Rezafungin acetate

来源：中华医学网，发布2026-09-27，刊发于《中国药业》2026年第1期 说明：德尔菲法、50位多学科专家共同制定；面向新结构、新靶点、抗多重耐药菌创新抗菌药，用于医院药事AMS遴选评估，仅作学习交流，不替代院内正式审批流程。 新药不断涌现，依拉环素、头孢洛生‑他唑巴坦、瑞扎芬净这类抗耐药菌新药，给难治感染带来新选择。但新药该不该进本院目录，不能只看文献疗效、跟风引进。这份共识搭建了一套可落地的量化评价框架，把临床刚需、耐药风险、微生物检测能力、卫生经济学全部纳入闭环管理。一、什么才是共识定义的“创新抗菌药物” ✅ 纳入范围： 全新化学结构、全新作用靶点的抗菌药物 针对多重耐药菌开发的新型复方制剂 改良型新药：明显优化PK/PD、降低毒性、克服原有耐药（单纯改剂型、改盐、仿制药不算） ❌ 不适用本遴选体系：仅增加给药途径、普通仿制药。 遴选初衷：补齐多重耐药菌治疗短板，同时避免新药过早、大范围使用，加速细菌耐药进化，拒绝“为引进新药而引进新药”。二、五大量化评价维度（核心框架）1. 有效性（权重最高） 体外活性：重点对照本院、本区域耐药谱，评估能不能覆盖现有药物难以对付的耐药菌株，参考MIC数据； 循证证据：临床试验等级、国内外指南推荐、目标适应症人群； PK‑PD特性：组织穿透、蛋白结合率、是否支持TDM、给药方案合理性； 横向对比：对比院内现有方案，区分是不可替代刚需，还是仅小幅优效、等效。 共识明确：如果院内已有可靠药物覆盖同类致病菌，不优先引进。2. 安全性 不良反应谱、严重不良反应发生率；老年、肝肾损伤、儿童、妊娠等特殊人群数据是否充足； 药物相互作用（DDI）风险； 次生风险：内毒素释放、肠道菌群紊乱、艰难梭菌感染风险； 上市周期短的新药，要客观识别证据缺口，做好风险预案。3. 适宜性（AMS管理核心） 匹配本院真实需求：看本院多重耐药菌检出率，有没有实实在在的救治缺口； 耐药诱导风险：评估突变选择窗口，预判使用后耐药快速上升的可能性； 给药方式、疗程、储存条件是否适配本院诊疗场景； 分级定位：绝大多数抗耐药创新药，直接划定为特殊使用级抗菌药物，执行严格会诊审批。 红线：本院没有对应的耐药流行压力，只是追求新药，不建议引进。4. 可获得性 药品供应稳定性、国内获批适应症范围； 储存运输要求，是否需要特殊冷链； 配套微生物药敏检测能力：医院能不能稳定开展对应的药敏检测。 重点警示：没有配套药敏检测能力的医院，应当慎重引进抗耐药新药。5. 经济性 不只看采购单价，要做卫生经济学评估： 和现有补救方案直接成本对比； 间接获益：缩短住院日、降低治疗失败率、减少ICU占用； 医保、集采覆盖情况，评估长期科室、医院预算压力。 三、完整引进流程（AMS多学科闭环） 临床发起申请：科室提交材料，说明当前抗感染困境、本院耐药现状； 药学初评：梳理五大维度资料，形成初评评估报告； AMS多学科论证（感染、微生物、院感、药学）：核心讨论三点——是不是刚需？替代方案是否已经穷尽？耐药风险与配套管控措施； 抗菌药物管理工作组审议：≥2/3成员同意方可进入下一环节； 药事管理与药物治疗学委员会终审：≥2/3同意，纳入本院抗菌药物目录。 ✅ 准入之后，管控不能缺位： 归入特殊使用级，落实MDT会诊审批； 限定适应症，严禁随意扩大经验性使用； 专项监测：用药强度、药敏结果、耐药变迁、不良反应； 周期性再评价（1～2年一次），无持续临床需求可以启动清退。 四、明确：这些情形，不建议引进 ❌ 只是剂型改良，没有带来实质临床获益 ❌ 本院几乎没有对应耐药菌株流行，不存在真实救治缺口 ❌ 关键临床证据不足，缺少中国人群研究数据 ❌ 缺少配套药敏检测手段，无法指导精准用药 ❌ 已有安全、可及、有效的替代治疗药物 ❌ 供货不稳定，存在较高断供风险五、准入后动态管理要点 创新抗菌药物不是“入目录永久保留”。 严禁当作常规经验用药，仅限目标耐药菌感染的针对性治疗；持续开展处方点评、病例登记、耐药预警。一旦目标菌株耐药率快速攀升，或是出现更优替代方案，及时评估，采取限制使用或者清退。小结 这份共识把新药遴选从“看文献、看热度”，变成循证量化、多学科把关、微生物检测能力前置、准入后持续监测退出的精细化模式。新药是对抗耐药菌的利器，但前提是：我们有检测能力、有明确刚需、有配套管控，才能用好，延缓耐药。参考文献 [1] 医疗机构创新抗菌药物遴选标准专家共识[J].中国药业,2026. [2] 抗菌药物临床应用管理办法[S].国家卫生健康委员会.

