Background:
Multiple myeloma (MM) is a type of blood cancer that affects a person s immunity. MM returns after treatment (relapse) in almost all people; MM may also not respond to initial treatment (refractory). Many people with relapsed refractory MM (RRMM) also have changes in their KRAS and NRAS genes. Researchers want to try a new drug treatment that targets cancer with these changed genes.
Objective:
To test 2 drugs (mirdametinib and sirolimus) in people with RRMM.
Eligibility:
People aged 18 and older with RRMM who have changes in their KRAS or NRAS genes.
Design:
Participants will be screened. They will have blood tests and imaging scans. They will have an eye exam and a test of their heart function. They will need to provide proof of their disease status and of their KRAS or NRAS status. If neither is available, the tests will be repeated.
Participants will have a bone marrow biopsy: A needle will be inserted into a hipbone to draw out some soft tissue.
This study will be done in two parts. In the first part of this study, we will find a safe dose of mirdametinib combined with sirolimus. In the second part, we will learn more about how mirdametinib combined with sirolimus may work against RRMM.
Mirdametinib (capsules) and sirolimus (tablets) are taken by mouth. Participants will take both drugs at home on a 4-week cycle. They will take mirdametinib twice a day for the first 3 weeks of each cycle. They will take sirolimus once a day, every day, during each cycle.
Participants will have study visits once a week during the first cycle, and then on the first day of subsequent cycles. Blood, heart, imaging scans, and other tests will be repeated.
Treatment with the study drugs will go on for 1 year. Then participants will have follow-up visits every 3 months for 4 more years.

开始日期2025-06-03

申办/合作机构

National Cancer Institute

NCT06997276

/ Recruiting临床1期

A Phase 1 Open-Label Study to Assess the Pharmacokinetics of Mirdametinib and Its Metabolite PD-0315209 in Participants With Impaired Hepatic Function and Participants With Normal Hepatic Function

The purposes of this study are to determine:
* The pharmacokinetics (the amount of study drug in your blood and how long it takes the body to get rid of it) of the study drug and its metabolites (substances produced as the body breaks down the study drug) in participants with moderate or severe liver function impairment compared to participants with normal liver function (also known as a healthy volunteer). Pharmacokinetics (or PK) is the study of how your body absorbs, breaks down, and removes a study drug.
* How well the study drug is tolerated and any side effects that may occur in participants with moderate or severe liver function impairment compared to participants with normal liver function.
This study is for research purposes only and is not intended to treat any medical condition.

开始日期2025-05-07

申办/合作机构

Springworks Therapeutics, Inc.

NCT06666348

/ Not yet recruiting临床1/2期

A Phase 1/2 Study of Mirdametinib and Vinblastine for Newly Diagnosed or Previously Untreated Patients With Pediatric Low-grade Glioma and Activation of the MAPK Pathway

This is a 3-part open-label study (feasibility phase, treatment phase and follow-up phase) of orally administered mirdametinib in combination with intravenous vinblastine chemotherapy in patients with PLGG with activation of MAPK pathway.
Feasibility Phase:
The maximum tolerated/recommended phase 2 dose (MTD/RP2D) of the mirdametinib plus vinblastine combination will be assessed using a modified Rolling-6 design.
Treatment Phase:
Patients will receive mirdametinib twice daily (continuously) at a fixed dose (2mg/m2 po BID up to 4 mg BID) for a total of 13 cycles (28 days cycle). Weekly intravenous vinblastine at MTD will be given for a total of 17 cycles. If adverse events occur, two dose reductions are allowed.
Follow-up Phase:
Following the end of treatment, patients will be scheduled for a follow-up visit every 6 months for 36 months to evaluate PFS, TTP and OS.

开始日期2025-04-01

申办/合作机构

C17 Council

100 项与 Mirdametinib 相关的临床结果

登录后查看更多信息

100 项与 Mirdametinib 相关的转化医学

登录后查看更多信息

100 项与 Mirdametinib 相关的专利（医药）

登录后查看更多信息

990

项与 Mirdametinib 相关的文献（医药）

2025-07-01·DEVELOPMENTAL BIOLOGY

Characterisation of the avascular mesenchyme during digit outgrowth

Article

作者: Batho-Samblas, Cameron ; Smith, Jonathan ; Keavey, Lois ; Clancy, Noah ; McTeir, Lynn ; Davey, Megan G

The avascular mesenchyme at the tip of the developing digit contributes to digit outgrowth and patterning, however, it has been poorly characterised. Using newly developed fate mapping approaches, tissue manipulation and single-cell mRNA sequencing data, we explore the transcriptional nature and developmental potential of this tissue. We find that the avascular mesenchyme is essential to normal segmental patterning of the digit and has a distinct transcriptional identity. In addition, we uncover an unexpected relationship between the unspecified tissue of the avascular mesenchyme and the committed phalanx forming region, which controls patterning, but not outgrowth of the digit. This multifaceted approach provides insights into the mechanics and genetic pathways that regulate digit outgrowth and patterning.

