Intraperitoneal treatment with fosfomycin, metronidazole and recombinant human granulocyte-macrophage colony-stimulating factor in patients with multi-quadrant peritonitis undergoing abdominal surgery: A randomized placebo-controlled trial (TRIPLE)

开始日期2025-04-01

申办/合作机构

Reponex Pharmaceuticals A/S

NCT05501340

/ Unknown status临床1/2期IIT

Phase I-II Clinical Study of PRaG（PD-1 Inhibitor Intravenous Injectio,Radiotherapy and GM-CSF）Combined With PD-1 Inhibitor Intraperitoneal Perfusion for Advanced Refractory Solid Tumors Combined With Malignant Ascites

The effect and safety of intraperitoneal infusion of PD-1 inhibitor is unclear for patients with peritoneal metastasis of advanced malignant tumors and malignant ascites. It is planned to determine the safety and efficacy of intraperitoneal infusion of PD-1 inhibitor combination with PRaG therapy.

开始日期2022-09-01

申办/合作机构

苏州大学附属第二医院

NCT04979832

/ Recruiting临床1/2期IIT

A Combined Treatment With GM-CSF, Fosfomycin and Metronidazole for Pouchitis in Ulcerative Colitis Patients After Restorative Ileal Pouch Anal Anastomosis Surgery

This study will examine whether the application of GM-CSF, fosfomycin and metronidazole locally in the pouch is safe and effective in the treatment of pouchitis for patients with ulcerative colitis, and whether treatment changes the microbiome of the pouch.

开始日期2021-09-06

申办/合作机构

Roskilde Sygehus

100 项与 Molgramostim 相关的临床结果

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100 项与 Molgramostim 相关的转化医学

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100 项与 Molgramostim 相关的专利（医药）

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项与 Molgramostim 相关的文献（医药）

2025-10-01·DRUGS

Pharmacotherapy for Autoimmune Pulmonary Alveolar Proteinosis

Review

作者: Benoît Painvin ; Stéphane Jouneau ; Mallorie Kerjouan ; Pierre Chauvin ; Mathieu Lederlin

Pulmonary alveolar proteinosis is suspected when a "crazy paving" pattern is observed on a chest CT scan. This diagnosis is confirmed by the presence of eosinophilic extracellular material that shows positive staining with Periodic Acid Schiff on bronchoalveolar lavage samples. The autoimmune form of pulmonary alveolar proteinosis is confirmed by detecting anti-granulocyte-macrophage colony-stimulating factor antibodies in the patient's serum. The historical first-line treatment for autoimmune pulmonary alveolar proteinosis is whole lung lavage, which should only be performed in expert centers. It remains the preferred treatment for patients experiencing respiratory failure, especially at the time of diagnosis. Inhaled granulocyte-macrophage colony-stimulating factor supplementation with molgramostim or sargramostim is now considered a first-line treatment in the international guidelines for autoimmune pulmonary alveolar proteinosis, following the positive results of recent randomized placebo-controlled studies. Rituximab and plasmapheresis can be prescribed as third- and fourth-line treatments, respectively. Lung transplantation may be considered for eligible patients experiencing terminal respiratory failure. A deeper understanding of the pathogenesis of autoimmune pulmonary alveolar proteinosis has opened up new therapeutic avenues, such as the use of PPARγ agonists or statins.

2025-09-22·Immunotherapy

Recombinant GM-CSF drug evaluation review

Review

作者: McCarthy, Jack ; McCarthy, Cormac ; Boyle, Niamh

Autoimmune Pulmonary Alveolar Proteinosis (aPAP) is a rare disease characterized by myeloid cell dysfunction, abnormal pulmonary surfactant accumulation, and innate immune deficiency. Pathogenesis is driven by GM-CSF neutralizing autoantibodies (GMAbs) present in high titer in serum and bronchoalveolar lavage fluid (BALF) of patients. GM-CSF is a cytokine and a hematopoietic growth factor produced by a variety of cells. In the lungs, GM-CSF regulates surfactant homeostasis and lung host defense through innate immune function. Whole lung lavage (WLL) is the current first line therapy for aPAP. It is a procedure in which excessive surfactant is mechanically removed from the alveoli. Pathogenesis-driven treatment with GM-CSF augmentation has been investigated over the last two decades with recombinant GM-CSF (rGM-CSF). Molgramostim and sargramostim are rGM-CSF which can be self-administered at home by patients with aPAP either subcutaneously or using a handheld nebulizer. In Phase II and III studies of adults with aPAP, daily administration of inhaled molgramostim resulted in greater improvements in pulmonary gas transfer and functional health status than placebo, with similar rates of adverse events. In all clinical studies to date, rGM-CSF has shown a favorable safety profile with no dose limiting toxicity. This supports the application of inhaled rGM-CSF for the treatment of aPAP and the possibility of WLL as a rescue therapy.

