A Single-Arm, Open-Label, Phase I Study to Determine the Safety, Tolerability and Preliminary Efficacy of Obecabtagene Autoleucel in Patients With Severe, Refractory Systemic Lupus Erythematosus

This is a Phase I study of obecabtagene autoleucel (obe-cel), autologous T cells engineered with a chimeric antigen receptor (CAR) targeting CD19, to establish the tolerability, safety, preliminary efficacy, and pharmacokinetics of obe-cel in patients with severe, refractory SLE.

开始日期2024-02-02

申办/合作机构

Autolus Ltd.

NCT06173518

/ Recruiting临床1期

A Single-Arm, Open-Label, Multi-Centre, Phase Ib Study Evaluating the Safety and Preliminary Efficacy of AUTO1 in Pediatric Patients With CD19-Positive Relapsed/ Refractory (r/r) B Cell Acute Lymphoblastic Leukemia (B ALL) and Aggressive Mature B Cell Non-Hodgkin Lymphoma (B NHL)

This is a Phase Ib study to evaluate the safety and efficacy of autologous T cells engineered with a chimeric antigen receptor (CAR) targeting cluster of differentiation (CD)19 in pediatric patients with relapsed or refractory (r/r) B cell acute lymphoblastic leukemia (B ALL) and r/r B cell Non-Hodgkin lymphoma (B NHL)

开始日期2023-11-16

申办/合作机构

Autolus Ltd.

ACTRN12621000762853

/ Recruiting临床1期IIT

Phase I Clinical Trial of MB.CART19.1 CD19 Chimeric Antigen Receptor (CAR)T Cells in Relapsed or Refractory CD19-Positive Haematological Malignancy

开始日期2021-07-21

申办/合作机构

Metro North Hospital & Health Service

100 项与欧贝妥基奥仑赛相关的临床结果

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100 项与欧贝妥基奥仑赛相关的转化医学

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100 项与欧贝妥基奥仑赛相关的专利（医药）

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项与欧贝妥基奥仑赛相关的文献（医药）

2025-05-12·LEUKEMIA & LYMPHOMA

Frontline immunotherapeutic combination strategies in adult B-cell acute lymphoblastic leukemia: reducing chemotherapy intensity and toxicity and harnessing efficacy

Review

作者: Haddad, Fadi G. ; Habib, Diane ; Senapati, Jayastu ; Short, Nicholas J. ; Kantarjian, Hagop ; Jabbour, Elias ; Jain, Nitin

Using immunotherapeutic agents like inotuzumab ozogamicin (InO), blinatumomab, or chimeric antigen receptor T (CAR T)-cell therapy in frontline adult B-cell acute lymphoblastic leukemia (B-ALL) therapy is promising. These agents are mostly well tolerated and have different toxicity profiles than conventional chemotherapy, enabling their combination with chemotherapy. Additionally, they have often been shown to overcome the traditional adverse ALL risk features. Recently blinatumomab was approved as part of consolidation therapy in MRD negative B-ALL; however, a significant proportion of patients had progressed or relapsed before reaching the timepoint of blinatumomab administration. Including InO/blinatumomab from induction onwards could induce earlier and deeper remissions. Modifications of dosing and administration schedules, as with the fractionated InO schedule with low-intensity chemotherapy, and subcutaneous blinatumomab, appear to reduce the toxicity and improve the anti-ALL efficacy. CAR T-cell therapies like brexucabtagene autoleucel as a consolidation approach have shown positive outcomes. The feasibility of using CAR T-cells to reduce the need for long-drawn maintenance and the need for allogeneic hematopoietic stem cell transplantation (HSCT) are questions of ongoing clinical trials. Newer generation CAR T-cell products like obecabtagene autoleucel appear as effective and safer. Better disease monitoring through next generation sequencing based measurable residual disease analysis could identify patients where treatment intensification including HSCT, or deintensification, is suitable.

2025-05-01·Molecular Diagnosis & Therapy

Obecabtagene Autoleucel: First Approval

Review

作者: Lee, Arnold

Obecabtagene autoleucel (AUCATZYL^®) is a CD19-directed genetically modified autologous T cell immunotherapy which is being developed by Autolus for the treatment of hematological cancers and systemic lupus erythematosus. In comparison with other chimeric antigen receptor T (CAR T) therapies, obecabtagene autoleucel has a fast off-rate binder for CD19. Obecabtagene autoleucel received approval following positive results from the FELIX phase I/II trial in adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL), and it is the first CAR T therapy that does not have mandatory Risk Evaluation Mitigation Strategy monitoring requirements. This article summarizes the milestones in the development of obecabtagene autoleucel leading to this first approval for the treatment of adults with relapsed or refractory B-cell precursor ALL.

