Nadofaragene firadenovec-vncg

PARSIPPANY, N.J.--(BUSINESS WIRE)--Ferring Pharmaceuticals announced today that three abstracts featuring ADSTILADRIN® (nadofaragene firadenovec-vncg) will be presented at the 101st Annual Meeting of the Western Section of the American Urological Association (AUA) being held November 2-6 in Napa, CA. ADSTILADRIN is the first and only intravesical non-replicating gene therapy approved by the U.S. Food and Drug Administration (FDA) for patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1). A Ferring-sponsored study will present baseline characteristics and demographics from the initial patient cohort enrolled in ABLE (ADSTILADRIN in BLadder CancEr)-41, a Phase 4 multicenter non-interventional study examining the use of ADSTILADRIN in a real-world setting. In addition, two independent real-world studies will be presented. This includes a study analyzing electronic billing records for 101 patients from 19 U.S. medical centers who received ADSTILADRIN and is exploring durability of remaining on ADSTILADRIN, and lack of progression to radical cystectomy. A third abstract is a case series evaluating re-induction with ADSTILADRIN among 17 patients across 8 academic and community settings, the findings of which were published recently in The Journal of Urology. “Real-world evidence provides an important view of how a treatment like ADSTILADRIN is being used in routine practice,” said Sia Daneshmand, MD, Professor of Urology and Medicine (Oncology) and Director of Urologic Oncology at Keck School of Medicine of University of Southern California. “The ABLE-41 study will add to this evidence by capturing early insights from a broader, more diverse patient population than prior trials, helping us understand patient experiences and inform care for those with BCG-unresponsive NMIBC.” “ADSTILADRIN is an important option for uro-oncologists, offering an effective and convenient quarterly dosing schedule administered in the urologist’s office, and a non-chemotherapy alternative to radical cystectomy,” said Daniel A. Shoskes, MD, FRCS(C), Vice President, Global Medical Director- Uro-Oncology, Ferring Pharmaceuticals. “The ongoing research exploring the real-world use of ADSTILADRIN adds to the body of evidence in NMIBC patients who no longer respond to BCG. I am thrilled that this research is also exploring treatment re-induction, helping to build on the strong foundation of our Phase 3 program and further inform care for NMIBC patients.” About ADSTILADRIN Presentations at the Western Section AUA ADSTILADRIN poster titles and presentation times at 101st Annual Meeting of the Western Section AUA, November 2-6, 2025, are: ABLE-41, a real-world evidence study for bladder cancer patients treated with nadofaragene firadenovec: baseline patient characteristics and demographics. Abstract #257 (Poster #23), November 2, 2025, 7:00-9:00 a.m. PST Real-world utilization of ADSTILADRIN in patients with BCG-unresponsive non-muscle invasive bladder cancer. Abstract #242 (Poster #18), November 2, 2025, 7:00-9:00 a.m. PST Re-induction with nadofaragene firadenovec for BCG-unresponsive NMIBC: real-world data from a multicenter cohort. Abstract #301 (Poster #11), November 2, 2025, 7:00-9:00 a.m. PST About ABLE-41 ABLE-41 is a non-interventional study following NMIBC