients will be randomized to receive PVEK + VEN + AZA or a matching-placebo for PVEK plus VEN + AZA. PVEK is given as an infusion into the vein, AZA is given as an injection under your skin (subcutaneous) or as an infusion into the vein (intravenous) (depending on country where patient enrolls), and VEN is a tablet given by mouth. The total study duration is approximately 71 months.
There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, and checking for side effects.

开始日期2026-07-22

申办/合作机构

AbbVie, Inc.

NCT07306832

/ Recruiting临床1期

A Phase 1b Study of the Safety and Pharmacokinetics of Pivekimab Sunirine in Pediatric Subjects With Relapsed or Refractory Acute Myeloid Leukemia (AML)

Acute myeloid leukemia (AML) is an aggressive blood cancer, withwith few options for participants who relapse after treatment or who don't respond to treatment. This study will assess the adverse events and how pivekimab sunirine moves through the body in pediatric participants with relapsed or refractory (R/R) AML.
Pivekimab sunirine is a drug being evaluated in the treatment of AML. This is an open label, single arm study, participants will be enrolled in 1 of the 3 cohorts based on their age and will receive pivekimab sunirine at a dose based on their weight. Around 18 pediatric participants with a diagnosis of AML will be enrolled in the study at approximately 30 sites around the world.
Participants will receive intravenous (IV) pivekimab sunirine alone. The total study duration is approximately 28 months.
There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, and checking for side effects.

开始日期2026-05-20

申办/合作机构

AbbVie, Inc.

NCT06034470

/ Active, not recruiting临床1期IIT

Phase 1 Study of FLAG-Ida With Pivekimab Sunirine (PVEK) for Adults With Newly Diagnosed Adverse-Risk Acute Myeloid Leukemia and Other High-Grade Myeloid Neoplasms

This phase I trial finds the best dose of PVEK when given together with fludarabine, cytarabine, granulocyte colony-stimulating factor (G-CSF), and idarubicin, (FLAG-Ida) regimen and studies the effectiveness of this combination therapy in treating patients with newly diagnosed adverse risk acute myeloid leukemia (AML) and other high-grade myeloid neoplasms. PVEK is a monoclonal antibody linked to a chemotherapy drug. PVEK is a form of targeted therapy because it attaches to specific molecules (receptors) on the surface of cancer cells, known as CD123 receptors, and delivers the chemotherapy drug to kill them. Chemotherapy drugs, such as idarubicin, fludarabine, high-dose cytarabine work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. G-CSF helps the bone marrow make more white blood cells in patients with low white blood cell count due to cancer treatment. Giving PVEK with the FLAG-Ida regimen may be a safe and effective treatment for patients with acute myeloid leukemia and other high-grade myeloid neoplasms.

开始日期2023-12-18

申办/合作机构

Fred Hutchinson Cancer Research Center

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100 项与 Pivekimab Sunirine 相关的专利（医药）

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项与 Pivekimab Sunirine 相关的文献（医药）

2025-12-02·EXPERT OPINION ON BIOLOGICAL THERAPY

Current status and future potential of CD123-based targeted therapies for acute leukemia

Review

作者: Aldoss, Ibrahim ; Arslan, Shukaib ; Stein, Anthony ; Aribi, Ahmed

INTRODUCTION:

Acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and blastic plasmacytoid dendritic cell neoplasm (BPDCN) are aggressive hematologic malignancies with poor outcomes, particularly in relapsed/refractory settings. CD123, the interleukin-3 receptor alpha chain, is highly expressed on leukemic blasts and leukemia stem cells, making it an attractive therapeutic target with limited expression on normal hematopoietic cells. CD123-directed therapies - including antibody-drug conjugates (ADCs), bispecific T-cell engagers (BiTEs), and CAR T-cell therapies - are under active investigation.

AREAS COVERED:

This review details the preclinical rationale, clinical development, efficacy, and toxicity of CD123-targeted therapies. It discusses completed and ongoing clinical trials for agents such as tagraxofusp, pivekimab sunirine, flotetuzumab, vibecotamab, and investigational CAR T-cell therapies (e.g. MB-102, UCART123v1.2). Toxicities, including myelosuppression, cytokine release syndrome (CRS), and hepatotoxicity - are characterized in detail, with attention to mitigation strategies. Future directions highlight combination regimens, switchable CAR designs, and biomarker-driven personalization.

EXPERT OPINION:

CD123-targeted therapy represents a promising yet challenging frontier in treating high-risk leukemias. While clinical activity has been demonstrated, especially in BPDCN, therapeutic durability and safety remain hurdles. Precision in antigen targeting, combination strategies, and toxicity management will be critical for successful integration of CD123-directed therapies into standard clinical practice.

2025-01-01·HemaSphere

The CD123 antibody–drug conjugate pivekimab sunirine exerts profound activity in preclinical models of pediatric acute lymphoblastic leukemia

Article

作者: Lock, Richard B. ; Bult, Carol J. ; Jocoy, Emily L. ; Mohamed, Sara M. A. ; Gifford, Andrew J. ; Evans, Kathryn ; Zweidler‐McKay, Patrick A. ; Philip, Vivek M. ; Lakshmikanthan, Sribalaji ; Neuhauser, Steven ; Smith, Malcolm A. ; Watts, Ben ; Earley, Eric J. ; Erickson, Stephen W. ; Smith, Christopher M. ; Stearns, Timothy M. ; Teicher, Beverly A. ; Chuang, Jeffrey H.