2025-04-22·Blood Advances

Phase 1 study of CD19 CAR T-cell therapy harboring a fully human scFv in CAR-naïve adult patients with B-ALL

Article

作者: Kimble, Erik L. ; Fiorenza, Salvatore ; Riddell, Stanley R. ; Braathen, Kristina ; Wu, Qian (Vicky) ; Cassaday, Ryan D. ; Maloney, David G. ; Huang, Jennifer J. ; Kopmar, Noam E. ; Basom, Ryan ; Gauthier, Jordan ; Turtle, Cameron J. ; Jaeger-Ruckstuhl, Carla A. ; Liang, Emily C. ; Voutsinas, Jenna M. ; Kirchmeier, Delaney R. ; Torkelson, Aiko ; Pender, Barbara S. ; Shadman, Mazyar ; Hirayama, Alexandre V.

Abstract:

CD19-directed chimeric antigen receptor–engineered (CAR) T-cell therapy elicits high response rates but fails to induce durable responses in most adults with relapsed or refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL). In a previous clinical trial, we observed anti-CAR immune responses associated with impaired in vivo CAR T-cell expansion after second infusions. Because these CD8+ T-cell responses were predominantly directed at peptides derived from the murine single-chain variable fragment (scFv) in the CAR, we conducted a clinical trial investigating the safety and efficacy of CD19 CAR T-cells engineered with a CAR incorporating a fully human scFv (JCAR021) in adults with R/R B-ALL (NCT03103971). Twenty-three patients received lymphodepletion chemotherapy and JCAR021 infusion. Nineteen patients developed cytokine release syndrome (any grade, 83%; grade 2, 61%) and 12 developed neurotoxicity (52%; grade ≥3, 35%). The overall response and complete response (CR)/CR with incomplete hematologic recovery (CRi) rates were 82% and 64%, respectively. We observed measurable residual disease–negative bone marrow (BM) responses in 82% of those with BM disease and extramedullary responses by positron emission tomography–computed tomography in 79% (CR, 50%) of those with measurable fluorodeoxyglucose-avid disease. The median duration of remission (DOR) was 10 months with a 4-year DOR probability of 29%. Four patients underwent allogeneic hematopoietic cell transplantation while in CR/CRi after JCAR021. Durable remissions were observed in patients with low BM disease burden. In contrast, the DOR was limited in those with high BM burden. We observed similar outcomes in CAR-naïve adult patients with B-ALL receiving CD19 CAR T cells expressing a fully human or murine scFv-containing CAR. This trial was registered at www.ClinicalTrials.gov as #NCT03103971.