2025-06-01·Animal Bioscience

Key events in the process of sex determination and differentiation in early chicken embryos

Article

作者: Gong, Wei ; Li, Zeyu ; Sun, Hongyan ; Qian, Hongwu ; Liu, Xin ; Zuo, Qisheng ; Li, Bichun ; Han, Wei ; Song, Jiuzhou ; Liu, Guanzheng ; Chen, Guo Hong ; Niu, Yingjie ; Zhu, Xilin ; Lv, Xiaoqian ; Wu, Jun ; Jin, Kai ; Sun, Changhua

Objective: Current understanding of sex determination and differentiation mechanisms during early chicken embryonic development remains incomplete. To address this, we applied RNA sequencing to identify male-female expression differences at critical developmental stages (E0 blastocysts, E3.5-E6.5 genital ridges, E18.5 gonads), focusing on glycolysis, histone acetylation, and DNA methylation. This approach aims to unravel key regulatory mechanisms and advance developmental biology insights.Methods: We analyzed molecular mechanisms of chicken sex determination at key stages (E0 blastocysts, E3.5-E6.5 genital ridges, E18.5 gonads) using RNA sequencing. Glycolysis, histone acetylation, and DNA methylation levels were assessed in embryonic stem cells and chicken embryonic fibroblasts. E18.5 gonads were treated with glycolytic activators (SB431542 and PD0325901 [2i]), a DNA demethylation activator (Vitamin C [Vc]), or an inhibitors of histone acetylation modification (valproic acid [VPA]). Sex-related gene expression, hormone levels, and gonad morphology were evaluated to determine treatment effects.Results: Key findings revealed that sex differences emerged as early as the blastocyst stage, intensified with embryonic development and were marked by a surge in sexually dimorphic gene expression. Gene Ontology and Kyoto encyclopedia of genes and genomes analyses highlighted the pivotal roles of energy metabolism and epigenetic modification process during this critical period. 2i, VC, or VPA interventions targeting E18.5 embryo gonads, induced a remarkable transformation of ovarian tissue into a testis-like structure, characterized by cortical thinning, medulla densification, downregulation of female-specific genes (FOXL2, WNT4), upregulation of male-specific genes (SOX9, AMH), and increased testosterone secretion. This phenotypic and molecular shift underscores the ability of metabolic and epigenetic modulators to reprogram ovarian development towards a male-like pattern, preserving male sexual characteristics.Conclusion: Our study establishes energy metabolism and epigenetic regulation as central drivers of avian sex determination. These findings advance understanding of vertebrate developmental biology and provide a framework for dissecting regulatory networks in avian sexual development.

2025-06-01·PHARMACOLOGICAL RESEARCH

Properties of FDA-approved small molecule protein kinase inhibitors: A 2025 update

Review

作者: Roskoski, Robert

Because of the deregulation of protein kinase action in many inflammatory diseases and cancer, the protein kinase family has become one of the most significant drug targets in the 21st century. There are 85 FDA-approved protein kinase antagonists that target about two dozen different enzymes and four of these drugs were approved in 2024 and a fifth was approved in 2025. Of these drugs, five target dual specificity protein kinases (MEK1/2), fourteen inhibit protein-serine/threonine protein kinases, twenty-one block nonreceptor protein-tyrosine kinases, and 45 target receptor protein-tyrosine kinases. The data indicate that 75 of these drugs are prescribed for the treatment of neoplasms. Seven drugs (abrocitinib, baricitinib, deucravacitinib, deuruxolitinib, ritlecitinib, tofacitinib, upadacitinib) are prescribed for the management of inflammatory diseases (atopic dermatitis, rheumatoid arthritis, psoriasis, alopecia areata, and ulcerative colitis). Of the 85 FDA-approved agents, about two dozen are used in the treatment of multiple diseases. The following four drugs received FDA approval in 2024 - deuruxolitinib (alopecia areata), ensartinib and lazertinib (non-small cell lung cancer), and tovorafenib (pediatric glioma) while mirdametinib was approved in 2025 for the treatment of type I neurofibromatosis (von Recklinghausen disease). Apart from netarsudil, temsirolimus, and trilaciclib, the approved protein kinase blockers are orally bioavailable. This article summarizes the physicochemical properties of all 85 FDA-approved small molecule protein kinase inhibitors including the molecular weight, number of hydrogen bond donors/acceptors, ligand efficiency, lipophilic efficiency, polar surface area, and solubility. A total of 39 of the 85 FDA-approved drugs have a least one Lipinski rule of 5 violation.