2025-09-01·BMJ Open Respiratory Research

Pharmacokinetics and pharmacodynamics of inhaled molgramostim in healthy people

Article

作者: Trapnell, Bruce C ; Robinson, Brian R ; Carey, Brenna C

Background:

Inhaled molgramostim, a form of recombinant human granulocyte–macrophage colony stimulating factor (GM-CSF), is a promising investigational pharmacotherapy for autoimmune pulmonary alveolar proteinosis (aPAP); however, its pharmacology in healthy subjects has not been reported.

Methods:

This randomised, double-blind, placebo-controlled, single-centre, phase 1 clinical trial assessed the safety, tolerability, pharmacokinetics and pharmacodynamics of inhaled molgramostim in healthy adults in single ascending dose (SAD) and multiple ascending dose (MAD) studies: one 150, 300 or 600 µg administration or six consecutive daily 300 or 600 µg administrations with evaluations over 28 or 34 days, respectively. The primary endpoint was safety, which was evaluated based on the number and severity of treatment-emergent AEs following single and multiple inhaled doses of molgramostim.

Results:

42 subjects were enrolled including 18 in the SAD study and 24 in the MAD study; all completed the study. Inhaled molgramostim in healthy people was well tolerated and no dose-limiting safety concerns or anti-drug antibody formation were observed in either study. GM-CSF was measurable in serum 30 min after administration of inhaled molgramostim, peaked at 2 hours for all three doses and had an elimination half-life of 1.7±0.0 to 5.9±0.9 hours in the SAD and MAD studies. Systemic GM-CSF exposure was non-linear in both the SAD and MAD studies. Inhaled molgramostim caused a rapid increase in white blood cells (WBC) counts and leucocyte subsets that normalised by 8 hours (SAD) or 15–21 days (MAD). Fractional exhaled nitric oxide remained within the normal range at all doses but was numerically, but not significantly, increased at the 600 μg dose.

Conclusions:

In healthy people, inhaled molgramostim was well-tolerated and resulted in systemic exposure at picogram levels, which had the expected PD effects on blood leucocyte levels that mostly remained within normal ranges.

Trial registration number:

NCT02468908 ; EudraCT No. 2013-001687-32

项与 Molgramostim 相关的新闻（医药）

2026-08-06

·科途医学K2Oncology

这个八月，全球医药正在密集“交卷”