106

项与欧贝妥基奥仑赛相关的新闻（医药）

2025-05-23

·FiercePharma

On the comeback trail, GSK's Blenrep scores thumbs-up from Europe's CHMP

Europe's CHMP has recommended 10 drugs for marketing authorization, including GSK's cancer treatment Blenrep, which has also picked up recent nods from regulators in Japan and the U.K. Who doesn’t love a comeback story? Certainly not GSK, which has found a way to resuscitate its cancer treatment Blenrep as a combo agent after it was pulled from the U.S. market as a monotherapy in late 2022.This week, Europe’s Committee for Medicinal Products for Human Use (CHMP) recommended Blenrep for approval for patients with relapsed or refractory multiple myeloma.The CHMP has given Blenrep a thumbs-up in two combinations for patients who have received at least one prior therapy including Bristol Myers Squibb’s Revlimid (lenalidomide). One combo pairs Blenrep with the protease inhibitor Velcade (bortezomib) plus the corticosteroid dexamethasone. The other regimen includes Blenrep with BMS's Pomalyst (pomalidomide) and dexamethasone.In 2020, the FDA signed off on the antibody-drug conjugate as a monotherapy for patients who had tried at least four other treatments, but two years later, Blenrep came up short in a confirmatory trial.Since then, Blenrep has emerged victorious in two phase 3 trials, topping standard of care as a combo agent. In both trials, the Blenrep regimen showed a statistically significant and clinically meaningful edge in improving patients' progression-free survival.Last month, Blenrep earned an approval in the U.K. Then earlier this week, officials in Japan endorsed the GSK regimen. The FDA has a target date of July 23 to decide on the Blenrep combo.Novartis pulls Lutathera request Meanwhile, Novartis’ bid to expand the label of its radiotherapy Lutathera in Europe is on hold after the company withdrew its filing, according to the CHMP. The committee explained that Novartis made the decision after “there were still some unresolved issues,” after the company answered a round of questions about the application, which was backed by a trial of 226 patients.The CHMP said that while the study found that Lutathera increased the amount of time patients lived without disease progression, “its impact on extending patients’ lives had not been established.” The committee concluded that the benefits of the treatment did not outweigh the risks. Lutathera has been on the market for eight years as a treatment for rare cancers of the digestive tract. The company had been seeking approval for Lutathera to treat adults with newly diagnosed unresectable or metastatic, well-differentiated high-grade somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors.Other CHMP decisions The CHMP also signed off on Autolus Therapeutics’ Aucatzyl as a treatment for relapsed or refractory B-cell precursor acute lymphoblastic leukemia. The FDA endorsed Aucatzyl in the indication in November of last year.Also making the grade at this week's CHMP meeting was SpringWorks Therapeutics’ MEK inhibitor Ezmekly for patients with symptomatic, inoperable neurofibromatosis type 1–associated plexiform neurofibromas (NF1-PN), which lead to tumor growth inside and along the nerves. The recommendation follows a February approval for the treatment in the U.S., where it goes by the commercial name of Gomekli. The CHMP also blessed Roche’s Itovebi as a combo treatment with palbociclib and fulvestrant as a first-line regimen for PIK3CA-mutated, estrogen-receptor (ER)-positive, HER2-negative locally advanced and metastatic breast cancer. The combo scored a nod from the FDA in October of last year.Receiving a thumbs-down from the CHMP were 4SC’s Kinselby, for the treatment of advanced stage mycosis fungoides and Sezary syndrome, and Fresenius Kabi’s Atropine sulfate for children with myopia.

上市批准临床结果临床3期抗体药物偶联物加速审批

2025-05-23

·Firstwordpharma

Blenrep comeback continues with EU recommendation, plus a thumbs up for Itovebi