Abstract:

Antibody–drug conjugates (ADCs) combining monoclonal antibodies with cytotoxic payloads are a rapidly emerging class of immune‐based therapeutics with the potential to improve the treatment of cancer, including children with relapse/refractory acute lymphoblastic leukemia (ALL). CD123, the α subunit of the interleukin‐3 receptor, is overexpressed in ALL and is a potential therapeutic target. Here, we show that pivekimab sunirine (PVEK), a recently developed ADC comprising the CD123‐targeting antibody, G4723A, and the cytotoxic payload, DGN549, was highly effective in vivo against a large panel of pediatric ALL patient‐derived xenograft (PDX) models (n = 39). PVEK administered once weekly for 3 weeks resulted in a median event‐free survival (EFS) of 57.2 days across all PDXs. CD123 mRNA and protein expression was significantly higher in B‐lineage (n = 65) compared with T‐lineage (n = 25) ALL PDXs (p < 0.0001), and mice engrafted with B‐lineage PDXs achieved significantly longer EFS than those engrafted with T‐lineage PDXs (p < 0.0001). PVEK treatment also resulted in significant clearance of human leukemia cells in hematolymphoid organs in mice engrafted with B‐ALL PDXs. Notably, our results showed no direct correlation between CD123 expression and mouse EFS, indicating that CD123 is necessary but not sufficient for in vivo PVEK activity. Importantly, a PDX with very high CD123 cell surface expression but resistant to in vivo PVEK treatment, failed to internalize the G4723A antibody while remaining sensitive to the PVEK payload, DGN549, suggesting a novel mechanism of resistance. In conclusion, PVEK was highly effective against a large panel of B‐ALL PDXs supporting its clinical translation for B‐lineage pediatric ALL.

2024-03-01·The Lancet. Oncology

Pivekimab sunirine (IMGN632), a novel CD123-targeting antibody–drug conjugate, in relapsed or refractory acute myeloid leukaemia: a phase 1/2 study

Article

作者: Lane, Andrew A ; Zweidler-McKay, Patrick A ; Du, Yining ; Todisco, Elisabetta ; Wang, Eunice S ; Pemmaraju, Naveen ; Konopleva, Marina Y ; DeAngelo, Daniel J ; Papadantonakis, Nikolaos ; Malcolm, Kara E ; Thompson, James E ; Kantarjian, Hagop M ; Montesinos, Pau ; Sloss, Callum M ; Bedse, Gaurav ; Sweet, Kendra L ; Daver, Naval G ; Torres-Miñana, Laura ; Watkins, Krystal

BACKGROUND:

Pivekimab sunirine (IMGN632) is a first-in-class antibody-drug conjugate comprising a high-affinity CD123 antibody, cleavable linker, and novel indolinobenzodiazepine pseudodimer payload. CD123 is overexpressed in several haematological malignancies, including acute myeloid leukaemia. We present clinical data on pivekimab sunirine in relapsed or refractory acute myeloid leukaemia.

METHODS:

This first-in-human, phase 1/2 dose-escalation and dose-expansion study enrolled participants aged 18 years or older at nine hospitals in France, Italy, Spain, and the USA with CD123+ haematological malignancies (Eastern Cooperative Oncology Group performance status of 0-1); participants reported here were in a cohort of participants with acute myeloid leukaemia who were refractory to or had relapsed on one or more previous treatments for acute myeloid leukaemia. The 3 + 3 dose-escalation phase evaluated two dosing schedules: schedule A (once every 3 weeks, on day 1 of a 3-week cycle) and fractionated schedule B (days 1, 4, and 8 of a 3-week cycle). The dose-expansion phase evaluated two cohorts: one cohort given 0·045 mg/kg of bodyweight (schedule A) and one cohort given 0·090 mg/kg of bodyweight (schedule A). The primary endpoints were the maximum tolerated dose and the recommended phase 2 dose. Antileukaemia activity (overall response and a composite complete remission assessment) was a secondary endpoint. The study is ongoing and registered with ClinicalTrials.gov, NCT03386513.

FINDINGS:

Between Dec 29, 2017, and May 27, 2020, 91 participants were enrolled (schedule A, n=68; schedule B, n=23). 30 (44%) of schedule A participants were female and 38 (56%) were male; 60 (88%) were White, six (9%) were Black or African American, and two (3%) were other races. Pivekimab sunirine at doses of 0·015 mg/kg to 0·450 mg/kg in schedule A was administered in six escalating doses with no maximum tolerated dose defined; three dose-limiting toxicities were observed (reversible veno-occlusive disease; 0·180 mg/kg, n=1 and 0·450 mg/kg, n=1; and neutropenia; 0·300 mg/kg, n=1). Schedule B was not pursued further on the basis of comparative safety and antileukaemia findings with schedule A. The recommended phase 2 dose was selected as 0·045 mg/kg once every 3 weeks. At the recommended phase 2 dose (n=29), the most common grade 3 or worse treatment-related adverse events were febrile neutropenia (three [10%]), infusion-related reactions (two [7%]), and anaemia (two [7%]). Treatment-related serious adverse events occurring in 5% or more of participants treated at the recommended phase 2 dose were febrile neutropenia (two [7%]) and infusion-related reactions (two [7%]). Among 68 participants who received schedule A, one death (1%) was considered to be treatment-related (cause unknown; 0·300 mg/kg cohort). At the recommended phase 2 dose, the overall response rate was 21% (95% CI 8-40; six of 29) and the composite complete remission rate was 17% (95% CI 6-36; five of 29).