2026年8月5日，美国FDA对Moderna mRNA-1010进行最终裁决——如果获批，它将成为全球首个mRNA季节性流感疫苗，也是mRNA技术第一次以“常规疫苗”而非“应急疫苗”的身份登陆主流市场（截止发稿，FDA还未公布审批结果）。而mRNA流感疫苗，只是这个八月密集审批风暴的开场。基因治疗、同种异体细胞疗法、核药、全新机制的骨髓瘤口服药、阿尔茨海默病皮下制剂……近十项关键PDUFA审批截止日挤在接下来的三周里。全球医药正在集体“交卷”。一、头条时刻：mRNA走出新冠，第一次“常态化” mRNA-1010是一条“三价”季节性流感mRNA疫苗，靶向H1N1、H3N2及B/Victoria流感毒株。它的审批之路一波三折： 2月12日，FDA罕见地发出“拒绝受理函”（RTF），理由是Moderna在III期关键研究中选用标准剂量疫苗而非高剂量/佐剂疫苗作为对照，不符合65岁以上人群的审评预期； 2月18日，Moderna与FDA召开会议，以“分年龄层”策略反转局面：50–64岁寻求传统批准，65岁及以上寻求加速批准（附带覆盖最多80万人的上市后确证研究）； 6月18日，FDA疫苗及相关生物制品咨询委员会（VRBPAC）以9:0、9:0两轮全票支持其获益-风险平衡； 8月5日，PDUFA裁决日。支撑这场反转的，是扎实的临床数据。关键III期研究（P304）入组40,817名成人、覆盖11个国家301个中心，结果显示mRNA-1010相比标准剂量流感疫苗，将流感样疾病风险相对降低26.6%（95%CI 16.7–35.4），相对保护效力达47.9%；在65岁以上人群的独立免疫原性研究中，对标Fluzone High-Dose（CDC为老年人首选的疫苗），在全部四个匹配毒株上均达到非劣且优效——Day 29抗体滴度与血清转化率全面领先。流感疫苗自1940年代起依赖鸡胚工艺，生产周期长达6个月以上，毒株匹配度常年波动，保护效力常在40%–60%之间徘徊。mRNA路线把“选株到量产”压缩到数周，让疫苗可以贴着流行季选株——这是对整个流感疫苗产业生产范式的松动。Moderna为此投入了四年时间和约7.5亿美元。若获批，mRNA-1010将赶上2026–27流感季，并率先在50岁及以上人群中铺开。二、八月“审批日历”：六场硬仗，六种技术形态 mRNA流感疫苗只是开场。翻开8月的PDUFA日历，几乎每一项都对应一种前沿技术形态——这是一个罕见的“多技术平台同台交卷”的月份。 8月13日 · 诊断先行：MK-6240（tau PET示踪剂）用于检测阿尔茨海默病相关认知障碍患者脑内tau神经原纤维缠结病理变化。诊断工具的率先审批，往往预示着治疗时代的下一步。 8月17日 · 骨髓瘤新机制：iberdomide百时美施贵宝开发的口服CELMoD（cereblon E3连接酶调节剂），联合达雷妥尤单抗+地塞米松治疗复发/难治性多发性骨髓瘤。EXCALIBER-RRMM III期中期分析显示MRD阴性率显著优于对照（DVd方案）。若获批，这将是骨髓瘤领域靶向泛素-蛋白酶体通路中“降解”全新作用机制的首款药物。 8月22日 · 细胞疗法与吸入生物药同日登场 deramiocel（CAP-1002）：由Capricor与日本新药株式会社开发的同种异体心源性细胞疗法，治疗杜氏肌营养不良（DMD）心肌病。HOPE-3 III期研究纳入106名男孩，上肢功能改善显著。这是“现货型”细胞疗法首次冲击DMD这一适应症； MOLBREEVI（molgramostim）：Savara开发的吸入型重组人GM-CSF，用于治疗自身免疫性肺泡蛋白沉积症（aPAP）——一种肺泡表面活性物质堆积的罕见肺病，目前无任何获批药物。该产品集First-in-class、罕见病、吸入递送三重属性于一身。 8月23日 · 基因治疗：DTX401Ultragenyx开发的AAV基因疗法，专门用于治疗糖原累积症Ia型（GSD Ia）——患者因葡萄糖-6-磷酸酶缺乏而无法正常释放肝糖原，需夜间持续进食玉米淀粉维持血糖。III期GlucoGene研究显示患者玉米淀粉摄入量显著减少了41%。若获批，这将是代谢性罕见病领域又一款从“终身管理”走向“单次修复”的AAV疗法。 8月24日 · 阿尔茨海默病：lecanemab皮下注射（LEQEMBI IQLIK）卫材与渤健将已获批的静脉注射lecanemab升级为皮下制剂。对于需要长期维持治疗的AD患者，依从性与给药便利性的提升，本身就是重塑治疗格局的关键环节。 8月25日 · 双抗补位：zanidatamab-hrii（Ziihera）Zymeworks研发的HER2双特异性抗体，正冲刺一线HER2阳性胃食管腺癌。该适应症目前缺乏获批的一线靶向选择——竞争格局真空，正是审批的高概率区。 8月27日 · HIV单片：bictegravir + lenacapavir吉利德创新性将整合酶抑制剂与衣壳抑制剂合成单片复方，ARTISTRY-1/2研究显示与现有方案维持病毒抑制等效，同时带来血脂改善与更小的药片体积——“最小的HIV单片方案”。 8月28日 · 核药再下一城：177Lu-edotreotideITM的放射性配体疗法，治疗胃肠胰神经内分泌肿瘤（GEP-NET）。COMPETE III期研究已发表于《柳叶刀》：相比依维莫司，中位PFS 23.9个月 vs 14.1个月（HR 0.67，P=0.022）。这是继177Lu-PSMA-617改写前列腺癌之后，放射性配体疗法向实体瘤版图扩张的又一重大信号。