CAMBRIDGE, England--(BUSINESS WIRE)--Today Mundipharma announced additional results from the global Phase III ReSPECT clinical trial evaluating REZZAYO® (rezafungin acetate) for prophylaxis of invasive fungal diseases (IFDs) in adult patients undergoing allogeneic haematopoietic stem cell transplantation (allo-HSCT). The Phase III findings were presented today as a late-breaking oral presentation at IDWeek 2026. The presentation provides additional detail following the top line ReSPECT results announced in April 2026, including further endpoint analyses, the components contributing to fungal-free survival (FFS), pre-specified sensitivity analyses and detailed safety outcomes. The additional analyses were consistent with the previously announced topline findings. ReSPECT was a global, multicentre, randomised, double-blind Phase III study in which 402 patients were randomised to rezafungin and 200 to the standard antimicrobial regimen. As previously announced, the study met its primary endpoint, demonstrating non-inferiority of rezafungin to the standard antimicrobial regimen for FFS at Day 90 following allo-HSCT. The primary endpoint result was supported by six pre-specified sensitivity analyses examining the handling of indeterminate outcomes, definitions of IFD, randomisation stratification factors and the use of non-study systemic antifungal therapy. Phase III findings were presented as a late-breaking oral presentation at IDWeek 2026 FFS at Day 90 was 60.7% with rezafungin and 59.0% with the standard antimicrobial regimen, corresponding to an estimated treatment difference of 1.6% (95% CI -6.2 to 9.3; P=0.0002 for non-inferiority) Proven or probable IFD contributing to FFS failure occurred in 3.5% of patients receiving rezafungin versus 4.5% receiving the standard antimicrobial regimen Data from ReSPECT have been submitted to the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) for regulatory review Antifungal prophylaxis is an important component of care for patients undergoing allo-HSCT, who are at risk of life-threatening IFD. Despite established prophylactic approaches, clinical challenges can include drug toxicities, drug–drug interactions, therapeutic drug monitoring, antifungal resistance and difficulties with daily oral administration, (including adherence to antimicrobial regimens). There remains an unmet need for continued research and evidence generation to optimise prevention in this complex, immunocompromised and medically vulnerable population.2 Additional findings from ReSPECT The composite FFS endpoint incorporated survival, absence of proven or probable IFD as assessed by an independent Data Review Committee, and no use of non-study systemic antifungal therapy for more than 10 cumulative days. Proven or probable IFD contributing to FFS failure occurred in 3.5% of patients receiving rezafungin and 4.5% receiving the standard antimicrobial regimen. Death contributing to FFS failure occurred in 4.5% and 5.5%, respectively, while use of non-study systemic antifungal therapy for more than 10 cumulative days was numerically lower in the rezafungin group, occurring in 27.4% and 32.5%, respectively. The study also reported IFD caused by individual fungal species. Candida infections occurred in 1.2% versus 1.0%, Aspergillus infections in 2.0% versus 3.0%, and Pneumocystis jirovecii infections in 0.2% versus 0% in the rezafungin and standard antimicrobial regimen groups, respectively. Treatment-emergent adverse events were reported in 99.7% of patients receiving rezafungin and 100% receiving the standard antimicrobial regimen. Serious adverse events occurred in 66.0% and 67.5% of patients, respectively. Treatment-emergent adverse events leading to study discontinuation occurred in 9.3% and 14.7%, while those leading to dose reduction, interruption or withdrawal of study treatment occurred in 31.0% and 47.7%, respectively. No new safety signals were identified in allo- HSCT recipients. “Patients undergoing allogeneic haematopoietic stem cell transplantation remain vulnerable to invasive fungal disease, and prevention is an important part of their clinical management. ReSPECT provides new Phase III evidence in this complex setting and contributes to the continuing effort to address unmet needs in antifungal prophylaxis,” said Johan Maertens, MD, PhD, University Hospitals Leuven. “Our continued investment in the rezafungin research programme reflects Mundipharma’s commitment to advancing antifungal science and generating robust clinical