INTERPRETATION:

Pivekimab sunirine showed single-agent activity across multiple doses, with a recommended phase 2 dose of 0·045 mg/kg once every 3 weeks. These findings led to a phase 1b/2 study of pivekimab sunirine plus azacitidine and venetoclax in patients with CD123-positive acute myeloid leukaemia.

FUNDING:

ImmunoGen.

105

项与 Pivekimab Sunirine 相关的新闻（医药）

2026-06-12

·药事纵横

全球首款CD123 ADC获批：为罕见血液肿瘤撕开治疗缺口

当地时间2026年5月27日，美国食品药品监督管理局（FDA）批准艾伯维药物pivekimab sunirine-pvzy（商品名：Decnupaz）上市，适应症为成人母细胞性浆细胞样树突状细胞肿瘤（BPDCN）。这是全球首款靶向CD123的抗体药物偶联物（ADC），同时也是艾伯维首款落地血液肿瘤领域的ADC 产品。该药物的上市，为临床治疗BPDCN提供了全新的靶向方案，也进一步丰富了全球ADC药物管线与罕见血液肿瘤治疗选择。图1. 美国FDA批准艾伯维药物pivekimab sunirine-pvzy上市一、BPDCN：临床治疗长期存在明显短板母细胞性浆细胞样树突状细胞肿瘤属于罕见高侵袭性血液恶性肿瘤，整体发病人数少，但疾病进展速度快，临床预后普遍较差，患病群体以中老年男性为主。该疾病初期临床表现缺乏特异性，多数患者首发症状集中在皮肤层面，常见紫红色皮损、皮下结节以及不明原因瘀斑，很容易被误诊为普通皮肤疾病。随着病情进展，肿瘤细胞会逐步侵犯骨髓、淋巴结，后期还可累及中枢神经系统，继而引发贫血、出血、反复感染以及各类神经系统症状。在这款新药获批之前，临床针对BPDCN并没有统一、规范的标准治疗方案。临床主流手段为高强度化疗，短期能够控制肿瘤进展，但复发问题十分突出，化疗后复发率超过70%。患者一旦出现复发，后续可选择的治疗方式极为有限。受限于病例数量少、研发投入回报周期长等因素，针对BPDCN的新药研发进展缓慢，不少患者确诊后生存期不足一年，临床对于精准靶向药物的需求十分迫切。 CD123作为白介素3受体α亚基，在BPDCN肿瘤细胞表面呈高表达状态，而在正常造血细胞中表达水平极低，具备靶向治疗的先天优势。pivekimab sunirine-pvzy正是依托ADC技术，以CD123为作用靶点，实现对肿瘤细胞的定向杀伤。图2. pivekimab sunirine-pvzy结构示意图二、CADENZA 临床研究：支撑药物顺利获批本次药物获批的核心依据，来自一项I/II期多中心、开放标签临床研究CADENZA，相关研究数据已发表于《Journal of Clinical Oncology》。研究共纳入84例CD123阳性血液恶性肿瘤患者，按照患病状态分为两组：33例为未累及中枢神经系统的初治BPDCN患者，另外 51 例为无活动性中枢病变的复发/难治性BPDCN患者，分层验证了药物在不同人群中的疗效与安全性。图3. 文章发表（一）初治患者：高缓解率助力造血干细胞移植 33 例初治患者接受治疗后，综合完全缓解率达到69.7%，大部分患者肿瘤病灶得到有效控制并达到临床缓解标准。患者中位缓解持续时间为9.7个月。在完成试验随访后，有13例患者顺利接受异基因造血干细胞移植。目前造血干细胞移植是临床唯一有机会实现BPDCN 临床治愈的手段，该药物带来的高缓解状态，为患者争取到了接受移植的窗口期。图4. 初治患者疗效结果（二）复发/难治患者：改善后线治疗困境 51例复发、难治性患者大多经历过多线化疗，肿瘤已产生耐药性，治疗难度远高于初治人群。本组患者综合完全缓解率为15.7%，中位缓解持续时间9.2个月，其中6例患者在治疗后成功接受造血干细胞移植。对比传统化疗在后线治疗中几乎无效的现状，该药物能够让部分晚期患者实现病情缓解，打破了复发后无有效治疗方案的局面。图5. 复发/难治患者疗效结果（三）安全性特征：不良反应可控，需重点监测肝损伤从整体安全性数据来看，该药物的不良反应表现与同类ADC产品基本一致，整体风险可控。发生率超过20% 的常见不良反应包括外周水肿、乏力、输注相关反应、肌肉骨骼疼痛、恶心等，这类反应多为1-2级轻度症状，经过对症处理后均可缓解。治疗过程中出现的3级及以上严重不良反应，以中性粒细胞减少、血小板减少、外周水肿为主；严重感染问题也需要临床重点关注，其中肺炎、发热性中性粒细胞减少均有一定发生比例。该药物附带黑框警告，核心风险为肝毒性，用药后可能出现重度肝损伤，甚至危及生命的肝静脉闭塞病。因此临床要求，每次给药前以及整个治疗周期内，都需要持续监测患者肝功能指标。综合评估，药物治疗收益高于潜在风险。三、药物上市带来的行业影响（一）完善艾伯维ADC产品布局 2023年11月，艾伯维完成对ImmunoGen的收购，交易金额101亿美元，借此拿下pivekimab sunirine-pvzy以及FRα靶向ADC药物Elahere等核心资产。此前Elahere已在实体瘤领域完成商业化，2025年实现销售额6.9亿美元。本次CD123靶向ADC获批上市，标志着艾伯维形成实体瘤+血液肿瘤双赛道ADC产品矩阵，整体管线布局进一步完善。（二）CD123靶点完成临床落地验证 CD123是血液肿瘤领域长期受到关注的热门靶点，此前行业内已布局双特异性抗体、CAR-T 细胞疗法、DAC等多种技术路线产品，但始终没有实现突破性商业化。pivekimab sunirine-pvzy 的上市，首次证实CD123靶点具备临床成药价值，完成了从靶点研究到商业化药物落地的转化。现阶段CD123赛道已形成多技术路线并行的格局，ADC、双特异性T细胞衔接器、抗体偶联降解剂、毒素融合蛋白等产品均在持续推进。需要区分的是，目前公开报道中CD123相关耐药机制，大多来源于双特异性T细胞衔接器产品，和这款ADC药物的耐药原因并不相同。