此外，Regeneron的garetosmab（Activin A单抗，治疗进行性骨化性纤维发育不良FOP这一超罕见病）也将在本月迎来优先审评裁决。将这份日历连起来看，能看到清晰的产业主线：mRNA疫苗、AAV基因治疗、同种异体细胞疗法、核药、CELMoD新机制小分子、吸入与皮下递送——几乎每一种技术形态，都在本月迎来了自己的“里程碑窗口”。三、中国创新药的最新注脚全球审批风暴之外，中国创新药也在近期贡献了高含金量的注脚。 7月24日，莱芒生物代谢增强型CD19 CAR-T（META 10-19）治疗复发/难治性弥漫大B细胞淋巴瘤的I期临床研究发表于《JAMA Oncology》——其核心亮点是“剂量低至传统CAR-T的千分之一”，通过代谢免疫工程让CAR-T在极低剂量下依然高效；7月28日，该产品在高危DLBCL中实现100%客观缓解率的研究摘要被《Journal of Clinical Oncology》收录。同时，META 10-19已获得FDA IND批准，并将适应症拓展至系统性红斑狼疮等自免领域。一个细节值得注意：“剂量千分之一”与“免清淋”——这是中国团队在细胞治疗“可及性”这一全球性难题上给出的原创解法。当全球CAR-T还在与百万美元级成本搏斗时，把有效剂量降三个数量级，本身就是一种产业级创新。四、观察：当“批准”变得密集，行业在交付什么？密集的审批窗口背后，藏着三个趋势。第一，技术平台进入“各就各位”阶段。mRNA证明了疫苗平台的通用性；AAV基因治疗从眼科、血液病走向代谢病；同种异体细胞疗法冲击神经肌肉病；核药从前列腺癌向神经内分泌肿瘤扩张。五年前还是“路线之争”的技术路线，如今各自在自己的适应症上拿到了“毕业证”。第二，递送与制剂成为“第二战场”。同样的分子，从静脉到皮下（lecanemab）、从每日口服到单片（HIV复方）、从注射到吸入（molgramostim）、从住院到居家——当疗效接近天花板，依从性、便利性与可及性成为差异化主战场。这一点对慢病与罕见病尤其重要：让患者“愿意坚持治疗”，本身就是疗效的一部分。第三，从“管理疾病”到“修复功能”。DTX401让GSD Ia患者有望摆脱夜间喂食淀粉；deramiocel瞄准DMD的心肌损伤；基因治疗与细胞治疗的核心叙事，正从“控制症状”转向“对因修复”。当审批名单里“一次性治疗”的比例越来越高，医药的底层承诺正在发生变化。对中国药企而言，这份8月日历是一面镜子：全球监管对创新形态的接纳度在提高，加速批准、优先审评、罕见病通道都在打开——窗口是真实的，但窗口只对“有真正差异化数据”的人敞开。能被审批者看见的，从来不是“赛道热度”，而是“未满足需求上的硬证据”。参考文献及信息来源 https://trials.modernatx.com/study/?id=mRNA-1010-P304 https://www.empr.com/news/fda-drug-approval-decisions-expected-in-august-2026/#xd_co_f=Y2NjNzUxMTQtN2VmNi00ZDdkLWI5MzctNDNiN2IwNjc2Nzgy https://www.cidrap.umn.edu/influenza-vaccines/moderna-s-mrna-flu-vaccine-gets-thumbs-federal-vaccine-panel https://www.fda.gov/media/193131/download Infectious Disease Advisor：FDA Drug Approval Decisions Expected in August 2026（2026-07-20） BiopharmaWatch：FDA Calendar August 2026: Key PDUFA Dates and Analysis（2026-08-03） Prime Therapeutics：FDA Decisions Expected: August 2026（2026-07-21） Hu Y, et al. Ultralow-Dose Interleukin 10-Expressing Chimeric Antigen Receptor T Cells in Relapsed/Refractory Diffuse Large B-Cell Lymphoma: A Nonrandomized Clinical Trial. JAMA Oncol. 2026 Jul 23: e262490. Qianwen Xu. et al. Phase 1 study of META 10-19, an IL-10-expressing, anti-CD19 CAR T cell product in patients with high-risk DLBCL. J Clin Oncol. 44, 2552（2026），44: 16. Walter T, et al. [177Lu]Lu-edotreotide versus everolimus for gastroenteropancreatic neuroendocrine tumours (COMPETE): a phase 3, multicentre, randomised, open-label, superiority trial. Lancet. 2026 Jul 18;408(10551):234-247. ---------------------- 更多推荐： 1，秋季开学促销； 2，科途类器官产品全国代理招募。