GSK's bid to bring back multiple myeloma therapy Blenrep (belantamab mafodotin) got a major boost on Friday when the antibody-drug conjugate (ADC) received a positive opinion from the EU's drug advisory panel. Also receiving a positive vote from the European Medicine Agency's Committee for Medicinal Products for Human Use (CHMP) was Roche's breast cancer drug Itovebi (inavolisib). Survival data for the PI3K inhibitor was recently spotlighted in an abstract released ahead of the American Society of Clinical Oncology (ASCO) meeting (see – ASCO25 abstract roundup: Roche's Itovebi cuts risk of death).Autolus' CAR-T cell therapy Aucatzyl (obecabtagene autoleucel) also received a thumbs up from CHMP, as well as mirdametinib, a MEK inhibitor recently acquired by Merck KGaA in its $3.9-billion takeout of SpringWorks Therapeutics. Blenrep is backBlenrep was withdrawn from global markets in 2022 following disappointing results from a confirmatory trial, but after positive readouts from two pivotal Phase III trials — DREAMM-7 and DREAMM-8 — GSK has been working to bring the BCMA-targeted ADC back. Specifically, CHMP recommended Blenrep to treat relapsed or refractory multiple myeloma in combination with bortezomib and dexamethasone (BVd) for adults who have received at least one prior therapy; and in combination with Bristol Myers Squibb's Pomalyst (pomalidomide) plus dexamethasone for patients who have received at least one prior treatment, including BMS's Revlimid (lenalidomide). In DREAMM-7, Blenrep plus BVd achieved a progression-free survival (PFS) of 36.6 months, versus 13.4 months for the comparator regimen of Johnson & Johnson's Darzalex (daratumumab) plus BVd.DREAMM-8 compared Blenrep to bortezomib, both in combination with Pomalyst and dexamethasone. At nearly 22 months follow-up, median PFS was not yet reached with Blenrep compared to 12.7 months in the control arm.In April, the UK became the first country to reauthorise Blenrep. Regulatory applications for the ADC are under review in a dozen other countries, including the US, Switzerland, Japan, China and Canada. The FDA is expected to make a decision by July 23, although there is concern that recent mass layoffs at the FDA could slow down the review process (see – Spotlight On: Could key drug approvals be put at risk of delay by FDA layoffs?).GSK is positioning the drug as a key growth driver, projecting peak annual sales exceeding £3 billion (see – Spotlight On: Can GSK deliver much needed pipeline momentum?).Itovebi impressesItovebi scored a positive opinion for its use in combination with palbociclib and fulvestrant to treat PIK3CA‑mutated, ER‑positive/HER2‑negative, locally advanced or metastatic breast cancer, following recurrence on or within 12 months of completing adjuvant endocrine treatment.The PI3K inhibitor won a similar approval in the US last October based on data from the Phase III INAVO120 study showing a 57% benefit for the Itovebi triplet on the primary endpoint of PFS compared to palbociclib and fulvestrant alone. Patients achieved PFS of 15 months in the experimental arm versus 7.3 months for placebo.At ASCO, Roche plans to share updated survival data for Itovebi. The abstract revealed that patients who received the triplet experienced a median overall survival of 34 months compared to 27 months for controls, representing a 33% reduction in the risk of death. Additionally, the combination therapy delayed the time to chemotherapy from 12.6 months in the control arm to 35.6 months with Itovebi.Go-ahead for Aucatzyl, mirdametinibA CHMP recommendation also went to Aucatzyl to treat patients aged 26 and older with relapsed or refractory B cell precursor acute lymphoblastic leukaemia (ALL).Autolus' CD19-directed cell therapy was cleared by the FDA in November for a similar indication, based on data from the Phase Ib/II FELIX trial. Of 65 patients with ALL who received the CAR-T, 27 (42%) achieved a complete response (CR) within three months, with a median CR duration of 14.1 months.Meanwhile, Merck KGaA's newly gained mirdametinib received a positive opinion to treat symptomatic, inoperable plexiform neurofibromas (PN) in paediatric and adult patients with neurofibromatosis type 1 (NF1) aged 2 years and above. If fully approved by the European Commission, it will have the brand name of Ezmekly.In February, the MEK inhibitor was approved in the US for the same patient population, where it's marketed as Gomekli.