124

项与 Mirdametinib 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

2025-05-07

·贝壳社

2025 FDA新药前瞻：首创疗法投资趋势

作者：布布2025年，医药领域将迎来一场激动人心的创新浪潮，多款first-in-class疗法将成为医学研究的新亮点。这些药物不仅代表着科学家们在治疗策略上的重大突破，更有可能在多个疾病领域打开前所未有的治疗新局面。从癌症、罕见病到免疫系统疾病，这些创新疗法的出现或许意味着治疗方案的彻底变革，挑战了我们对传统药物和治疗模式的固有认知。它们通过全新的作用机制，精准锁定疾病根源，带来了更加个性化和高效的治疗方式。对于那些深受病痛折磨的患者来说，这些药物无疑是曙光，在医疗领域激发出无限的希望和可能性。01Donidalorsen: 首个靶向PKK的RNA疗法，有望改变HAE预防格局· 开发商：Ionis Pharmaceuticals· 模态：反义寡核苷酸· 适应症：预防遗传性血管性水肿（HAE）· 作用机制：靶向抑制血管性水肿相关基因的合成· PDUFA时间：2025年8月21日Donidalorsen 是 Ionis Pharmaceuticals 基于反义核酸技术平台开发的 RNA 靶向药物，用于预防遗传性血管性水肿（HAE）发作。遗传性血管性水肿是一种罕见的遗传性疾病，主要特点是反复发作性的皮肤、黏膜或内脏深部组织水肿，发作时可表现为手脚肿胀、面部浮肿、腹痛，严重时可出现咽喉水肿，危及生命。HAE的病因通常与C1酯酶抑制物（C1-INH）缺乏或功能异常有关，属于补体系统调节障碍所致。Donidalorsen通过抑制前激肽释放酶（prekallikrein, PKK）的生成，干预激肽-激肽酶系统，从源头阻断HAE发作的炎症通路。这一机制区别于现有C1酯酶抑制剂或bradykinin受体拮抗剂等治疗方案，使其具备first-in-class潜力。在III期OASIS-HAE与OASISplus研究中，Donidalorsen以每月或每两月给药的频率显著降低HAE发作频率，配合II期OLE研究中三年维持96%发作减少率的结果，显示其在疗效与长期控制方面的优势。其安全性良好，未观察到严重药物相关不良事件。Donidalorsen已获得孤儿药资格，PDUFA日期为2025年8月21日。作为首个以RNA技术靶向PKK的候选药物，该疗法有望为HAE预防治疗带来新的治疗机制和更便捷的用药方式。02Qfitlia（fitusiran）: 创新性治疗重度血友病的新突破· 开发商：赛诺菲· 模态：siRNA（小干扰RNA）· 适应症：A型和B型血友病的出血预防（适用于12岁及以上、无论是否携带凝血因子VIII或IX抑制物的患者）· 作用机制：抑制ANT3因子生成，减少抗凝血因子· 批准时间：2025年3月28日Qfitlia（fitusiran）是赛诺菲开发的siRNA疗法，也是首个获得FDA批准、以降低抗凝血酶（AT）水平为作用机制的血友病预防治疗药物。该药适用于12岁及以上A型或B型血友病患者，无论是否存在凝血因子VIII或IX的抑制物。其通过靶向肝脏合成抗凝血酶的mRNA，从源头上减少抗凝血酶水平，从而增强凝血酶生成，重建止血平衡。Qfitlia 的作用机制区别于传统的因子替代疗法或抗体疗法（如 emicizumab），具备first-in-class特征，并显著降低用药频率，可每年仅注射6次，采用预充式皮下注射笔，简化了患者的治疗体验。Qfitlia已获得FDA的孤儿药资格及突破性疗法认定（适用于有IX抑制物的B型血友病），全球多地区的注册工作正在推进，预计2025年下半年将在中国获得监管决定。作为一种具备长期保护、用药负担低和适应症覆盖广的创新疗法，Qfitlia 有望改变现有血友病预防治疗格局，特别是在抑制物阳性患者中的应用前景值得关注。03Ivonescimab：全球首个PD-1/VEGF双特异性抗体在肺癌治疗中的突破性进展· 开发商：康方生物· 模态：抗体· 适应症：晚期非小细胞肺癌一线治疗· 作用机制：PD-1/VEGF双特异性抗体Ivonescimab 是康方生物开发的全球首个PD-1/VEGF双特异性抗体，该药通过结合PD-1抑制免疫逃逸机制与VEGF抑制肿瘤血管生成，提供了免疫治疗和抗血管生成治疗的双重优势，从而有效增强抗肿瘤免疫反应。Ivonescimab在III期临床研究AK112-306/HARMONi-6中显示出显著的疗效。该研究的初期分析结果表明，Ivonescimab联合化疗在无进展生存期（PFS）方面显著优于BeiGene的PD-1抑制剂Tevimbra (tislelizumab-jsgr) 联合化疗。