2026-08-05

·PharmaFun

吸入制剂总台｜双重专利壁垒落地！Savara雾化吸入GM-CSF MOLBREEVI夯实海外市场独家权，罕见肺病First-in-class商业化再进一步

苏州9月11以药企为核心主导吸入制剂产学研论坛｜恒瑞、润生、阿斯利康原研出身专家领衔，聚焦全球化：共探前沿吸入技术、注射剂转吸入化 2026年7月30日，临床阶段罕见呼吸药企Savara（Nasdaq:SVRA）重磅公告：欧洲专利局、澳大利亚专利局双双授予旗下核心候选药MOLBREEVI（莫拉司亭吸入液）液体制剂基础专利，两大市场保护期统一至2041年3月。叠加2025年底获批的药械组合欧洲联合专利（保护至2043年），产品形成「单方制剂+专属给药器械」双层知识产权护城河。一、事件核心完整复盘：两份新专利关键信息拆解 1️⃣本次新增2项液体制剂专利（单方制剂专利） 1. 欧洲专利：专利号No.4121000，保护标的为MOLBREEVI吸入液液体配方本身，覆盖全部欧盟成员国，专利有效期至2041年3月； 2. 澳大利亚专利：专利号No.62/990,810，同样保护莫拉司亭吸入液雾化处方，澳洲市场保护期限与欧洲同步至2041年3月。 Savara首席法务官Kate McCabe官方解读：本次专利补齐欧洲、澳洲核心海外市场的制剂配方IP，在暂无获批疗法的aPAP赛道构筑第一层独家防护；公司同步推进美国等同专利申报，含药械组合专利，目标实现全球主流商业市场全覆盖知识产权保护。 2️⃣存量重磅专利：2025年12月药械组合联合专利（复方/组合专利） Savara与德国器械企业PARI Pharma共享欧洲联合专利，保护MOLBREEVI药物+专属eFlow®振动筛网雾化器整套给药系统，保护周期更长，至2043年3月，比单方制剂专利多2年壁垒期。 3️⃣单方制剂专利VS药械组合专利：核心差异、商业价值对比很多读者难以区分两类专利的保护边界，下表清晰拆解二者定位、保护范围、行业壁垒差异：行业专业解读：罕见病药物研发投入极高、患者基数小，单一专利极易被竞品绕开；Savara采用「制剂底层专利+药械组合上层专利」双轨布局，形成闭环防护。竞品即便绕开药械专利自研雾化器，也无法仿制核心液体配方；若强行仿制药液，直接触发单方制剂专利侵权，双重阻断仿制药竞争路径。二、核心药物MOLBREEVI与靶点疾病：专业科普，看懂临床价值 1. 靶点疾病：自身免疫性肺泡蛋白沉积症（Autoimmune PAP，aPAP）（1）发病机制（原文权威科普）肺泡内存在肺泡巨噬细胞，依靠粒细胞-巨噬细胞集落刺激因子（GM-CSF）激活，负责清除肺泡多余表面活性物质（肺泡润滑脂蛋白）；aPAP患者体内会产生中和性抗GM-CSF自身抗体，完全阻断GM-CSF信号，巨噬细胞彻底失活，表面活性物质持续堆积在肺泡，阻碍氧气交换。（2）临床症状与现有治疗短板 • 典型表现：持续性气短、慢性咳嗽、极度疲劳，继发肺部感染时出现胸痛、咯血；长期进展可诱发肺纤维化，最终需要肺移植，存在致命风险； • 传统唯一手段：全肺灌洗手术，属于创伤性治疗，需定期重复灌洗，无法根治，国内、欧美均无获批靶向药物，存在巨大未满足临床需求，这也是该药物拿到全球多重突破性认定的核心依据。