ADC药物耐药多和肿瘤细胞抗原表达量、药物内吞效率、细胞内载荷外排等因素相关。不过两类产品的研发经验可以相互参考，单一药物方案容易出现肿瘤逃逸，联合用药、更换作用靶点，是目前业内公认的解决方向。（三）罕见病成为ADC研发新方向截至2026年5月，全球已获批ADC药物数量稳步增长，靶点覆盖HER2、TROP2、CD22、CD123等，适应症也从传统高发实体瘤，逐步向血液肿瘤、罕见肿瘤延伸。以往ADC研发资源多集中在乳腺癌、肺癌等大适应症领域，而本次BPDCN靶向ADC的获批，也印证了罕见肿瘤药物的研发价值。这类疾病虽然患者群体规模小，但赛道竞争压力低，临床治疗需求刚性，同时更容易获得FDA孤儿药、突破性疗法等审批倾斜，具备相应的临床价值与商业价值。图6. CD123 ADC研发情况四、现存问题与后续研发方向这款CD123靶向ADC为BPDCN治疗提供了新选择，但现阶段仍存在不少待解决的问题，也是后续研发和临床探索的主要方向。首先是疗效维持时间有限。无论是初治还是复发难治患者，中位缓解持续时间均不足10个月，多数患者最终仍会出现疾病进展、产生耐药。后续临床可以探索该药物与造血干细胞移植、免疫检查点抑制剂、去甲基化药物联合使用，通过联合方案延长缓解周期，降低复发概率。其次是肝毒性风险仍需优化。药物黑框警告提示的肝静脉闭塞病，虽然整体发生率不高，但致死风险较大。后续可以从药物分子结构入手，优化连接子稳定性，减少载荷在血液中提前脱落，降低脱靶造成的肝损伤；同时进一步细化肝功能监测标准，实现不良反应早发现、早干预。最后是适应症范围有待拓展。目前该药物仅获批用于BPDCN，但CD123抗原同样在急性髓系白血病、骨髓增生异常综合征等常见血液肿瘤中高表达。后续可开展拓展适应症临床试验，评估药物在这类疾病中的疗效，让更多血液肿瘤患者获益。从行业角度来看，首款CD123 ADC的落地，意味着该靶点正式进入系统性开发阶段。靶点耐药机制、药代/药效动力学监测、联合用药方案等相关研究，都会持续推进。CD123靶向疗法一直是高危白血病领域的重点研究方向，这款新药只是该赛道发展的开端。对于国内患者与药企而言，该药物目前尚未在国内递交上市申请，但为国内BPDCN临床治疗提供了新的思路。国内ADC企业近些年在连接子、毒素载荷等核心技术上不断突破，多款CD123靶向候选药物已进入临床阶段。随着本土研发推进，未来国内有望出现同靶点产品，进一步提升药物可及性。五、结语 pivekimab sunirine-pvzy的获批，补齐了BPDCN无精准靶向药的短板，也让全球CD123靶向治疗、罕见病 ADC 研发迈出关键一步。依托ADC技术，药物实现了肿瘤定向杀伤，切实改善了患者的治疗现状。客观来讲，该药物无法彻底根治BPDCN，耐药、安全性、适应症拓展等问题还需要长期探索。随着药物结构迭代、联合治疗方案优化，以及国内创新药企的持续发力，CD123靶向疗法会逐步走向成熟，也将为更多罕见血液肿瘤、血液系统恶性肿瘤患者带来实际的治疗帮助。参考资料： [1]U.S. FDA Approves DECNUPAZTM (pivekimab sunirine-pvzy) for Treatment of Adult Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm, an Ultra-Rare and Aggressive Blood Cancer With Limited Treatment Options. https://news.abbvie.com/2026-05-27-U-S-FDA-Approves-DECNUPAZTM-pivekimab-sunirine-pvzy-for-Treatment-of-Adult-Patients-with-Blastic-Plasmacytoid-Dendritic-Cell-Neoplasm,-an-Ultra-Rare-and-Aggressive-Blood-Cancer-With-Limited-Treatment-Options [2]Pemmaraju N, Marconi G, Montesinos P, Lane AA, Mazzarella L, Sallman DA, Ulrickson ML, Schiller GJ, Erba HP, Wang ES, Walter RB, Deconinck E, Aribi A, Legrand O, Lebon D, Maisano V, Martinelli G, DeAngelo DJ, Derenzini E, Du Y, Lakshmikanthan S, Potluri J, Kantarjian HM, Daver NG. Pivekimab Sunirine in Blastic Plasmacytoid Dendritic Cell Neoplasm. J Clin Oncol. 2026 Apr;44(10):861-873. doi: 10.1200/JCO-25-02083. [3] Shimony S, Luskin MR, Gangat N, LeBoeuf NR, Feraco AM, Lane AA. Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN): 2025 Update on Diagnosis, Pathophysiology, Risk Assessment, and Management. Am J Hematol. 2025 Aug;100(8):1408-1422. doi: 10.1002/ajh.27737. [4] https://clinicaltrials.gov/study/NCT03386513 [5] https://www.accessdata.fda.gov/drugsatfda_docs/label/2026/761460Orig1s000lbl.pdf 作者介绍：Ketty，主要从事药物研发和科普，制药行业政策和发展研究工立即扫码加入药事纵横交流群