值得一提的是，Ivonescimab的这一成功是在与全球标杆药物Keytruda的头对头竞争后取得的又一重大胜利。早在HARMONi-2研究中，Ivonescimab就已经证明了其在与Keytruda单药治疗的比较中展现出强大的临床优势，显著降低了疾病进展或死亡的风险。与传统PD-1抑制剂相比，Ivonescimab的双特异性抗体结构不仅提供了免疫增强的效果，还通过抑制VEGF发挥抗血管生成作用，增强了肿瘤的免疫逃逸防线的突破力。这一创新机制使其在治疗上展现出比现有治疗方案更广泛的适应性与疗效。2025年4月，Ivonescimab新适应症获批，用于单药一线治疗PD-L1阳性（TPS≥1%）、表皮生长因子受体（EGFR）基因突变和间变性淋巴瘤激酶（ALK）基因突变阴性的非小细胞肺癌（NSCLC）。此前，该药物于2024年5月24日获得了国家药品监督管理局的首次批准，联合培美曲塞和卡铂，用于经 EGFR 酪氨酸激酶抑制剂（EGFR-TKI）治疗后进展的 EGFR 突变的局部晚期或转移性非鳞 NSCLC 的治疗。尽管市场上已有多种PD-1抑制剂，Ivonescimab通过其独特的机制和与化疗的协同作用，展现出强大的竞争力。随着HARMONi-6研究的成功，Ivonescimab的全球临床开发将在未来进一步推动其在美国、欧洲和日本的上市进程，并有望成为NSCLC治疗的新标准。04RGX-121：基因疗法的开创性突破，首个治疗MPS II的创新药物· 开发商：REGENXBIO/Nippon Shinyaku· 模态：基因疗法· 适应症：粘多糖贮积症II型（MPS II）· 作用机制：基因疗法修复酶缺失· PDUFA时间：待定RGX-121是首个针对粘多糖贮积症II型（MPS II，亨特综合症）开发的基因疗法。MPS II是一种由iduronate-2-sulfatase酶缺乏引起的罕见遗传代谢性疾病，导致糖胺聚糖（GAGs）的异常积累，进而引发严重的全身器官损害，包括中枢神经系统。当前该病没有治愈方法，治疗主要依赖酶替代疗法（ERT）来缓解症状。RGX-121通过基因疗法方式直接补充缺失的酶活性，提供了全新的治疗选择。作为first-in-class的药物，RGX-121的创新之处在于它通过基因疗法修复病，直接向患者体内传递正常基因，以恢复缺失的酶活性。这种机制区别于传统的酶替代疗法（ERT）和骨髓/干细胞移植，它不仅能够针对病因进行治疗，还可能实现长期的疗效，减少患者对常规治疗的依赖。通过这种方式，RGX-121有望成为MPS II治疗的里程碑药物，标志着基因疗法在遗传代谢病中的应用迈出了重要的一步。RGX-121已获得美国FDA的快速通道、罕见儿科疾病、再生医学先进疗法以及孤儿药物资格认证。其滚动生物制品许可申请（Rolling Biologics License Application）正在进行中，并显示出较好的临床前景。05Suzetrigine（Journavx）：非阿片类止痛药物的开创性突破，首个治疗急性疼痛的NaV1.8抑制剂· 开发商：Vertex Pharmaceuticals· 模态：小分子· 适应症：中度至重度急性疼痛· 作用机制：NaV1.8抑制剂· FDA批准时间：2025年1月30日Suzetrigine（Journavx）是Vertex制药公司首个获批的NaV1.8抑制剂，代表了止痛药物领域的重大创新。2025年1月30日获得FDA批准，成为20多年来首个新类别的止痛药物，也是全球首个非成瘾、可口服的NaV1.8抑制剂，标志着非阿片类止痛药物治疗的新时代。作为首个非阿片类NaV1.8抑制剂，Suzetrigine具有快速起效、与Vicodin®（复方阿片类镇痛药）相当的疗效以及无成瘾风险的独特优势。其精准选择性和强大的镇痛作用，使其成为解决急性疼痛的创新疗法，并成为治疗急性疼痛的新标杆。06Telisotuzumab Vedotin：首个靶向c-Met的ADC，开启NSCLC精准治疗新时代· 开发商：艾伯维· 模态：抗体偶联药物（ADC）· 适应症：c-Met高表达的非鳞状非小细胞肺癌（NSCLC）· 作用机制：靶向c-Met，释放细胞毒素杀伤肿瘤· PDUFA时间：2025年Telisotuzumab vedotin（Teliso-V）是一款靶向c-Met蛋白的first-in-class抗体偶联药物，专为既往接受过治疗的EGFR野生型、c-Met高表达的非鳞状NSCLC患者而设计。目前尚无已上市药物专门针对这一患者群体，Teliso-V有望填补重要空白，并获得了FDA的加速审批认定。FDA已将其纳入RTOR（实时肿瘤审评）计划并给予突破性疗法认定。