（3）疾病流行病学定位 aPAP占肺泡蛋白沉积症90%，属于典型罕见病，欧美患者均不足20万人，符合各国孤儿药认定标准，因此MOLBREEVI全线拿下全球罕见病激励政策背书。 2. MOLBREEVI（莫拉司亭吸入溶液）药物机制与剂型优势 1. 药物本质：重组人GM-CSF无菌吸入液，商品名MOLBREEVI，活性成分为molgramostim（莫拉司亭），通过雾化直接递送至肺泡，绕开血液中中和抗体，原位激活巨噬细胞，从根源清除堆积的表面活性物质，属于病因治疗而非对症缓解； 2. 剂型创新价值：传统GM-CSF仅注射剂型，全身给药肺部药物浓度极低；吸入制剂直达病灶、全身副作用更小，每日一次雾化，单次仅5分钟，患者依从性大幅优于手术与注射方案； 3. 给药配套：PARI eFlow®雾化器核心技术采用振动筛网雾化技术，产出1-5μm均匀可吸入颗粒，肺部药物递送率超60%，远高于传统压缩雾化器30%利用率；设备可精准控剂量，全程维持GM-CSF生物活性，避免雾化过程蛋白降解，是这款药物临床疗效达标的核心配套硬件。三、全球多重监管特殊认定梳理：全部权威资质科普 MOLBREEVI是全球少有的集齐美、欧、英三地全部加速审评通道的罕见病吸入生物药，每一项认定均对应明确政策红利，逐一拆解： 1️⃣美国FDA授予资质 1. 突破性疗法认定（BTD）：临床初步数据证明相比现有治疗（全肺灌洗）有显著临床终点改善，享有FDA高层专人指导、滚动提交资料、优先审评全流程加速福利； 2. 快速通道认定（Fast Track）：针对重症、无有效疗法疾病，允许分批次提交上市申报资料，随时与审评团队沟通优化方案； 3. 孤儿药认定（Orphan Drug）：针对美国患病人数＜20万罕见病，获批后享有7年美国市场独占权、研发税收减免、申报费用减免等政策激励； 4. 最新审评进度（2026年4月）：FDA延长MOLBREEVI生物制剂许可申请（BLA）审评周期，公司仍冲刺全球首款aPAP获批药物；同步英国MHRA当月受理其上市申请。 2️⃣欧洲EMA资质孤儿药认定，获批后欧盟10年市场独占，简化临床试验数据要求，降低上市门槛。 3️⃣英国MHRA（英国药监）双创新资质 1. 创新护照（Innovation Passport, IP）：英国针对first-in-class新药全流程扶持通道； 2. 潜力创新药物认定（PIM）：等同于英国版突破性疗法，大幅缩短上市审评时长，优先对接临床专家资源。行业总结：一套候选药集齐六大加速审评资质，足以证明全球药监机构高度认可MOLBREEVI的临床刚需与创新程度，也是资本市场看好其商业化潜力的核心逻辑。四、相关核心企业与机构深度介绍 1️⃣Savara Inc.（纳斯达克：SVRA） • 企业定位：专注罕见呼吸道疾病的临床阶段生物制药企业，管线核心资产仅MOLBREEVI一款三期阶段核心产品，管理团队深耕罕见肺病、呼吸制剂商业化领域； • 发展战略：全产业链布局MOLBREEVI，自主掌握液体制剂配方知识产权，器械端与德国PARI独家合作，同步推进欧美澳全球专利布局、多国上市申报； • 风险提示：公司为临床阶段企业，药物尚未在任何国家获批；存在研发失败、审评延迟、现金流不足、融资不及预期等经营风险，所有专利、审评进展不代表必然商业化成功。 