FDA approves gemcitabine intravesical system for non-muscle invasive bladder cancer
正文 · 中文译文
FDA批准吉西他滨膀胱内灌注系统用于非肌层浸润性膀胱癌 癌症批准 肿瘤（癌症）/血液系统恶性肿瘤批准通知 进行中 | 癌症加速批准 已验证临床获益 | 癌症加速批准 已撤回 | 癌症加速批准 其他 | 癌症加速批准 2025年9月9日，美国食品药品监督管理局批准吉西他滨膀胱内灌注系统（Inlexzo，Janssen Biotech， Inc.）用于成人卡介苗（BCG）无应答的非肌层浸润性膀胱癌（NMIBC）伴原位癌（CIS），伴或不伴乳头状肿瘤。吉西他滨膀胱内灌注系统与导尿管及用于经导尿管插入膀胱的探条共同包装。 Inlexzo的完整处方信息将在Drugs@FDA上发布。 疗效在SunRISe-1（NCT04640623）第2队列中评估，该单臂、多中心试验入组83例经尿道切除术后BCG无应答的NMIBC伴CIS、伴或不伴乳头状肿瘤患者。患者接受吉西他滨膀胱内灌注系统注入膀胱，每3周一次，持续6个月，随后每12周一次，最长18个月。 在治疗最初两年内，每12周通过膀胱镜检查和尿细胞学评估肿瘤状态，之后至少每24周进行膀胱镜检查。在治疗开始后第24周和第48周进行强制性活检。 主要疗效结局指标为任何时间的完全缓解（CR）（定义为膀胱镜检查［酌情行经尿道膀胱肿瘤电切术（TURBT）/活检］和尿细胞学结果均为阴性）以及缓解持续时间（DOR）。各时间点的CR评估均基于中心病理学审查。 CR率为82%（95% CI：72，90），51%的CR患者DOR≥12个月。 处方信息包括警告和注意事项，描述了在膀胱穿孔患者中给予膀胱内吉西他滨系统的风险、延迟膀胱切除术导致转移性膀胱癌的风险、磁共振成像（MRI）安全性以及胚胎-胎儿毒性。 膀胱内吉西他滨系统向膀胱内递送225 mg吉西他滨，每次置入后留置3周再取出。膀胱内吉西他滨系统每3周置入一次，最长6个月（8剂），随后每12周一次，最长18个月（6剂），或直至持续或复发的高级别非肌层浸润性膀胱癌（NMIBC）、疾病进展或不可接受的毒性。 本审评采用了实时肿瘤学审评（RTOR）试点项目，该项目在完整临床应用申报前简化了数据提交，并采用了评估辅助工具（Assessment Aid），这是申请人自愿提交以促进FDA评估的工具。 该申请获得了优先审评资格。吉西他滨膀胱内灌注系统获得了突破性疗法认定。FDA加速审评项目在《行业指南：严重疾病药物和生物制品的加速审评项目》中有详细描述。 医疗专业人员应将所有怀疑与任何药物和器械使用相关的严重不良事件报告至FDA的MedWatch报告系统，或致电1-800-FDA-1088。 如需关于研究性肿瘤产品单患者IND的协助，医疗专业人员可联系OCE的Project Facilitate，电话240-402-0004，或发送电子邮件至OncProjectFacilitate@fda.hhs.gov。 在X上关注肿瘤学卓越中心：@FDAOncology。 内容更新日期： 09/09/2025 受监管产品 药物 肿瘤学
正文 · 英文原文
FDA approves gemcitabine intravesical system for non-muscle invasive bladder cancer Cancer Approvals Oncology (Cancer)/Hematologic Malignancies Approval Notifications Ongoing | Cancer Accelerated Approvals Verified Clinical Benefit | Cancer Accelerated Approvals Withdrawn | Cancer Accelerated Approvals Other | Cancer Accelerated Approvals On September 9, 2025, the Food and Drug Administration approved gemcitabine intravesical system (Inlexzo, Janssen Biotech, Inc.) for adults with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. Gemcitabine intravesical system is co-packaged with a urinary catheter and stylet used for insertion through the urinary catheter into the bladder. Full prescribing information for Inlexzo will be posted on Drugs@FDA. Efficacy was evaluated in Cohort 2 of SunRISe-1 (NCT04640623), a single-arm, multi-center trial enrolling 83 patients with BCG-unresponsive NMIBC with CIS with or without papillary tumors following transurethral resection. Patients received the gemcitabine intravesical system into the bladder every 3 weeks for 6 months, followed by once every 12 weeks for up to 18 months. Tumor status was assessed with cystoscopy and urine cytology every 12 weeks during the initial two years of treatment, after which cystoscopy was performed at least every 24 weeks. Mandatory biopsies were performed at 24 and 48 weeks after treatment initiation. The major efficacy outcome measures were complete response (CR) at any time (defined as negative results for cystoscopy [with TURBT/biopsies as applicable] and urine cytology) and duration of response (DOR). CR assessment at each timepoint was based on central pathology review. The CR rate was 82% (95% CI: 72, 90) and 51% of patients with a CR had a DOR ≥ 12 months. The prescribing information includes warnings and precautions describing risks of administering gemcitabine intravesical system in patients with a perforated bladder, risk of metastatic bladder cancer with delayed cystectomy, magnetic resonance imaging (MRI) safety, and embryo-fetal toxicity. The gemcitabine intravesical system delivers 225 mg of gemcitabine into the bladder, with an indwelling period following each insertion of 3 weeks prior to removal. The gemcitabine intravesical system is inserted once every 3 weeks for up to 6 months (8 doses), followed by once every 12 weeks for up to 18 months (6 doses), or until persistent or recurrent high-grade NMIBC, disease progression, or unacceptable toxicity. This review used the Real-Time Oncology Review (RTOR) pilot program, which streamlined data submission prior to the filing of the entire clinical application, and the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment. This application was granted priority review. The gemcitabine intravesical system received breakthrough therapy designation. FDA expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics. Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088. For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov. Follow the Oncology Center of Excellence on X: @FDAOncology. Content current as of: 09/09/2025 Regulated Product(s) Drugs Oncology
摘要 待译
On September 9, 2025, the Food and Drug Administration approved gemcitabine intravesical system (Inlexzo, Janssen Biotech, Inc.) for adults with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors