MaaT-013

2026年6月26日，欧洲药品管理局CHMP完成审评，建议撤销2022年获批的ANCA相关性血管炎治疗药物Tavneos的欧盟上市授权。因核心临床试验存在数据失真、违反临床规范等问题，该药获益风险比不再成立，欧盟已停止新患者用药。与此同时，该药在美国遭遇监管争议，安进仍积极对接FDA推进审评。 No new patients will be treated with Tavneos in the EU, and patients currently on the drug should talk their physician about next steps, CSL, which markets the drug in Europe, said.  欧洲市场负责销售 Tavneos 的 CSL 公司表示：欧盟范围内不再为新患者开具 Tavneos 治疗；正在使用该药物的患者应咨询主治医生后续诊疗方案。 As Amgen prepares to defend Tavneos’ market approval in the U.S., the company has received a bad omen overseas, with regulators recommending that the ANCA-associated vasculitis drug lose its authorization in the European Union after a new review led to the conclusion that its benefits are “no longer proven to outweigh its risks.” 安进公司正准备在美国为 Tavneos 的上市许可进行辩护，而该药物在海外市场却迎来不利消息：欧洲药品监管机构完成新一轮审评后得出结论，现有证据无法证实该药获益大于风险，现已建议欧盟撤销这款 ANCA 相关性血管炎治疗药物的上市授权。 That was the take from the European Medicines Agency, which issued a roundup of recent approval decisions—both positive and negative—from its Committee for Medicinal Products for Human Use (CHMP) on Friday. Once the CHMP has delivered marketing authorization recommendations, final sign-off on the decisions must still be conferred by the European Commission. 以上为欧洲药品管理局（EMA）周五发布的结论，该局汇总了人用医药产品委员会（CHMP）近期全部获批与不予获批审评意见。CHMP 出具上市许可建议后，最终审批决议仍需欧盟委员会正式签发。 In the case of Tavneos—originally approved in two rare forms of ANCA-associated vasculitis in the EU in 2022—the CHMP has now wrapped up a review of the drug that started earlier this year over data integrity questions from the main study that supported its approval. Tavneos 于 2022 年在欧盟获批用于两种罕见 ANCA 相关性血管炎；今年年初，因支撑其获批的核心临床试验存在数据完整性问题，CHMP 启动对该药的重新审评，目前该审评工作已全部完成。 At the time of its original authorization, and based on data from the 331-patient Advocate study, European regulators had determined that Tavneos was “at least as effective” as a 20-week course of high-dose corticosteroids at inducing remission in patients with active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA). 当初获批时，监管机构依托纳入 331 名受试者的 Advocate 研究数据判定：针对活动性肉芽肿性多血管炎（GPA）、显微镜下多血管炎（MPA）患者，Tavneos 在诱导病情缓解方面疗效不劣于 20 周高剂量糖皮质激素治疗方案。 But the CHMP has now determined that the Advocate study was “conducted in breach of good clinical practice (GCP) principles,” adding that the data provided for the assessment “were found to be incorrect and misleading and could no longer be relied upon for demonstrating Tavneos’ effectiveness.” 但 CHMP 本次审评认定，Advocate 研究开展过程违反药物临床试验质量管理规范（GCP）原则，审评所用研究数据存在错误、具有误导性，无法再作为证明 Tavneos 疗效的依据。 