2️⃣PARI Pharma GmbH（德国）全球雾化吸入给药器械龙头企业，eFlow®振动膜雾化技术独家持有，长期与跨国药企合作开发生物制剂专用雾化系统；为MOLBREEVI定制专属雾化设备，共享药械组合专利，是药物实现临床疗效不可分割的合作方。百年气溶胶之路：德国PARI全球气动雾化吸入治疗龙头完整发展历史纪实｜吸入制剂总台 3️⃣三大专利/药监官方机构 1. EPO欧洲专利局：欧盟统一发明专利授权机构，专利覆盖全部欧盟成员国，统一保护期限； 2. 澳大利亚专利局：独立知识产权审批机构，海外医药企业拓展亚太市场核心专利布局阵地； 3. FDA/EMA/MHRA：美、欧、英主流药品监管机构，分别负责北美、欧盟、英国市场上市审批与加速政策落地。五、事件深度分析：三层行业与商业逻辑解读 1. 短期层面：欧洲、澳洲知识产权空白补齐，海外商业化底盘稳固此前Savara仅持有欧洲药械组合专利，缺少液体配方底层保护；本次单方制剂专利落地后，即便未来竞品开发其他雾化设备，也无法仿制核心吸入药液，彻底守住欧盟、澳洲两大无竞品市场，为药物上市后独家销售扫清仿制药风险。 2. 中期层面：全球专利矩阵成型，美国市场布局进入关键期公司明确同步推进美国等同液体制剂、药械组合专利申报，若美国专利顺利授权，将实现美、欧、澳三大核心市场完整双层专利壁垒；叠加各地孤儿药市场独占期，药物上市后20年内难以出现直接竞品，罕见病稀缺价值持续放大。 3. 长期行业层面：罕见病吸入生物药「药械一体化」成标准研发范式过往罕见药仅重视药物分子专利，忽略制剂剂型与给药器械；Savara本次双专利布局验证行业新趋势：针对吸入类生物制剂，制剂配方专利（单方底层）+药械组合专利（配套上层）双重IP防护，是锁定长期商业收益、对冲研发成本的最优路径，为同类呼吸罕见病新药提供参考模板。咨询联系：13380632632（司徒老师）六、全文核心总结 1. 事件事实：2026年7月30日Savara获欧、澳两份MOLBREEVI液体单方制剂专利，保护至2041年3月；叠加2025年欧洲药械组合联合专利（至2043年），形成双层知识产权护城河； 2. 产品核心价值：MOLBREEVI为全球潜在First-in-class吸入GM-CSF，直击无获批药物的罕见病aPAP，集齐美欧英全部加速审评通道，临床需求刚性极强； 3. 专利差异化逻辑：单方制剂专利锁死药物配方底层壁垒，药械组合专利绑定专属雾化器械，两者保护范围、周期、侵权限制互补，双重阻断仿制药竞争； 4. 商业化前景：专利覆盖欧美澳主流市场，各国孤儿药政策加持，若BLA顺利获批，将成为自身免疫性肺泡蛋白沉积症全球首款靶向治疗药物，但仍存在审评、资金、临床开发不确定性； 5. 行业启示：吸入型罕见生物药单一专利防护力度不足，「制剂+药械组合」双轨专利布局将成为未来药企标准知识产权策略。吸入制剂总台｜中国吸入制剂领域独家及头部平台，全国首创商务生态链BD模式，推动行业和助力企业走向创新出海高壁垒Inhalation Global 合规声明本文全部数据、专利信息、企业观点、临床机制均来源于Savara官方公告、SEC披露文件、EMA/FDA公开审评政策、呼吸疾病权威医学文献，无推演、编造未证实信息，内容保持客观中立，同步完整披露企业前瞻性风险提示，适合医药行业从业者、创新药投资者、呼吸临床医师阅读参考。