临床结果上市批准临床3期并购ASCO会议

2025-04-29

·蒲公英Ouryao

一周 | 国际法规药事(04.21-04.27)

◆ ◆ ◆ ◆Paul's Insight2025年4月21日-4月27日◆ ◆ ◆ ◆过去一周（4月21日至4月27日），全球药事监管和行业机构齐齐发声，犹如一场精彩的药品政策与科技盛宴。从FDA的合成色素退市行动，到EMA的罕见病新疗法推荐；从EDQM对生物制品检测标准的升级，到MHRA多款创新疗法的批准；再到行业协会PDA和ISPE在指南与实践层面的多重发力，这一连串新闻不仅彰显了各大机构的监管力度，更映射出制药行业在创新、合规与可持续性间的不断平衡。美国FDA从食药安全到创新药物的“双轨”发力1. 合成食用色素“退场”行动4月22日，美国FDA宣布，计划逐步淘汰美国食品供应中的合成色素（Petroleum-Based Synthetic Dyes），为儿童健康撑起防护伞。FDA表示，这一举措，是“让美国再次健康”（Make America Healthy Again）计划的重要里程碑。此前，1月15日赤藓红（FD&C Red No. 3）禁用已拉开了帷幕，后续这一更广泛的色素退市，将持续推动产业寻求天然、低风险的替代方案。图：赤藓红禁用后，FDA再推新规，八大合成食用色素将退场https://www.fda.gov/news-events/press-announcements/hhs-fda-phase-out-petroleum-based-synthetic-dyes-nations-food-supply2. 药品安全敲响警钟：外用非那雄胺安全红线4月22日，FDA对市场上未经批准的外用非那雄胺配药制剂发出警示，提醒医疗机构、药房及消费者：目前无任何局部非那雄胺产品拥有FDA批准的标签和安全、有效性验证，配制药剂可能隐藏严重风险，包括与米诺地尔等混合制剂的混用场景。FDA直指部分处方药店和远程医疗平台的药品安全风险行为，呼吁行业加强自律与审查。图：就非那雄胺外用安全问题，FDA发布脱发常用药警示https://www.fda.gov/drugs/human-drug-compounding/fda-alerts-health-care-providers-compounders-and-consumers-potential-risks-associated-compounded3. 中国创新药的海外“登台”——派安普利单抗获批4月24日，FDA官网放出重磅消息：康方生物自主研发的PD1单抗派安普利单抗（Penpulimabkcqx）获批，用于复发或转移性非角化型鼻咽癌（NPC）治疗。该产品采用IgG1亚型Fc段改造设计，依托突破性疗法认定、孤儿药资格和快速通道三重加速，联袂正大天晴药业集团布局后续商业化。此举不仅为全球鼻咽癌患者带来曙光，也标志着中国创新药在国际舞台上稳步迈进。（点击此处查看相关阅读）图：中国制药行业又一里程碑，派安普利单抗获FDA批准进入美国市场https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-penpulimab-kcqx-non-keratinizing-nasopharyngeal-carcinoma美国PDA发布新闻季刊4月24日，美国注射剂协会（PDA）发布新闻季刊，总结其在过去一个季度的工作，要点如下。图：PDA发布新闻季刊https://www.pda.org/pda-letter-portal/home/full-article/community-news-quarterly-april-20251）2024年PDA出版奖揭晓PDA颁发奖项给Novartis&Ogilvy团队，表彰其用于无菌性检测的创新方法，该论文名为《一种利用固相流式细胞术进行细胞制备、培养基和缓冲液的快速无菌方法》。年度PDA Letter文章奖项授予武田制药的《理解日本品质》。2）区域分会动态：德国、奥地利和瑞士分会（DACH）亮点纷呈DACH分会“目视检测与颗粒挑战”网络研讨会吸引400余名专家，聚焦AI时代的风险基础方法与质量设计。PDA表示，此次网络研讨会超出预期，如此高的参与度凸显了业界对目视检测和颗粒物相关挑战的关注，同时也强化了DACH分会促进制药行业知识交流和最佳实践的使命。欧盟EMA欧盟罕见病与适应症扩展双管齐下1. DMD新疗法——组蛋白去乙酰化酶（HDAC）抑制剂获“有条件上市许可”4月25日，欧洲药品管理局（EMA）发布杜氏肌营养不良症（DMD）创新药物Duvyzat（givinostat）“有条件上市许可”建议。该组蛋白去乙酰化酶（HDAC）抑制剂在120例6岁以上患者的18个月研究中显著延缓台阶攀爬时间，虽次要终点未全部显著，但整体数据朝积极方向发展。获得“有条件上市许可”，这是欧盟的监管机制之一，旨在促进早期上市药物，以满足未满足医疗需求的。EMA要求申请人后续进行一项随机、安慰剂对照研究，并进行长期随访将补足盲点。上市许可人为Italfarmaco S.p.A.，是是一家总部位于意大利米兰的制药跨国公司。图：杜氏肌营养不良症（DMD）创新药物获“有条件上市许可”https://www.ema.europa.eu/en/news/new-treatment-against-duchenne-muscular-dystrophy2. 甲状腺眼病首创疗法Tepezza获积极审评意见4月25日，EMA对甲状腺眼病（TED）首款靶向IGF1受体的单抗Teprotumumab（Tepezza）给出积极评审意见。三项RCT临床显示活动期患者24周内眼球突出度平均减少2.0–2.3mm，临床活动评分大幅改善；慢性期亦有约1.5mm降低。基于肌肉痉挛、脱发等可管理风险，EMA将附加风险最小化措施并转交欧委会审批。申请人为Amgen Europe B.V.，是全球领先的生物技术公司安进在欧洲的子公司。图：甲状腺眼病首创疗法Tepezza获积极审评意见https://www.ema.europa.eu/en/news/first-treatment-against-severe-thyroid-eye-disease3. CHMP会议要点：新药、扩展、再审与撤回4月22–25日的EMA人药委员会（CHMP）会议，推荐16款新药（包含Vertex囊性纤维化三联疗法、差异化适应症的多款孤用药及九款生物类似药），推动10项适应症扩展，如武田制药的Adcetris多淋巴瘤适应等；同时，礼来的阿尔茨海默药物Kisunla申请再审，Biohaven和辉瑞的两款产品适应失利撤回。