作为c-Met过表达与预后不良显著相关的精准亚群，约25%的EGFR野生型NSCLC患者可能从Teliso-V中获益，因此其有潜力改变晚期肺癌的治疗格局。07Mirdametinib（Gomekli）：首个针对NF1相关性PN的口服MEK抑制剂，开启精准儿童肿瘤治疗新篇章· 开发商：SpringWorks Therapeutics· 模态：小分子· 适应症：I型神经纤维瘤病相关的不可切除丛状神经鞘瘤（NF1-PN）· 作用机制：抑制MEK1/2，阻断Ras-MAPK信号通路· FDA批准时间：2025年2月11日Mirdametinib是一款first-in-class的口服MEK1/2（丝裂原活化蛋白激酶激酶1/2）抑制剂，成为首个治疗NF1相关性PN的口服靶向药。丛状神经鞘瘤（plexiform neurofibromas, PNs）是生长在多个神经束上的良性肿瘤，起源于外周神经鞘细胞，但由于生长方式呈“网状”“丛状”，常深入肌肉、骨骼或其他组织，结构复杂，因此难以彻底切除。PNs多见于儿童期出现，可能随着年龄增长而不断增大，对患者造成疼痛、神经功能障碍、外观畸形和器官受压等。Mirdametinib的NDA申请获得了FDA优先审评资格，并于2025年2月11日获得批准。申请数据显示，Mirdametinib显著改善了患者肿瘤体积与疼痛程度，具备良好的耐受性，特别适用于儿童和青少年患者。相比现有静脉制剂，Mirdametinib的口服便利性为长期治疗提供更多可能，也体现出其在儿科精准肿瘤治疗领域的创新意义。08Plozasiran：填补FCS治疗空白的RNAi创新药· 开发商：Arrowhead Pharmaceuticals· 模态：RNA干扰（RNAi）疗法· 适应症：家族性乳糜微粒血症综合征（FCS）· 作用机制：靶向抑制APOC3，显著降低血浆甘油三酯水平· PDUFA时间：2025年11月18日家族性乳糜微粒血症综合征 (FCS) 是一种罕见但危及生命的遗传性高甘油三酯血症，其特征是由于乳糜微粒积聚导致血浆甘油三酯水平过度升高，最终导致甘油三酯水平极高。甘油三酯水平严重升高可引发严重症状，包括急性且可能致命的胰腺炎、慢性腹痛、糖尿病、肝脏脂肪变性和认知问题，目前尚无针对性疗法。Plozasiran作为first-in-class RNAi新药，通过干预APOC3这一关键致病蛋白，从机制上调控脂质代谢，显著改善病情。其三期PALISADE研究结果积极，已获得FDA突破性疗法、孤儿药和快速通道资格，或将成为FCS治疗领域的重大突破。09UGN-102 (Mitomycin): 创新的非侵入性治疗新选择· 开发公司：UroGen Pharma· 模态：小分子· 适应症：低级别中度风险非肌层浸润性膀胱癌（LG-IR-NMIBC）· 治疗机制：局部灌注应用美托霉素，美托霉素通过与DNA结合形成交联，抑制DNA的复制和转录· PDUFA时间：2025年6月13日UGN-102（Mitomycin）是一种用于治疗低级别中度风险非肌层浸润性膀胱癌（LG-IR-NMIBC）的非侵入性局部药物治疗。非肌层浸润性膀胱癌（NMIBC）是膀胱癌的一个常见类型，其特征是肿瘤局限于膀胱内壁的上皮层，并没有侵入膀胱肌层。LG-IR-NMIBC是一种较低风险但复发率较高的膀胱癌亚型，通常需要通过反复治疗来管理。NMIBC患者通常接受标准的手术治疗，如经尿道膀胱肿瘤切除术（TURBT），但这些治疗往往无法防止疾病的复发或进展。低级别中度风险膀胱癌（LG-IR-NMIBC）的治疗选择较为有限，且许多现有的药物治疗方法存在着治疗耐药性或较高的副作用。UGN-102作为一种非侵入性局部治疗药物，通过尿道灌注将药物直接送入膀胱，减少了传统药物治疗所带来的全身性副作用。其主要成分为Mitomycin，一种已知的化疗药物，通常用于治疗各种类型的癌症。UGN-102通过这种局部灌注的方式，直接作用于膀胱内的肿瘤细胞，从而有效地抑制肿瘤生长和复发。与传统的侵入性手术和系统性药物治疗不同，UGN-102通过非侵入性灌注方式提供了患者较低的治疗风险和更便捷的治疗体验。10UX111（rebisufligene etisparvovec）: MPS IIIA（圣菲利波综合征A型）革新性基因疗法· 开发公司：Ultragenyx· 模态：基因疗法· 适应症：MPS IIIA型（圣菲利波综合征A型）· 作用机制：AAV9载体递送正常SGSH基因，补充缺失的酶，促进肝素硫酸降解，改善MPS IIIA型患者的神经功能。· PDUFA日期：2025年8月18日UX111是一种基因治疗药物，针对罕见遗传病MPS IIIA型（圣菲利波综合征A型），这是一种由遗传缺陷引起的溶酶体贮积病。MPS IIIA型的根本病因是SGSH（硫酸氨基葡萄糖水解酶）基因的缺陷，导致硫酸肝素在体内积聚，进而引发神经退行性疾病。