Moreover, post-marketing data and other post-hoc analyses “are not considered sufficient” to argue in favor of the drug’s benefits, per the CHMP. CHMP 补充说明，上市后临床数据及各类事后分析结果，均不足以佐证该药具备明确临床获益。 A final decision is now expected by the European Commission “shortly,” Vifor Fresenius Medical Care Renal Pharma and CSL—which market Tavneos in Europe—said in a June 26 release. 负责欧洲地区 Tavneos 商业化的维福费森尤斯医疗肾病制药公司与 CSL 于 6 月 26 日发布公告称，欧盟委员会将很快出具最终审批决议。 "While we are disappointed in the outcome of the Article 20 procedure, we will respect the outcome of the regulatory process and are committed to implementing it in full, Bill Mezzanotte, head of R&D at CSL, said in a statement. CSL 研发负责人比尔・梅扎诺特在声明中表示：“我们对本次第 20 条款审评程序的结果感到遗憾，但我们尊重监管流程给出的结论，并将全面落实相关要求。” No new patients will be treated with Tavneos in the EU, and patients currently on the drug should discuss next steps with their physician about next steps, CSL added. CSL 补充称，欧盟不再接纳新患者使用 Tavneos，正在用药的患者需与主治医生沟通后续诊疗方案。 Stateside, Amgen is gearing up for an FDA hearing to address the regulator’s efficacy and safety concerns over Tavneos, recently winning a one-month extension on a data deadline, which is now set for July 29, according to Bloomberg News. 彭博社消息，美国本土方面，安进正筹备 FDA 听证会，回应监管机构针对 Tavneos 疗效与安全性提出的质疑；公司近期成功申请将数据提交截止日期延长一个月，新截止日定为 7 月 29 日。 Earlier this year, Amgen refused an FDA request to stop selling Tavneos in the U.S., with the agency stepping up its efforts to potentially curtail its use in late April when it formally proposed to withdraw the medication. 今年早些时候，FDA 曾要求安进暂停在美国销售 Tavneos，遭到企业拒绝；4 月末监管机构加大管控力度，正式提议撤销该药物的美国上市许可。 "We are deeply concerned about the potential impact that the CHMP recommendation will have on patients and healthcare providers who value access to Tavneos as a treatment option," a spokesperson for Amgen told Fierce in an emailed statement, reaffirming that CSL is leading all EMA interactions on the drug across the pond. 安进发言人通过邮件向医药行业媒体 Fierce 表示：“CHMP 的审评建议可能会影响依赖 Tavneos 开展治疗的患者与医护人员，我们对此深感担忧”，并重申由 CSL 全权负责该药与欧洲 EMA 的全部对接工作。 "Amgen continues to believe that Tavneos is an important treatment option for people living with AAV with a favorable benefit-risk profile based on all available data, including over 20 real-world studies," the spokesperson added. "The deadline for our hearing submission has been extended to July 29, and we remain engaged with the FDA as the regulatory process continues in the United States." 该发言人补充道：“依托包含 20 余项真实世界研究在内的全部现有数据，安进始终认为 Tavneos 是 ANCA 相关性血管炎患者的重要治疗药物，具备良好的获益风险比。听证会材料提交期限已延至 7 月 29 日，美国监管程序推进期间，我们会持续与 FDA 保持沟通。” A busy week for CHMP CHMP 本周审评工作密集 Tavneos isn’t the only drug to get the cold shoulder from European regulators this week. 