2026-06-12

·ChenFJMU

十年首批｜国产原研创新药Beqalzi获FDA批准上市，套细胞淋巴瘤再添强效新方案！

ChenFJMUer团队编辑（原创）思语湖畔Quote FDA于2026年5月批准 1️⃣用于治疗ER阳性、HER2阴性、ESR1突变的晚期或转移性乳腺癌的Veppanu； 2️⃣用于治疗复发或难治的成人套细胞淋巴瘤患者的Beqalzi； 3️⃣用于治疗使用其他药物后血压控制不佳的成人高血压的Baxfendy； 4️⃣用于治疗丁型肝炎病毒慢性感染的Hepcludex； 5️⃣用于治疗成人浆细胞样树突状细胞肿瘤的Decnupaz； 6️⃣用于治疗由指定敏感微生物引起的复杂性尿路感染的Zaynich； 7️⃣作为COVID-19的暴露后预防措施的Xocova； 8️⃣用于对接受手术的成人实施全身麻醉的Cypsedo。细说Beqalzi （ChenFJMU原创） Beqalzi（sonrotoclax，索托克拉片），由BeiGene（百济神州）公司研发，获FDA优先审评与孤儿药资格认定，于2026年5月13日批准上市，是一种高选择性BCL-2（B细胞淋巴瘤2）抑制剂，单药用于治疗既往接受过至少两线系统性治疗（含BTK抑制剂）复发/难治性套细胞淋巴瘤成人患者。这是十年来首个获批用于套细胞淋巴瘤的BCL-2抑制剂。结构与剂型处方 Beqalzi片剂含有sonrotoclax，一种B细胞淋巴瘤2（BCL-2）抑制剂。sonrotoclax的分子式为C49H59N7O7S，Sonrotoclax的分子量为890.11，结构式为： Beqalzi口服片剂含有1、5、20或80 mg sonrotoclax。每片片剂含有以下非活性成分：无水磷酸氢钙、胶体二氧化硅（仅20和80 mg）、交联羧甲基纤维素钠、醋酸羟丙基甲基纤维素琥珀酸酯、硬脂酸镁、微晶纤维素，片剂薄膜包衣含有FD&C蓝1号/亮蓝FCF铝色淀、FD&C红40号/诱惑红ac铝色淀、聚乙二醇、聚乙烯醇、大豆卵磷脂、滑石和二氧化钛。作用机制 Sonrotoclax是高选择性BCL-2抑制剂，BCL-2在多种癌症中的过表达介导细胞存活并与化疗耐药性相关。Sonrotoclax通过特异性结合并抑制抗凋亡蛋白BCL-2，解除BCL-2对促凋亡蛋白（如Bax/Bak）的“扣押”，重启线粒体凋亡通路，诱导BCL-2依赖的肿瘤细胞（如套细胞淋巴瘤细胞）发生程序性死亡，从而发挥抗肿瘤作用。在非临床研究中，sonrotoclax在过度表达BCL-2的癌细胞中表现出细胞毒性，伴随一系列内在凋亡事件，包括半胱天冬酶激活。 Xu J, Current Advances and Future Strategies for BCL-2 Inhibitors: Potent Weapons against Cancers. Cancers. 2023; 15(20):4957. 药效学与药代动力学药效学： Sonrotoclax的安全性-效应关系尚未完全表征。心脏电生理学: Sonrotoclax给药有可能增加QTc间期。成熟B细胞恶性肿瘤患者在低脂餐中给予sonrotoclax（320 mg，每日一次）后，QTc间期的最大平均增加为7 ms。没有足够的信息来描述Sonrotoclax在高于推荐剂量的较高浓度下的QTc效应。药代动力学： Sonrotoclax的药代动力学是在单次给药后或稳态时以批准的推荐剂量320 mg每日一次测定的。