整体来看，EMA通过CMA平衡未满足需求与证据要求，也显示出对成熟产品生命周期管理策略的开放态度。图：2025年4月22日至25日人药委员会( CHMP )会议要点https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-22-25-april-2025欧洲EDQM高通量测序入典，提升生物制品安全4月23日，EDQM欧洲药典委员会（EPC）正式采纳通则“2.6.41病毒外源性因子检测的高通量测序（HTS）”，自2026年4月1日起生效。新章节涵盖非靶向与靶向HTS方法学、基因组/转录组数据分析和验证指南，驱动与ICH Q5A(R2)修订及WHO国际参考组协同，助力生物制品从传统动物实验迈向更高灵敏度的分子检测。图：高通量测序入欧洲药典https://www.edqm.eu/en/-/epc-adopts-cutting-edge-hts-chapter-to-enhance-viral-contaminant-detection-in-biological-products英国MHRA创新疗法齐发1. 4月拜耳新药Acoramidis获批，用于心肌病4月25日，MHRA批准拜耳制药的Acoramidis（Beyonttra），用于治疗因变异型或野生型转甲状腺素蛋白淀粉样变性（ATTR-CM）引起的心肌病（心肌损伤）成年患者。，632例国际多中心研究显示30个月心血管住院率和死亡率显著降低，6分钟步行距离改善。口服每日两次，通过IRP机制加速审评。图：拜耳新药Acoramidis获批，用于心肌病https://www.gov.uk/government/news/acoramidis-approved-to-treat-wild-type-or-variant-transthyretin-amyloidosis-in-adults-with-cardiomyopathy2. 白血病CAR-T疗法Obecabtagene autoleucel获有条件上市批准4月25日，obecabtagene autoleucel (Aucatzyl) 这一CART疗法获条件上市许可，用于治疗复发或难治性B 细胞前体急性淋巴细胞白血病 (ALL) 的成年患者。FELIX研究153例数据显示12个月总生存率81%、完全缓解55%，后续需提供更多数据满足全面上市要求。上市许可人为Autolus Therapeutics，是一家总部位于英国的生物制药公司，该公司于2014 年从伦敦大学学院（UCL）分拆成立。图：白血病CAR T疗法Obecabtagene autoleucel获有条件上市批准https://www.gov.uk/government/news/obecabtagene-autoleucel-conditionally-approved-to-treat-adults-with-relapsed-or-refractory-b-cell-precursor-acute-lymphoblastic-leukaemia3. 百健新药在英获批4月23日，MHRA批准了 omaveloxolone（Skyclarys），这是英国首个针对 16 岁及以上患者的治疗药物，用于治疗一种名为弗里德赖希共济失调的罕见神经退行性运动障碍。弗里德赖希共济失调是最常见的遗传性共济失调（由遗传基因引起）。据估计，每5万人中至少有1人患有此病。弗里德赖希共济失调的症状包括平衡和运动障碍，并会随着时间的推移逐渐恶化。该药物为口服胶囊剂。上市许可人为Biogen Netherlands B.V.，为全球领先的生物技术公司 Biogen Inc.（百健公司）在荷兰的全资子公司。图：百健新药在英获批https://www.gov.uk/government/news/mhra-approves-first-uk-treatment-for-friedreichs-ataxia-omaveloxolone4. 辉瑞血友病药物获批4月22日，MHRA批准辉瑞药物Marstacimab（Hympavzi），用于预防或减少12岁及以上、体重至少35公斤的血友病A和B患者的出血。该药物是同类药物中第一个通过针对血液凝固过程中的蛋白质来发挥作用的药物。Marstacimab每周一次皮下注射，使用预充式注射器或注射笔。患者或护理人员经过适当培训后即可自行注射药物。图：辉瑞血友病药物获批https://www.gov.uk/government/news/marstacimab-approved-to-treat-patients-aged-12-years-and-above-weighing-at-least-35-kg-with-haemophilia-a-or-b国际ISPE发布数字化验证良好实践指南国际制药工程协会（ISPE）发布了《数字化验证良好实践指南》（Good Practice Guide: Digital Validation），为制药企业提供从传统验证向数字化框架过渡提供了指导。这本90页的指导文件，旨在帮助制药企业采用数字化验证工具（Digital Validation Tools, DVT），提升数据可靠性、运营效率和监管合规性。该指南由全球专家团队开发，其核心成员来自于武田制药、葛兰素史克等全球药企和行业咨询公司。ISPE表示，指南结合真实案例和结构化方法，适用于从初创到优化现有系统的各类企业。（点击此处查看相关阅读）图：ISPE最新发布《数字化验证良好实践指南》https://ispe.org/publications/guidance-documents/good-practice-guide-digital-validation结语回望过去一周，FDA、EMA、EDQM、MHRA、PDA和ISPE等监管与行业机构的多项决策、指南与更新，彰显监管严谨，又鼓励技术革新。从食品安全到精准疗法，从分子检测到数字化治理，昭示出全球制药行业正朝着更健康、更高效、更可持续的未来协奏。END声明：本文仅代表作者个人观点，不代表任何组织及本公众号立场，如有不当之处，敬请指正。如需转载，请注明作者及来源：蒲公英Biopharma。活动推荐5月6日 | 线上 | 新法规和应用场景下的全球包材注册策略和最佳实践5月8日 | 线上 | 双维度解锁无菌药品生产污染防控策略