patients aged 18 years or older who are being treated with ADSTILADRIN in a clinical setting and had not previously received this therapy in a clinical trial. In September 2023, Ferring announced the first patient enrolled in ABLE-41. Eligible patients include those who were prescribed and scheduled to receive treatment, per their physician’s discretion, or patients who received their first instillation (per physician discretion) after September 5, 2023, but before the site was activated in the trial. The primary objective is to assess whether patients achieve a complete response (CR) at three months and/or at any time within a year of their first instillation. Participants in ABLE-41 will be followed for 24 months, or until study discontinuation or withdrawal. Key secondary outcomes include the following: treatment patterns of use; duration of CR; recurrence-free survival, cystectomy-free survival, progression-free survival, overall survival, and bladder cancer–specific mortality; patient, caregiver, and physician experiences; adjunctive use of molecular markers; and safety. Patient experiences will be assessed with a commonly used quality of life questionnaire (EuroQol 5 Dimension 5 Level), measuring mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Caregiver experiences will be measured with the Work Productivity and Activity Impairment questionnaire, adapted for caregiving, which assesses the impact of health problems on paid and unpaid work. Results from this prospective, multi-institutional study are expected at the end of 2026. Learn more at www.clinicaltrials.gov/study/NCT06026332. About ADSTILADRIN ADSTILADRIN® (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical non-replicating gene-therapy for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. It is a non-replicating adenovirus vector-based therapy containing the gene interferon alfa-2b, administered locally as a monotherapy by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder’s cell walls to secrete high and transient local expression of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body’s own natural defenses against the cancer. ADSTILADRIN has been studied in a clinical trial program that includes 157 patients with high-risk, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849 and final five-year follow-up analysis published in The Journal of Urology®).1-2 About Ferring Uro-Oncology Ferring is committed to investing in novel therapies, developing life-changing solutions that address unmet medical needs, and aiding the uro-oncology community in helping patients live better lives. More information is available in the U.S. at FerringUroOncology.com and on the dedicated Ferring Uro-Oncology channels on LinkedIn and X. About Non-Muscle Invasive Bladder Cancer (NMIBC) NMIBC is a form of bladder cancer that is found in the inner layer cells of the bladder and does not invade into or beyond the muscle wall.3 In the United States, bladder cancer is the sixth most common cancer,4 fourth among men,5 and it is estimated that there will be approximately 84,870 new cases of bladder cancer in the U.S. in 2025.5 Historically, 75% of bladder cancer presents as NMIBC.6 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard-of-care. However, approximately one third of patients with NMIBC will not respond to BCG therapy and 50% of those with an initial response will experience recurrence or progression of their disease.7 Current treatment options for BCG-unresponsive patients are very limited, and National Comprehensive Cancer Network (NCCN) guidelines recommend cystectomy (partial or complete removal of the bladder).8 INDICATION ADSTILADRIN is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS: ADSTILADRIN is contraindicated in patients with prior hypersensitivity reactions to interferon alfa or to any component of the product. WARNINGS AND PRECAUTIONS: DOSAGE AND ADMINISTRATION: Administer ADSTILADRIN by intravesical instillation only. ADSTILADRIN is not for intravenous use, topical use, or oral administration. USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 3 months after the last dose. ADVERSE REACTIONS: The most common (>10%) adverse reactions, including laboratory abnormalities (>15%), were glucose increased, instillation site discharge, triglycerides increased, fatigue, bladder spasm, micturition (urination urgency), creatinine increased, hematuria (blood in urine), phosphate decreased, chills, pyrexia (fever), and dysuria (painful urination). You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.FDA.gov/medwatch or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING. Please click to see the full Prescribing Information. About Ferring Pharmaceuticals Ferring Pharmaceuticals is a privately-owned, specialty biopharmaceutical group committed to building families and helping people live better lives. In the United States, Ferring is a leader in reproductive medicine, and in areas of gastroenterology and orthopaedics. We are at the forefront of innovation in microbiome-based therapeutics and uro-oncology intravesical gene therapy. Our company was founded in 1950 and is headquartered in Saint-Prex, Switzerland. Ferring employs more than 7,000 people worldwide and markets its medicines in over 100 countries. Ferring USA is based in Parsippany, New Jersey, and employs more than 900 employees. For more information, please visit www.ferringusa.com, call 1-888-FERRING (1-888-337-7464), or connect with us on LinkedIn, and X. References: ADSTILADRIN in Patients With High-Grade, Bacillus Calmette-Guerin (BCG) Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC). Gov Identifier: NCT02773849. Available at: https://clinicaltrials.gov/ct2/show/NCT02773849 . Accessed October 27, 2025. Narayan VM, Boorjian SA, Alemozaffar M, et al. Efficacy of intravesical nadofaragene firadenovec for patients with Bacillus Calmette-Guérin-unresponsive nonmuscle-invasive bladder cancer: 5-year follow-up from a phase 3 trial. J Urol. 2024;212(1):1-12. Urology Care Foundation. Non-muscle Invasive Bladder Cancer. Available at: https://www.urologyhealth.org/urology-a-z/n/non-muscle-invasive-bladder-cancer . Accessed October 27, 2025. National Cancer Institute. Cancer Statistics. Available at: https://www.cancer.gov/about-cancer/understanding/statistics#:~:text=The%20most%20common%20cancers%20%28listed%20in%20descending%20order,pancreatic%20cancer%2C%20leukemia%2C%20thyroid%20cancer%2C%20and%20liver%20cancer . Accessed October 27, 2025. American Cancer Society. Cancer Facts & Figures 2025. Available at: https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/annual-cancer-facts-and-figures/2025/2025-cancer-facts-and-figures-acs.pdf . Accessed October 27, 2025. Babjuk M, Burger M, Capoun O, et al. European Association of Urology Guidelines on Non-muscle-invasive Bladder Cancer. Eur Urol. 2022 Jan;81(1):75-94. Lidagoster S, et al. BCG and Alternative Therapies to BCG Therapy for Non-Muscle-Invasive Bladder Cancer. Curr Oncol. 