evidence in areas where important unmet needs remain,” said Yuri Martina, MD, PhD, Chief Development and Medical Officer, Mundipharma. “ReSPECT is an important part of that commitment, and we are pleased to see these Phase III findings shared with the scientific community as a late-breaking oral presentation at IDWeek. We are planning to communicate progress and emerging findings through international scientific meetings including ASH, ESCMID, EHA, TIMM and EBMT, as well as through peer-reviewed publications in specialized medical journals. The submission of these data to the FDA and EMA represents another important milestone in our ongoing research programme.” Data from the ReSPECT programme have been submitted to the FDA and EMA for regulatory review. Rezafungin prophylaxis in adults undergoing allo-HSCT represents the investigational use evaluated in ReSPECT. This prophylaxis indication is not currently approved, and approved indications for rezafungin vary by jurisdiction. In the United States, the FDA has granted Priority Review, with a PDUFA (Prescription Drug User Fee Act) target action date in Q1 2027. About ReSPECT ReSPECT is a Phase III, multicentre, randomised, double-blind study comparing once-weekly intravenous rezafungin with a daily standard antimicrobial regimen for prevention of invasive fungal disease following allo-HSCT through Week 13. Adults were randomised 2:1 to rezafungin or standard antimicrobial prophylaxis. Rezafungin was administered as a 400 mg intravenous loading dose on Day 1 followed by 200 mg once weekly. The standard antimicrobial regimen consisted of a daily oral azole, fluconazole or posaconazole, together with trimethoprim/sulfamethoxazole following engraftment. The primary endpoint was FFS survival at Day 90 following HSCT, a composite incorporating survival, absence of proven or probable IFD, and no use of non-study systemic antifungal therapy for more than 10 cumulative days. Regulatory status REZZAYO® (rezafungin) is the differentiated echinocandin with a unique PK/PD profile indicated for the treatment of invasive candidiasis in adults. REZZAYO has approved indications for the treatment of Candida infections in both the United States and European Union; 4 these differ from the investigational prophylaxis setting evaluated in ReSPECT. In the United States, REZZAYO is an echinocandin antifungal indicated in adult and paediatric patients 12 years of age or older who have limited or no alternative options for the treatment of candidemia and invasive candidiasis. Approval of this indication is based on limited clinical safety and efficacy data for REZZAYO.3 In the European Union, REZZAYO is indicated for the treatment of invasive candidiasis in adults. The EU Summary of Product Characteristics states that consideration should be given to official guidance on the appropriate use of antifungal agents.4 The use of rezafungin for prophylaxis of invasive fungal disease in adults undergoing allogeneic haematopoietic stem cell transplantation, as evaluated in the Phase III ReSPECT study, is investigational and is not currently an approved indication in either the United States or European Union. About Mundipharma Mundipharma is a global healthcare company with a presence in Africa, Asia Pacific, Canada, Europe, Latin America and the Middle East. In line with its mission, United for Patients, Mundipharma is dedicated to bringing innovative treatments to patients in the areas of pain management, infectious disease, ophthalmology, oncology, respiratory and central nervous system. For more information visit: www.mundipharma.com. References: Maertens J. et al, Rezafungin (Once Weekly) versus Standard Antimicrobial Regimen for Antifungal Prophylaxis in Allogeneic Hematopoietic Stem Cell Transplantation: ReSPECT, a Multicentre, Randomized, Double-Blind, Phase III, Oral presentation at IDWeek 2026, 22 October 2026. Quattrone M. et al, Managing Invasive Fungal Infections During Allogeneic Hematopoietic Transplantation: A 2025 Update. Mediterr J Hematol Infect Dis. 2025 Sep 1;17(1):e2025064. doi: 10.4084/MJHID.2025.064. PMID: 40937308; PMCID: PMC12422250. HIGHLIGHTS OF PRESCRIBING INFORMATION REZZAYO https://www.accessdata.fda.gov/drugsatfda_docs/label/2026/217417s007lbl.pdf (last accessed October 2026) Rezzayo: EPAR - Product information https://www.ema.europa.eu/en/documents/product-information/rezzayo-epar-product-information_en.pdf (last accessed October 2026) Job Code: GBL-S-RZF-2600007 Date of preparation: October 2026