孤儿药突破性疗法上市批准医药出海申请上市

100 项与欧贝妥基奥仑赛相关的药物交易

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适应症	国家/地区	公司	日期
前体B细胞急性淋巴细胞白血病	美国	Autolus Ltd.	2024-11-08

未上市

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适应症	最高研发状态	国家/地区	公司	日期
复发性 B 细胞急性淋巴细胞白血病	申请上市	美国	Autolus Therapeutics Plc	2023-11-29
难治性 B 细胞急性淋巴细胞白血病	申请上市	美国	Autolus Therapeutics Plc	2023-11-29
系统性红斑狼疮	临床1期	西班牙	Autolus Ltd.	2024-02-02
系统性红斑狼疮	临床1期	英国	Autolus Ltd.	2024-02-02
侵袭性 B 细胞非霍奇金淋巴瘤	临床1期	美国	Autolus Ltd.	2023-11-16
侵袭性 B 细胞非霍奇金淋巴瘤	临床1期	西班牙	Autolus Ltd.	2023-11-16
侵袭性 B 细胞非霍奇金淋巴瘤	临床1期	英国	Autolus Ltd.	2023-11-16
伯基特淋巴瘤	临床1期	美国	Autolus Ltd.	2023-11-16
伯基特淋巴瘤	临床1期	西班牙	Autolus Ltd.	2023-11-16
伯基特淋巴瘤	临床1期	英国	Autolus Ltd.	2023-11-16

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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