MPS IIIA型通常在2至6岁之间开始表现出症状，最初的症状通常是全身发育迟缓，并逐渐表现为认知和语言退化（随着病情进展，患儿会出现认知能力下降、语言能力丧失）、行为异常（如自闭症样症状、易怒和冲动行为）、运动和神经问题（肌肉无力、步态不稳，最终导致严重的行动不便）和早期死亡（患者通常在青少年或年轻成人时期因重度的神经退行性疾病而去世，平均寿命为15年左右）。MPS IIIA型目前没有批准的治疗方法。UX111通过一次性静脉注射AAV9（腺相关病毒9型）载体，将功能性SGSH基因传递到患者的细胞，恢复该酶的活性，从而减缓或逆转硫酸肝素积聚引起的神经退行性改变。它能够从基因层面解决疾病的根本原因，提供了突破性的治疗方案。UX111的临床试验数据已获得多个监管机构的支持，获得了美国FDA的再生医学先进疗法（RMAT）认证、快速通道认证、罕见儿科疾病认证和孤儿药认证。此外，EMA也授予该药物PRIME认证和孤儿药认证。这些认证反映了UX111在治疗MPS IIIA型方面的创新性及其临床应用的潜力。往期推荐1医健企业前沿动态2医健行业BD/出海/投融资洞察___*声明：本文仅为作者观点，不构成任何投资建议，且非治疗方案推荐。Ref.Ionis announces FDA acceptance of New Drug Application for donidalorsen for prophylactic treatment of HAE. Ionis Press Release. 04. 11. 2024.Press Release: Qfitlia approved as the first therapy in the US to treat hemophilia A or B with or without inhibitors. Sanofi Press Release. 28. 03. 2025.Dunleavy, K. With new trial win, Akeso bispecific shows prior success against Keytruda is no 'fluke'. Fierce Pharma. 23. 04. 2025.Nippon Shinyaku and REGENXBIO Enter into an Exclusive Partnership to Develop and Commercialize RGX-121 and RGX-111 for Mucopolysaccharidosis Diseases in the U.S. and Asia. Nippon Shinyaku Press Release. 13. 01. 2025.McAtee, R. Suzetrigine (VX-548): The 2024 Molecule of the Year. Drug Hunter. 08. 04. 2025.AbbVie Submits Biologics License Application to the FDA for Telisotuzumab Vedotin (Teliso-V) in Previously Treated Non-Small Cell Lung Cancer. AbbVie Press Release. 27. 09. 2024.Gomekli FDA Approval History. Drugs.com. 11. 02. 2025.Plozasiran FDA Approval Status. Drugs.com. 20. 01. 2025.Fabbricatore, R. FDA Accepts New Drug Application for UGN-102 for Bladder Cancer. Cancer Network. 16. 10. 2024.Ultragenyx Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA). UltraGenyx Press Release. 18. 02. 2025.Maryam Daneshpour. LinkedIn.关于贝壳社贝壳社是国内领先的医健创新创业服务平台，构建了"产业空间+创业教育+投资孵化+资本运作"四位一体服务体系，为医健领域创业企业提供全周期赋能。通过深度挖掘高成长项目、精准匹配产业资源及全球化生态网络，覆盖企业从孵化培育到加速发展的全链路，重点提供包括空间落地、创业培训、资本运作及跨境资源链接服务。贝壳社以加速科学技术成果转化与产业升级为目标，致力于成为医健领域的孵化器、加速器、连接器。