本周遭到欧洲监管机构出具负面审评意见的药物并非仅有 Tavneos 一款。 In particular, MaaT Pharma said Friday that it will seek a reexamination of its application after the CHMP adopted a negative opinion on conditional marketing authorization for its acute Graft-versus-Host disease (aGvHD) prospect, MaaT013. 其中，微生物制药企业 MaaT Pharma 周五公告，CHMP 针对其用于急性移植物抗宿主病（aGvHD）的在研药物 MaaT013 出具附条件上市许可负面意见，企业将申请复审。 As it stands, the CHMP believes that given the use of concomitant therapies to manage aGvHD, MaaT’s data package “does not allow sufficient attribution of the observed clinical effect and safety to the study treatment alone,” the company said in a Friday release. 企业周五公告称，CHMP 认为，临床中会联合其他药物共同干预急性移植物抗宿主病，现有申报数据无法充分证明观测到的临床疗效与安全性仅由试验药物单独产生。 Under EMA procedures, MaaT expects to receive its new opinion within 60 days of its reexamination request potentially being approved, which could set up a new verdict sometime around the middle of September, per the company. 依据 EMA 审评流程，若复审申请获批，MaaT 预计 60 日内可收到全新审评意见，新决议或于 9 月中旬前后出炉。 MaaT is developing what it calls microbiome ecosystem therapies to help “restore balance and maximize clinical benefits for patients with severe, treatment-induced dysbiosis in acute diseases.” MaaT 专注研发微生物组生态疗法，旨在为急性重症、因治疗引发菌群失调的患者重建菌群平衡，最大化临床获益。 Aside from MaaT’s drug, which the company hopes to market as Xervyteg, the CHMP also handed out negative opinions to the Netherlands Cancer Institute’s Tacquell in advanced melanoma and Yartemlea, from Omeros, to treat adults and kids ages two and older with hematopoietic stem cell transplant (HSCT)-associated thrombotic microangiopathy. 除 MaaT 这款计划商品名为 Xervyteg 的候选药外，CHMP 还对两款药物出具负面意见：荷兰癌症研究所用于晚期黑色素瘤的 Tacquell、奥默罗斯公司 Yartemlea（用于 2 岁及以上成人与儿童造血干细胞移植相关血栓性微血管病）。 On the other end of the spectrum, the CHMP also doled out several positive recommendations to big-name drugs this week, including Eli Lilly’s BTK inhibitor Jaypirca, which has received the regulator’s blessing to treat adults with chronic lymphocytic leukemia (CLL) across all lines of therapy, regardless of prior BTK inhibitor treatment. The European Commission is expected to make a final determination “in the next one to two months,” according to Lilly. 与此同时，CHMP 本周也为多款重磅药物出具积极审评意见，其中礼来 BTK 抑制剂 Jaypirca 获批推荐用于成人慢性淋巴细胞白血病（CLL）全线治疗，无论患者既往是否使用过其他 BTK 抑制剂。礼来表示，欧盟委员会预计将在 1 至 2 个月内出具最终审批结果。 The positive recommendation, fueled by results from the BRUIN CLL-313 and -314 studies, bodes well for the drug’s chances in the indication in the U.S., where the FDA is expected to weigh in on approval in the second half of 2026. 本次积极意见依托 BRUIN CLL-313、BRUIN CLL-314 两项研究数据，也为该适应症在美国的审批前景带来利好，FDA 预计将于 2026 年下半年给出审批结论。 Meanwhile, Incyte’s topical JAK inhibitor is inching closer to its second EU indication after the CHMP put its weight behind the cream-based treatment’s approvability in adults with moderate atopic dermatitis. If given EC sign off, Opzelura would become the first steroid-free, topical JAK treatment available for this patient population in Europe, Incyte noted Friday. 此外，因赛特外用 JAK 抑制剂距离欧盟第二项适应症获批更进一步，CHMP 支持这款乳膏制剂用于成人中度特应性皮炎。企业周五称，若获欧盟委员会批准，Opzelura 将成为欧洲首款可用于该类患者人群、不含激素的外用 JAK 制剂。 The CHMP based its decision on data from the phase 3 TRuE-AD4 study, plus supporting data from the separate late-stage trials TRuE-AD1 and TRuE-AD2, with the first, pivotal study showing that Incyte’s product significantly improved clinical signs and symptoms of moderate atopic dermatitis, including itch, as early as day 2 of treatment. CHMP 的审评结论依托 3 期关键研究 TRuE-AD4 数据，同时辅以两项独立后期试验 TRuE-AD1、TRuE-AD2 佐证；该关键研究证实，患者用药第 2 天即可观察到中度特应性皮炎各项临床体征与瘙痒症状显著改善。 Opzelura was first approved in the U.S. as a treatment for atopic dermatitis in 2021 before scoring a green light in vitiligo the following year. Over in Europe, it currently holds one approval to treat non-segmental vitiligo with facial involvement in adults and adolescents aged 12 years and older. Opzelura2021 年率先在美国获批特应性皮炎适应症，次年获批白癜风适应症；欧洲地区目前仅获批一项适应症，用于 12 岁及以上成人、青少年伴面部受累的非节段型白癜风。 Aside from a clutch of other potential label extensions in the EU, the CHMP further granted recommendations to the following new drugs: The flu vaccine Aujemflu; the Parkinson’s disease with motor fluctuations treatment Hopledo; and Onswik to treat type 2 diabetes in adults. 除多款药物有望在欧盟拓展适应症外，CHMP 还为以下三款新药出具上市推荐意见：流感疫苗 Aujemflu、用于伴运动波动帕金森病的 Hopledo、成人 2 型糖尿病治疗药物 Onswik。 And, following a reexamination of the sort MaaT is now seeking, Acadia Pharmaceuticals reversed the negative opinion it had previously received from the CHMP on its Rett syndrome treatment Daybu. 另有企业完成与 MaaT 当前申请同类的复审流程：阿卡迪亚制药用于雷特综合征的药物 Daybu 推翻此前 CHMP 给出的负面意见，本次复审获积极推荐。 The regulator also gave its blessing to a pair of biosimilars referencing Prolia and Xgeva and Neulasta. 监管机构同时批准两款生物类似药，分别对标原研药普罗力 / 地诺单抗（Prolia/Xgeva）、培非格司亭（Neulasta）。

喜欢我的分享，点击上方 关注我吧！ 2026 年 6 月 22–25 日 人用医药产品委员会（CHMP）会议要点 2026 年 6 月人用医药产品委员会（CHMP）会议议程已发布于欧洲药品管理局（EMA）官网；本次会议纪要将于未来数周内对外公示。 2026 年 6 月统计数据 ——CHMP 审评意见及撤回申请月度、累计数据 新药正面审评意见：6 份 其中普通新药 3 款、罕见病药 1 款、生物类似药 2 款； 2026 年累计：42 份 新药否定审评意见：3 份 2026 年累计：6 份 适应症拓展正面审评意见：13 份 2026 年累计：72 份 新药上市申请撤回：0 份 2026 年累计：5 份 6款新药获上市推荐许可 欧洲药品管理局人用医药产品委员会（CHMP）在 2026 年 6 月会议上，推荐六款药品获批上市。 委员会建议授予Aujemflu（灭活流感病毒表面抗原：血凝素 + 神经氨酸酶）上市许可，该疫苗用于 50 周岁及以上人群流感预防。 