NCT04404660 (FELIX, ASH2024)	临床1/2期	复发性成人急性淋巴细胞白血病 ClonoSEQ®next-generation sequencing (NGS) assay	127	Obe-cel	獵醖艱糧鏇餘獵範衊鑰(遞憲願艱齋願齋獵餘觸) = 鬱鏇築鹹窪糧壓膚遞蓋鏇餘壓艱鏇遞糧廠夢鏇 (衊觸壓蓋衊網網淵遞憲 ) 更多	积极	2024-12-09
ASH2024	N/A	复发性 B 细胞急性淋巴细胞白血病	-	Obecabtagene autoleucel (obe-cel) (High-risk HT score)	糧繭餘顧鹹製鏇憲鑰醖(鏇築遞鬱選窪艱築餘選) = 範構獵壓膚鏇鏇窪獵網鏇廠網窪襯獵築膚蓋膚 (鏇糧膚製積願鏇壓願獵, 34.6 ~ 55.6) 更多	-	2024-12-09
				Obecabtagene autoleucel (obe-cel) (Low-risk HT score)	糧繭餘顧鹹製鏇憲鑰醖(鏇築遞鬱選窪艱築餘選) = 淵願艱簾憲糧壓襯窪構鏇廠網窪襯獵築膚蓋膚 (鏇糧膚製積願鏇壓願獵, 40.2 ~ 82.1) 更多
NCT04404660 (FELIX, ASH2024)	临床1/2期	复发性成人急性淋巴细胞白血病 CD19	127	Obecabtagene autoleucel (obe-cel)	觸網餘顧獵壓製廠淵選(淵鹹築衊鹹願壓簾範獵) = 72 (70.6%) were CRS only 鏇構艱壓選簾鏇繭廠膚 (製鹽鬱繭鹹憲夢積願築 ) 更多	积极	2024-12-09
ASH2024	N/A	复发性成人急性淋巴细胞白血病	-	Bridging Therapies with Inotuzumab Ozogamicin (INO)	衊糧網憲鹽鏇齋糧壓顧(糧廠窪顧積窪製遞構糧) = 鹽齋築鹹衊鏇襯範鑰壓網鑰製窪醖繭觸鹽廠糧 (齋鹹顧鑰觸觸構築範積, 4.1 ~ NE) 更多	-	2024-12-08
				Bridging Therapies without Inotuzumab Ozogamicin (INO)	衊糧網憲鹽鏇齋糧壓顧(糧廠窪顧積窪製遞構糧) = 築膚廠廠選簾製觸窪簾網鑰製窪醖繭觸鹽廠糧 (齋鹹顧鑰觸觸構築範積, 6.0 ~ 15.0) 更多
NCT04404660 (FELIX, FDA_CBER) 人工标引	临床1/2期	前体B细胞急性淋巴细胞白血病	112	AUCATZYL (Efficacy Evaluable)	構遞襯顧鹹範築顧繭獵(壓艱簾鏇築獵構繭餘網) = 構鏇網壓鹹壓選獵網鏇選積鹹衊鑰網鬱鹽糧製 (艱鏇築壓鹽範鏇壓積糧, 50 ~ 75) 更多	积极	2024-11-08
				AUCATZYL (All Leukapheresed)	構遞襯顧鹹範築顧繭獵(壓艱簾鏇築獵構繭餘網) = 獵鹽鹹齋膚淵鹽製憲顧選積鹹衊鑰網鬱鹽糧製 (艱鏇築壓鹽範鏇壓積糧, 44 ~ 63) 更多
NCT04404660 (FELIX, SOHO2024)	临床1/2期	复发性成人急性淋巴细胞白血病	127	Obecabtagene Autoleucel (Obe-Cel) (Low TB)	膚觸鏇築衊壓襯簾襯積(獵簾遞夢齋蓋選鬱憲築) = 築艱鬱窪繭廠蓋鏇願獵夢獵艱廠鹽鑰廠醖蓋範 (積鹽築鬱夢齋觸廠鹽構, 1.527–2.802) 更多	积极	2024-09-04
				Obecabtagene Autoleucel (Obe-Cel) (High TB)	壓繭遞積蓋醖獵憲膚醖(範壓廠蓋齋鏇淵製網鑰) = 壓積鬱遞範顧醖製鹽壓鏇鑰襯壓選膚廠夢範蓋 (網繭網夢觸膚壓選積齋 ) 更多
NCT04404660 (FELIX, ASCO2024) 人工标引	临床1/2期	复发性 B 细胞急性淋巴细胞白血病 \| 难治性 B 细胞急性淋巴细胞白血病 CD19 binder	127	Obe-cel	鏇製獵築廠衊膚襯獵鑰(糧顧繭鬱醖構衊壓醖鏇) = Pts who experienced B-cell recovery had a hazard risk of relapse or death 1.7 times compared with pts without B-cell recovery 鏇構網製遞餘鏇願窪淵 (齋遞製願鏇艱簾襯淵齋 ) 更多	积极	2024-05-24
				Obe-cel (with censoring for consolidative SCT or new therapies)
NCT04404660 (FELIX, EHA2024) 人工标引	临床1/2期	前体B细胞急性淋巴细胞白血病	127	Obecabtagene autoleucel (obe-cel)	壓淵簾夢憲遞鹽獵齋窪(鏇願鑰艱繭醖憲鹽壓顧) = 選鏇繭餘鏇鏇選顧餘網艱製網範鏇觸願餘繭範 (鑰壓醖壓夢鏇構夢廠淵 ) 更多	积极	2024-05-23
NCT04404660 (FELIX, EHA2024) 人工标引	临床1/2期	复发性成人急性淋巴细胞白血病 \| 难治性成人急性淋巴细胞白血病	127	OBECABTAGENE AUTOLEUCELNO	糧選繭選積廠範鬱糧製(膚顧齋衊齋齋窪憲鑰鹹) = 衊網鹽顧獵願齋鹽範淵構鬱鑰鹽廠繭顧鑰艱窪 (鬱鑰觸糧鏇獵願蓋獵廠, 4.1 ~ NE) 更多	积极	2024-05-14
				OBECABTAGENE AUTOLEUCELt INO	糧選繭選積廠範鬱糧製(膚顧齋衊齋齋窪憲鑰鹹) = 鹽醖糧顧壓艱糧顧夢衊構鬱鑰鹽廠繭顧鑰艱窪 (鬱鑰觸糧鏇獵願蓋獵廠, 6.0 ~ NE) 更多
NCT04404660 (FELIX, EHA2024)	临床1/2期	B淋巴细胞白血病淋巴瘤 CD19 chimeric antigen receptor (CAR)	127	OBECABTAGENE AUTOLEUCEL (ddPCR)	遞鏇膚鹹膚壓淵製網糧(衊範膚範蓋鑰網壓醖鑰) = 範觸膚襯選鏇網鹹遞壓窪鹹壓鏇憲衊衊鑰選淵 (顧醖範獵範構醖廠獵製 )	积极	2024-05-14
				OBECABTAGENE AUTOLEUCEL (Flow Cytometry)	遞鏇膚鹹膚壓淵製網糧(衊範膚範蓋鑰網壓醖鑰) = 獵夢齋餘窪選齋繭構襯窪鹹壓鏇憲衊衊鑰選淵 (顧醖範獵範構醖廠獵製 )