Sonrotoclax血药浓度-时间曲线下面积（AUC0-24 h）为3395 ng/h/mL，最大血药浓度（Cmax）为353 ng/mL，在320 mg至640 mg剂量范围内，sonrotoclax Cmax和AUC0-tau以低于剂量比例的方式增加。重复给药后观察到sonrotoclax的有限全身蓄积。吸收：320 mg每日一次给药后，sonrotoclax的中位Tmax为4 h。食物影响：与低脂餐（约333-500千卡，25%脂肪热量）一起给药后，sonrotoclax AUC和Cmax增加约1.5倍。与高脂餐（1000千卡，50%脂肪）一起给药后，sonrotoclax AUC增加2倍，Cmax增加2.4倍。分布：sonrotoclax的几何平均表观分布容积为482 L。在1至10 μM的浓度范围内，sonrotoclax的血浆蛋白结合率为99%。血液与血浆的比值为0.6至0.7。消除：sonrotoclax的平均终末消除半衰期（t½）范围为4至6 h。sonrotoclax的几何平均表观口服清除率（CL/F）为94 L/h。代谢：sonrotoclax体外主要通过CYP3A代谢，较小程度上通过CYP2C8代谢。排泄：健康受试者单次服用20 mg放射性标记sonrotoclax后，大约86%的剂量在粪便中回收，0.28%在尿液中回收。特殊人群用药： ①妊娠：Beqalzi可致胎儿伤害，妊娠期禁用；育龄女性用药前需确认妊娠状态，治疗期间及停药后1周需有效避孕。 ②哺乳期：尚不明确药物是否分泌入乳，建议治疗及停药后1周内暂停哺乳。 ③儿童：儿童患者安全性与有效性未确立，不推荐使用。 ④老年人：≥65岁患者严重不良反应发生率更高，需监测，无需常规调整起始剂量。 ⑤肾功能不全：轻至中度肾损（eGFR≥30 mL/min）无需调整剂量；重度肾损未开展研究，慎用。 ⑥肝功能不全：轻至中度肝损（总胆红素≤3×ULN）无需调整剂量；重度肝损未开展研究，慎用。药物相互作用： ①强效CYP3A抑制剂：在开始使用时和剂量增加阶段禁用，剂量增加后减少目标剂量。 ②中效CYP3A抑制剂：避免与1 mg和2 mg剂量的Beqalzi同时使用，并减少所有其他剂量。 ③强效或中效CYP3A诱导剂：避免与Beqalzi同时使用。非临床毒理学 Beqalzi未开展致癌性试验；致突变性相关检测为阴性，无遗传毒性；未进行专门生育力试验，但重复给药毒性试验表明其可致小鼠雌性生殖器官异常、犬雄性生殖器官损伤，且该损伤具有可逆性；胚胎‑胎仔发育毒性研究证实，妊娠动物于器官形成期给药可引发胎仔生长迟缓与结构畸形，提示存在胎儿发育风险。临床研究 Beqalzi的临床研究核心基于全球多中心、单臂、开放标签Ⅱ期试验BGB‑11417‑201（NCT：05471843），纳入既往接受过含抗CD20方案与BTK抑制剂治疗的复发/难治性套细胞淋巴瘤（MCL）成人患者，采用4周剂量递增后320 mg每日一次方案，由独立评审委员会（IRC）按Lugano标准评估疗效。疗效结果见表9，结果显示总缓解率（ORR）52%、完全缓解率（CR）16%，中位起效时间1.9个月，中位缓解持续时间11.9个月，安全性以中性粒细胞减少、血小板减少、感染等为主，整体可管理，该结果支持其获FDA加速批准，目前正开展Ⅲ期试验以进一步验证临床获益。用药指南编辑：魏欣怡排版：魏欣怡补充和审核：陈昌买推送：FDA新药图鉴欢迎个人转发分享，企业及其他公众号需授权后转载，并注明出处！）