CHMP 建议授予Hopledo（左旋多巴 / 卡比多巴）上市许可，用于成人帕金森病，且经口服左旋多巴 - 多巴脱羧酶抑制剂方案治疗后病情控制不佳、伴中重度运动波动（运动功能异常）患者。 Onswik（艾夫西托阿法胰岛素）获 CHMP 正面审评意见，适应症为成人 2 型糖尿病。 委员会对2款生物类似药出具支持意见： Denosumab Ascend（地舒单抗）：用于伴骨转移晚期实体瘤成人患者骨相关并发症预防；以及成人、骨骼发育成熟青少年骨巨细胞瘤治疗。 Nylaspeg（培非格司亭）：用于缩短化疗所致中性粒细胞减少症（中性粒细胞为一类白细胞）持续时间，降低发热性中性粒细胞减少症发生率。 复审结果 经复审后，委员会建议授予Daybu（曲非奈肽）上市许可，用于 5 周岁及以上儿童与成人雷特综合征神经行为相关症状治疗。相关神经行为症状包括手部重复刻板动作、躁动、情绪异常、焦虑、睡眠障碍及沟通障碍。 CHMP 最初曾驳回本品用于雷特综合征的上市申请；本次复审后同意批准上市，但适应症范围受限。 如需了解本次复审详细结论，可查阅下方表格配套问答文件。 三款药品获否定审评意见 委员会对三款药品出具不予批准意见： Tacquell（自体黑色素瘤来源肿瘤浸润淋巴细胞，体外扩增）：用于晚期黑色素瘤（已发生全身转移的皮肤恶性肿瘤）。 Yartemlea（那索普利单抗）：用于 2 周岁及以上造血干细胞移植（HSCT）相关血栓性微血管病成人及儿童患者。血栓性微血管病是造血干细胞移植后可危及生命的严重并发症；造血干细胞移植指替换患者自身骨髓，植入供者干细胞重建可生成健康血细胞的骨髓。 Xervyteg（混合异体粪便微生物制剂）：用于急性移植物抗宿主病（aGvHD）。该病为移植术后高危致命病症，骨髓 / 干细胞移植后供体细胞攻击受者机体。 如需了解上述三款药品不予批准的详细说明，可查阅下方表格配套问答文件。 、 12 款药品获批拓展治疗适应症 CHMP 出具正面意见，建议修订Imvanex上市许可条款。本品为天花、猴痘及痘苗病毒（痘病毒科另一毒株）预防疫苗，本次建议适用人群最低年龄下调至 2 周岁及以上（原 12 周岁及以上）。 委员会同时建议欧盟境内已上市的另外 11 款药品新增适应症，分别为：Datroway、Jaypirca、Leqvio、Menquadfi、Opzelura、Orladeyo、Rezolsta、Rinvoq、Stelara、Symtuza、Tecvayli。 两项适应症拓展申请撤回 两款复方制剂拓展适应症申请已撤回：DuoResp Spiromax、BiResp Spiromax（布地奈德 / 福莫特罗）原申请新增 “仅用于轻度哮喘急救缓解治疗” 的适应症。 启动欧盟法规第 31 条集中审评程序 委员会启动对利福平口服混悬液 / 糖浆（20 毫克 / 毫升） 的专项审评。本品含抗生素利福平，用于结核病及其他重度感染治疗。 启动审评原因是监管方担忧辅料二乙醇胺（DEA）含量存在安全隐患；啮齿动物长期高剂量暴露试验提示二乙醇胺可能具有致癌性。 本次审评由荷兰药品管理局依据《欧盟指令 2001/83/EC》第 31 条提请启动，相关启动通告详见下方表格。 集中审评程序收尾结论 CHMP 完成对Tavneos（阿伐可泮） 的全部审评工作，建议欧盟撤销该药品上市许可，现有证据无法证实其临床获益大于用药风险。本品用于成人重度活动性肉芽肿性多血管炎（GPA）、显微镜下多血管炎（MPA），二者均为罕见血管炎症。本次专项审评启动，是因新出现信息对支撑本品欧盟获批的核心临床试验数据完整性提出重大质疑。 其他更新事项 CHMP 批准Fluenz（鼻喷减毒活流感疫苗）可由非医疗专业人员在医护人员监督下完成接种。本品为鼻喷剂型，用于 2 周岁至 18 周岁以下儿童青少年季节性流感预防。 委员会建议修订Ixchiq（减毒基孔肯雅疫苗）上市许可条款，限定仅适用于 12 周岁及以上、基孔肯雅病毒高感染风险人群。 CHMP 完成Mounjaro（替尔泊肽） 新增适应症申请审评：原申请拓展适应症为合并心血管疾病的成人 2 型糖尿病，用于降低主要不良心血管事件（心、循环系统严重病变）风险。委员会未批准该新增适应症，但同意将申报配套研究数据纳入药品说明书，确保临床医师可查阅替尔泊肽在合并心血管疾病 2 型糖尿病人群中的完整研究数据。 【关键专业术语注解】 marketing authorisation：上市许可 positive/negative opinion：正面审评意见 / 不予批准（否定）意见 biosimilar medicines：生物类似药 re-examination：上市申请复审 restricted indication：受限适应症 referral：欧盟集中专项审评程序 excipient：药用辅料 possible carcinogen：潜在致癌物 product information：药品说明书（欧盟统一称谓） febrile neutropenia：发热性中性粒细胞减少症 autologous /allogeneic：自体 / 异体 graft-versus-host disease (GvHD)：移植物抗宿主病 cardiovascular events：心血管不良事件 reliever-only treatment：仅急救缓解治疗 live nasal influenza vaccine：鼻喷减毒活流感疫苗 Agenda：会议议程 minutes：（官方）会议纪要（药监机构固定专用表述，非普通分钟） 原文延伸： https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-22-25-june-2026 内容来源于EMA,翻译仅供参考学习 希望搭建一个理性交流的平台，与志同道合者共同探讨化工的未来图景。    ——专注化工领域   懂您的需求—— 化工综合服务提供商 企业官网建设|产品国内外推广|撮合交易|行情资讯|供应链金融 业务联系：潘经理    手     机：13958177281(微信同号） 邮     箱：472097288@qq.com 加我微信，获取更多福利 化工潘说——化工一起说  关注我，获取更多信息！