2024 Feb 16;31(2):1063-1078. National Comprehensive Cancer Network. Bladder Cancer (Version 4.2024). Available at: https://www.nccn.org/professionals/physician_gls/pdf/bladder.pdf . Accessed October 27, 2025. More information is available at the following: Healthcare Providers: If you are interested in ordering ADSTILADRIN, please sign up for information and updates at www.ADSTILADRINHCP.com . Patients and Consumers: For more information about ADSTILADRIN, please visit www.ADSTILADRIN.com , or call 1-888-FERRING (888-337-7464), and select option number one. Media: Members of the press can contact Carol Ready , Director, Brand Communications by phone at (973) 765-7307, or email at carol.ready@ferring.com .

▲2004页2025 WCLC摘要集免费领取！ 2025年9月9日，强生宣布，其创新疗法INLEXZO®（吉西他滨膀胱内释放系统，TAR-200）获得美国食品药品监督管理局（FDA）批准上市，用于治疗拒绝或不适合接受根治性膀胱切除术、对卡介苗（BCG）无反应、伴有原位癌（CIS）伴或不伴乳头状肿瘤的非肌层浸润性膀胱癌（NMIBC）成人患者。 该疗法凭借其创新的给药方式和卓越的临床数据，为这个四十余年缺乏突破的治疗领域带来了颠覆性的改变，让希望保留膀胱的患者看到了新的曙光。 1 关于BCG无反应NMIBC 非肌层浸润性膀胱癌（NMIBC）是最常见的膀胱癌类型，约占所有新发病例的75%。其中，约10%的NMIBC患者伴有原位癌（CIS），这是一种高风险、扁平状、高侵袭性的病变。目前，膀胱内灌注卡介苗（BCG）是高风险NMIBC的标准一线治疗方案。 然而，高达40%的患者对BCG治疗无反应或会在治疗后复发。对于这类“BCG无反应”的高危NMIBC患者，国际指南推荐的标准治疗方案是根治性膀胱切除术，即手术切除整个膀胱。这是一个重大的、不可逆的、且伴随显著风险和并发症的手术，术后死亡率在3%-8%之间。 此外，NMIBC好发于老年患者，许多人因年龄、合并症或个人意愿，无法或不愿接受此根治性手术。因此，临床上亟需一种能有效保留膀胱、替代手术的有效挽救性治疗方案。 2 新型给药系统INLEXZO INLEXZO并非一种新分子实体药物，而是一种革命性的膀胱内药物释放系统（iDRS）。其活性成分是经典的化疗药物吉西他滨，但INLEXZO通过一种精巧的装置，彻底改变了它的递送方式。 该系统由一个微型、可弯曲的硅胶管组成，内部充满吉西他滨，两端用半透膜密封。在门诊环境中，医生通过专用的导尿管和管心针，可在短短几分钟内将其经尿道植入膀胱内，无需全身麻醉或住院。一旦就位，膀胱内的尿液会通过半透膜缓慢渗入装置，溶解内部的吉西他滨，药物再以可控的速率持续释放到膀胱腔内。 这种设计实现了长达数周的局部、持续、高浓度的药物暴露，从而能更持久、更强效地杀伤癌细胞，同时最大程度地避免了吉西他滨经全身吸收所带来的骨髓抑制等副作用。每个治疗周期装置留置约3周，最多可进行14个周期治疗。 3 关键临床试验数据 FDA的批准主要基于关键性IIb期SunRISe-1研究队列2的积极结果。这是一项单臂、开放标签的临床试验，旨在评估INLEXZO单药治疗BCG无反应、伴CIS的NMIBC患者的疗效和安全性。 研究结果显示，经INLEXZO治疗后，82.4%（95% CI: 72-90）的患者达到了完全缓解（CR），即在膀胱镜检查和活检中未再检测到任何癌症迹象。更令人振奋的是，这种缓解表现出强大的持久性。在达到CR的患者中，有51%的患者其完全缓解状态维持了至少12个月。 这一数据在当前的治疗格局下堪称突破，为大量原本只能选择手术的患者提供了一个高效且可保留器官的替代方案。 安全性方面，INLEXZO的安全性特征良好。最常见的不良反应（发生率≥15%）主要集中于局部和实验室指标，如尿频、尿路感染、排尿困难、尿急、血尿、膀胱刺激以及一过性的实验室指标异常（如血红蛋白降低、肌酐升高等）。这些反应大多与药物在膀胱局的局部作用相关，且通常可控，未发现新的重大安全性信号。 4 创新机制独占鳌头 在BCG无反应NMIBC的治疗领域，INLEXZO的获批使其占据了独特的竞争地位。 传统治疗方面，BCG灌注后失败，标准选择即为根治性膀胱切除术。其他化疗药物（如吉西他滨、多西他赛）的膀胱灌注疗效有限且不持久。 免疫治疗方面，默沙东的帕博利珠单抗（Keytruda）已获FDA批准用于同一人群，其完全缓解率（CR率）约在41%左右，但缓解持续时间中位数约为13个月。INLEXZO在CR率上展现出显著优势（82.4% vs ~41%）。 基因疗法方面，Ferring Pharmaceuticals的腺病毒载体基因疗法Adstiladrin®（nadofaragene firadenovec-vncg）也已获批，其三个月时的CR率为51%，疗效可持续约半年。INLEXZO在缓解率和持久性上同样显示出优势。 创新给药系统方面，INLEXZO是全球首个获批的、能够在膀胱内持续数周释放化疗药物的腔内给药系统，这一创新机制目前尚无直接竞品。 因此，INLEXZO凭借其卓越的疗效数据和创新的给药方式，在该治疗领域建立了强大的竞争力。 结 语 强生INLEXZO的获批，是膀胱癌治疗领域一个值得载入史册的里程碑。 它不仅仅是一款新药或新设备的上市，更是为BCG无反应的高危NMIBC患者提供了一个有效且可保留膀胱的“手术替代”方案。其惊人的82.4%完全缓解率，让医生和患者在面对BCG治疗失败这一棘手难题时，首次拥有了一个比肩甚至可能优于现有所有非手术方案的强大武器。随着INLEXZO的应用，预计将有很大一部分患者得以避免膀胱切除术，在有效控制癌症的同时，维持正常排尿功能和身体形象，重获高质量的生活。这不仅是一次技术的胜利，更是对患者生命尊严和人文关怀的深刻体现。 膀胱癌的治疗新时代，已然开启。 识别微信二维码，添加抗体圈小编，符合条件者即可加入抗体圈微信群！ 请注明：姓名+研究方向！ 本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。