孤儿药上市批准优先审批

2026-06-10

·捷信医药科技

2026年5月国内外[NMPA&FDA]获批新药大盘点

2026年5月，NMPA批准上市了8款新药(仅包含1类新药/治疗用生物制品3.1类/化药5.1类)，具体为：尼卡利单抗注射液作用机制：FcRn拮抗剂适应症：用于12 岁及以上抗乙酰胆碱受体（AChR）或抗肌肉特异性受体酪氨酸激酶（MuSK）抗体阳性全身型重症肌无力（gMG）成人及青少年患者，联合常规治疗。法赞雷生片作用机制：食欲素（Orexin）受体抑制剂适应症：治疗以入睡困难和 / 或睡眠维持困难为特征的成人失眠患者。注射用塔拉妥单抗作用机制：DLL3*CD3双特异性抗体适应症：既往接受过1种系统性治疗（包括含铂化疗）失败的广泛期小细胞肺癌（ES-SCLC）成人患者。甲苯磺酸纳地美定片作用机制：外周μ受体拮抗剂适应症：用于治疗成人因阿片类药物引起的便秘（OIC）。安尼妥单抗注射液作用机制：HER2双表位双特异性抗体适应症：联合化疗治疗既往至少接受过一种含曲妥珠单抗治疗方案的局部晚期或转移性HER2阳性成人胃或胃食管结合部腺癌患者。鲁兹诺雷钠片作用机制：URAT1 抑制剂适应症：治疗痛风伴高尿酸血症。塞多明基注射液作用机制：裸质粒型基因治疗药物适应症：治疗不适合血运重建手术或手术效果不佳的严重下肢缺血（下肢动脉硬化闭塞症、血栓闭塞性脉管炎和糖尿病下肢缺血等）导致的肢体溃疡。磷奈匹坦帕洛诺司琼注射用浓溶液作用机制：NK1 受体拮抗剂 + 5-HT3 受体拮抗剂复方制剂适应症：预防化疗引起恶心呕吐。与此同时，FDA公布了8款首次获批的创新药（仅含新分子实体和新生物制剂）。亮点首个用于晚期乳腺癌的口服选择性雌激素受体降解剂(PROTAC)获FDA批准--vepdegestrant 美国首个用于MCL治疗的BCL-2抑制剂获批--sonrotoclax 全球首款高选择性醛固酮合成酶抑制剂获批--baxdrostat 首个用于治疗慢性丁型肝炎的药物获批--bulevirtide-gmod 全球首个治疗母细胞性浆细胞样树突细胞肿瘤药物获FDA批准--pivekimab sunirine-pvzy 复杂性尿路感染迎来新型抗感染治疗药物--cefepime and zidebactam 全球首个用于COVID-19暴露后预防的口服药物获批--ensitrelvir 中国自主创新静脉麻醉药物获美国FDA批准--cipepofol 中国NMPA批准的8款新药List： FDA批准的8款新分子实体/新生物制剂（不含新增适应症）List：我们会在每个月上线国内外（NMPA&FDA）获批新药盘点，敬请期待！期待2026年有更多好药新药获批上市，为众多患者带来更多希望和生命之光！ *以上内容仅供专业人士参考数据来源：以上数据均来自NMPA和FDA官网。点击下方小程序卡片了解更多在研新药临床捷信医药成立于2008年，是国内领先的数字化患者解决方案企业，也是亚洲患者招募组织发起人之一。公司专注临床研究患者招募及管理，覆盖城市230多个，服务项目1700个，在 250多个疾病领域积累了成功经验，助力100多个新药在中国上市。公司具有领先的数字化业务体系，具有成熟的全渠道推广及招募管理经验。公司自有平台“新药之光"——以患者为中心的临床研究服务平台，致力于患者招募、患者保留及依从性管理、患者访谈与调研、患教活动及直播、视频策划与制作(包括临床研究推广和普及、项目宣传、疾病领域科普和教育、故事和生命教育等)、全平台内容推广(大V医生、垂直博主和素人)、大平台内容收录与优化。同时，公司持续研发创新，推出以AI赋能的临床研究项目协同平台，包括AI自动脱敏、AI辅助筛选，协同讨论等模块，更好地联结各方，精准匹配，提高效率。捷信作为研发型药企优选供应商，我们连接大众端、患者端、医生端、研究机构，保障临床研究试验高质量合规入组，实现从患者移动端报名到入组的多方在线协同，为企业用户提供数字化解决方案。

100 项与 Pivekimab Sunirine 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
Blastic浆细胞样树突状细胞肿瘤	美国	AbbVie, Inc.	2026-05-27

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
急性髓性白血病	临床3期	-	AbbVie, Inc.	2026-07-22
复发性 B 细胞急性淋巴细胞白血病	临床2期	-	ImmunoGen, Inc.	2023-08-01
复发性混合表型急性白血病	临床2期	-	ImmunoGen, Inc.	2023-08-01
难治性急性髓细胞白血病	临床2期	-	ImmunoGen, Inc.	2023-08-01
难治性 B 细胞急性淋巴细胞白血病	临床2期	-	ImmunoGen, Inc.	2023-08-01
难治性混合表型急性白血病	临床2期	-	ImmunoGen, Inc.	2023-08-01
难治性 T 急性淋巴细胞白血病	临床2期	-	ImmunoGen, Inc.	2023-08-01
复发性急性髓细胞白血病	临床2期	-	ImmunoGen, Inc.	2023-08-01
CD123阳性急性髓性白血病	临床2期	美国	AbbVie, Inc.	2018-01-02
CD123阳性急性髓性白血病	临床2期	法国	AbbVie, Inc.	2018-01-02

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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