2026-07-30

·BioSpace

Savara Announces European and Australian Patent Offices Have Granted Patents for the Liquid Formulation of MOLBREEVI*, Providing Protection in Europe and Australia Until March 2041

-- In December 2025, Savara and PARI Were Granted a Joint European Patent Covering the Investigational Drug-Device Combination of MOLBREEVI, Delivered Via PARI’s Proprietary eFlow ® Nebulizer System, Through March 2043 -- LANGHORNE, Pa.--(BUSINESS WIRE)-- Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, today announced that the European Patent Office (EPO) has granted a patent covering the liquid formulation of MOLBREEVI, an orally inhaled recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF). European patent No.4121000 provides protection for MOLBREEVI through March 2041. Separately, the Australian Patent Office has also granted a patent, No. 62/990,810, covering the liquid formulation of MOLBREEVI, and securing protection in Australia through the same date. “These patents further strengthen the Company’s intellectual property portfolio for MOLBREEVI, providing an additional layer of defense in markets with no approved alternatives,” said Kate McCabe, Chief Legal Officer, Savara. “Together, these patents reflect the strength of our liquid formulation IP across key ex-US markets. In parallel, we are advancing equivalent patent filings in the U.S., including a patent for the drug-device combination, with the aim of securing comprehensive protection across major commercial territories.” MOLBREEVI, delivered via the proprietary eFlow ® Nebulizer System, has been granted Fast Track and Breakthrough Therapy Designations by the U.S. Food and Drug Administration (FDA), Orphan Drug Designation by the FDA and by the European Medicines Agency (EMA), and Innovation Passport (IP) and Promising Innovative Medicine (PIM) designations by the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) for the treatment of autoimmune PAP. ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); About Autoimmune Pulmonary Alveolar Proteinosis (Autoimmune PAP) Autoimmune PAP is a rare lung disease characterized by the abnormal build-up of surfactant in the alveoli of the lungs. Surfactant consists of proteins and lipids and is an important physiological substance that lines the alveoli to prevent them from collapsing. In a healthy lung, excess surfactant is cleared and digested by immune cells called alveolar macrophages. Alveolar macrophages need to be stimulated by granulocyte-macrophage colony-stimulating factor (GM-CSF) to function properly in clearing surfactant, but in autoimmune PAP, GM-CSF is neutralized by antibodies against GM-CSF, rendering macrophages unable to adequately clear surfactant. As a result, an excess of surfactant accumulates in the alveoli, causing impaired gas exchange, resulting in clinical symptoms of shortness of breath, often with cough and frequent fatigue. Patients may also experience episodes of fever, chest pain, or coughing up blood, especially if secondary lung infection develops. In the long term, the disease can lead to serious complications, including lung fibrosis and the need for a lung transplant. About Savara Savara is a clinical stage biopharmaceutical company focused on rare respiratory diseases. Our lead program, MOLBREEVI*, is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis (autoimmune PAP). MOLBREEVI is delivered via the proprietary investigational eFlow ® Nebulizer System (PARI Pharma GmbH) specifically developed for inhalation of MOLBREEVI. Our management team has significant experience in rare respiratory diseases and pulmonary medicine, identifying unmet needs, and effectively advancing product candidates to approval and commercialization. More information can be found at and LinkedIn. *MOLBREEVI is the proposed trade name for molgramostim inhalation solution. It is not approved in any indication. MOLBREEVI is a trademark of Savara Inc. Forward-Looking Statements Savara cautions you that statements in this press release that are not a description of historical fact are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances such as “expect,” “intend,” “plan,” “anticipate,” “believe,” and “will,” among others. Such statements include, but are not limited to, statements related to the impact of the patents and the duration of protection and that we are advancing equivalent patent filings in the U.S. Savara may not actually achieve any of the matters referred to in such forward-looking statements, and you should not place undue reliance on these forward-looking statements. These forward-looking statements are based upon Savara’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, the risks associated with our ability to successfully develop, obtain regulatory approval for, and commercialize MOLBREEVI for autoimmune PAP; the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations; the availability of sufficient resources for Savara’s operations and to conduct or continue planned clinical development programs; and the timing and ability of Savara to raise additional capital as needed to fund continued operations. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. For a detailed description of our risks and uncertainties, you are encouraged to review our documents filed with the SEC including our recent filings on Form 8-K, Form 10-K and Form 10-Q. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Savara undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as may be required by law. Contacts Media and Investor Relations Contact Savara Inc. Temre Johnson, Executive Director, Corporate Affairs ir@savarapharma.com

100 项与 Molgramostim 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D05066	Molgramostim	L03AA03	DB12525

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
粒细胞减少症	瑞典	Schering-Plough Europe SA	1991-11-08

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
获得性肺泡蛋白沉积症	申请上市	美国	Savara, Inc.	2025-12-01

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临床结果

适应症

分期

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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


ATS2025	临床3期	获得性肺泡蛋白沉积症 autoantibodies to granulocyte-macrophage colony-stimulating factor (GM-CSF)	164	Molgramostim 300 µg	淵範選製遞憲壓願範簾(積築範繭鹽憲糧選獵鑰) = 選網積選顧艱繭顧範顧選壓觸膚觸鹹觸鑰糧壓 (糧範獵艱廠淵願繭遞鏇, 2.8)	积极	2025-05-16
				Placebo	淵範選製遞憲壓願範簾(積築範繭鹽憲糧選獵鑰) = 願願構觸鬱衊衊醖鹽壓選壓觸膚觸鹹觸鑰糧壓 (糧範獵艱廠淵願繭遞鏇, 2.3)
IMPALA-2 (ERS2024)	临床3期	获得性肺泡蛋白沉积症	164	Inhaled molgramostim 300 μg	糧簾齋醖繭淵艱繭艱窪(糧淵齋鏇蓋鑰窪觸顧夢): P-Value = 0.0007 更多	积极	2024-09-07
				Placebo
ERS2021	N/A	非结核分枝杆菌感染	32	Inhaled GM-CSF + ongoing GBT	鏇願衊壓蓋窪蓋觸糧廠(壓醖簾鬱鬱鹹願夢餘觸) = 選齋簾鏇淵膚壓憲願觸艱憲鹹襯夢願餘衊築網 (鏇鏇簾窪憲蓋簾範構襯 ) 更多	不佳	2021-09-05
				Inhaled GM-CSF monotherapy	鏇願衊壓蓋窪蓋觸糧廠(壓醖簾鬱鬱鹹願夢餘觸) = 鏇窪衊鑰鏇窪糧積築築艱憲鹹襯夢願餘衊築網 (鏇鏇簾窪憲蓋簾範構襯 ) 更多