NCT03386513 (CADENZA, ASH2025)	临床1/2期	Blastic浆细胞样树突状细胞肿瘤一线	11	Pivekimab sunirine 0.045 mg/kg (Prior or concomitant hematologic malignancy)	壓壓鹽壓築糧願願襯糧(糧網鏇選構鏇鏇顧網顧) = 獵積窪膚範廠繭構窪網構艱遞構簾獵獵鏇糧簾 (構鑰膚簾鏇壓網鑰鏇範, 8.3 ~ NR) 更多	积极	2025-12-06
ASH2025	临床1/2期	急性髓性白血病一线 CD123-positive	49	Pivekimab sunirine+Venetoclax+Azacitidine	構廠糧鏇廠繭醖繭壓夢(糧選簾築選構範蓋選窪) = 窪窪淵廠壓襯鹽遞艱蓋積蓋繭觸選鹽築艱廠窪 (夢憲糧醖衊獵衊襯繭壓, 48.3 ~ 76.6) 更多	积极	2025-12-06
NCT03386513 (CADENZA, ASCO2025) 人工标引	临床1/2期	Blastic浆细胞样树突状细胞肿瘤 Interleukin-3 Receptor Subunit Alpha Positive	84	Pivekimab sunirine 0.045 mg/kgPivekimab sunirine 0.045 mg/kg (1L treatment)	憲襯遞壓糧壓範艱廠膚(築鹽願鏇獵顧選夢壓窪) = 衊艱鹹觸網艱範夢壓襯積築艱獵鑰醖衊選願鬱 (獵鬱淵糧蓋齋積願鹽積, 51.3 ~ 84.4) 更多	积极	2025-05-30
NCT03386513 (CADENZA, ASCO2025) 人工标引	临床1/2期		84	Pivekimab sunirine 0.045 mg/kgPivekimab sunirine 0.045 mg/kg (relapsed/refractory pts received 1-3 lines)	憲襯遞壓糧壓範艱廠膚(築鹽願鏇獵顧選夢壓窪) = 醖蓋襯遞膚積積顧願窪積築艱獵鑰醖衊選願鬱 (獵鬱淵糧蓋齋積願鹽積 ) 更多	积极	2025-05-30
NCT03386513 (CADENZA, EHA2025)	临床1/2期	Blastic浆细胞样树突状细胞肿瘤 CD123 (IL-3Rα)	84	Pivekimab Sunirine (PVEK) 0.045 mg/kg	選選衊艱獵憲鏇顧蓋範(願餘積憲衊顧醖製淵構) = 製鹹獵選衊艱糧壓觸觸構膚願獵鏇鏇網衊憲觸 (遞範獵夢鏇艱範淵蓋觸, 51.3 ~ 84.4) 更多	积极	2025-05-22
EHA2024	临床1/2期	CD123阳性急性髓性白血病 CD123-positive	-	Pivekimab sunirine (PVEK)	獵壓構繭獵積鬱醖築願(選鏇淵鬱夢膚網壓製齋) = 鏇築襯醖鬱壓獵鬱艱膚廠膚選齋簾範窪艱繭繭 (餘廠鹽鹹廠鹽顧繭鏇廠 )	-	2024-05-14
NCT03386513 (Literature) 人工标引	临床1/2期	急性髓性白血病 Interleukin-3 Receptor Subunit Alpha Positive	91	IMGN632	餘遞夢鹽餘襯遞窪範艱(鬱簾齋鬱壓願鹽繭醖構) = 蓋製襯製繭繭齋鹹醖蓋糧構獵積構膚製繭衊廠 (鹽糧築願鏇淵壓觸鹹憲 ) 更多	积极	2024-03-01
ASH2023	临床1/2期	急性髓性白血病	50	Pivekimab Sunirine (PVEK)	壓範觸膚鹽鹹壓衊鑰構(願鹹獵膚積構壓構糧壓) = 鹹獵簾繭糧鏇網鹹製衊夢獵繭淵築選繭願願窪 (繭鑰廠鏇蓋繭鏇窪鹽鑰 ) 更多	-	2023-12-10
NCT04086264 (ASH 12022 \| ASH 22022) 人工标引	临床1/2期	复发性急性髓细胞白血病	71	Azacitidine+Venetoclax+Pivekimab Sunirine	範觸願齋淵憲艱鬱製鏇(鏇窪憲範艱網獵遞觸壓) = 膚鬱簾襯鬱鹹遞壓窪壓壓築艱願襯鏇製淵齋蓋 (製窪築顧願淵構糧窪鑰 ) 更多	积极	2022-11-15
NCT04086264 (AML-262, Pubmed \| Clin Lymphoma Myeloma Leuk)	临床1/2期	复发性急性髓细胞白血病	-	Pivekimab sunirine (PVEK)	淵製膚構鑰網齋選窪壓(夢艱糧糧窪積選窪鹽鹹) = IRRs were reported in 33% (n=17, one grade 4) of patients given 1 dose of dexamethasone (8 mg) as premedication (n=51); these IRRs were most frequently tachycardia and chills, with no anaphylactic reactions reported. Following the data cut-off, there was a second grade 4 IRR, and the prophylactic regimen was increased with two additional doses of dexamethasone on the day prior to the PVEK dose. The IRR rate has dropped to 8% (3 of 38), with no grade 3+; all were grades 1-2 that resolved with limited intervention (P<0.01) 簾壓願願鏇醖齋廠選鬱 (遞壓鏇築製簾鬱願淵廠 )	-	2022-10-01
NCT03386513 (CADENZA, Corporate Publications) 人工标引	临床1/2期	Blastic浆细胞样树突状细胞肿瘤	10	pivekimab (de novo patients)	壓築餘繭鑰艱築窪鏇憲(顧選製構鹽淵膚遞憲顧) = 淵願鏇選積鏇繭顧鹽襯遞繭夢壓觸廠築醖構繭 (遞簾築網膚膚鏇獵網憲 )	积极	2022-08-31
NCT03386513 (CADENZA, Corporate Publications) 人工标引	临床1/2期	Blastic浆细胞样树突状细胞肿瘤	10	pivekimab (PCHM patients)	艱襯鹽積選淵淵蓋壓憲(艱淵範觸廠鹽鹽膚糧鏇) = 簾繭夢蓋廠積窪鬱繭餘獵衊觸築鏇壓夢網積築 (壓鏇鹽鹹衊壓範製構遞 )	积极	2022-08-31