孤儿药siRNA寡核苷酸

100 项与 Mirdametinib 相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D11675	-	-	DB07101

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
神经纤维瘤病1型	美国	Springworks Therapeutics, Inc.	2025-02-11

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
NF1突变型丛状神经纤维瘤	申请上市	美国	Springworks Therapeutics, Inc.	2024-03-04
去分化脂肪肉瘤	临床2期	美国	Springworks Therapeutics, Inc.	2025-02-19
粘液样脂肪肉瘤	临床2期	美国	Springworks Therapeutics, Inc.	2025-02-19
晚期恶性实体瘤	临床2期	美国	Springworks Therapeutics, Inc.	2023-02-03
晚期恶性实体瘤	临床2期	澳大利亚	Springworks Therapeutics, Inc.	2023-02-03
皮肤黑色素瘤	临床2期	美国	Springworks Therapeutics, Inc.	2023-02-03
皮肤黑色素瘤	临床2期	澳大利亚	Springworks Therapeutics, Inc.	2023-02-03
低级神经胶质瘤	临床2期	美国	Springworks Therapeutics, Inc.	2021-06-21
结直肠癌	临床2期	荷兰	Pfizer Inc.	2014-01-01
KRAS突变非小细胞肺癌	临床2期	荷兰	Pfizer Inc.	2014-01-01

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03962543 (ReNeu, AAN2025)	临床2期	NF1突变型丛状神经纤维瘤	-	Mirdametinib capsule	膚簾鏇餘醖夢積齋壓壓(範糧製衊構醖艱顧製選) = 鬱觸糧淵製選鏇願鑰襯獵鹽積窪遞顧蓋遞積醖 (鏇鬱壓窪醖顧膚繭壓築, 29% ~ 55%) 更多	积极	2025-04-07
				Mirdametinib dispersible tablet	膚簾鏇餘醖夢積齋壓壓(範糧製衊構醖艱顧製選) = 艱膚窪窪鹹淵簾糧積範獵鹽積窪遞顧蓋遞積醖 (鏇鬱壓窪醖顧膚繭壓築, 38% ~ 65%) 更多
NCT03962543 (ReNeu, Pubmed \| J Clin Oncol)	临床2期	NF1突变型丛状神经纤维瘤	-	Mirdametinib capsules	鹹築衊壓顧鑰襯衊齋糧(願襯構築獵窪齋醖衊觸) = 遞築廠淵顧繭鹹鹹鏇醖遞壓範醖願選遞鹽糧鑰 (繭鹹築顧淵築鬱製顧製 )	积极	2025-02-20
				Mirdametinib tablets for oral suspension	鹹築衊壓顧鑰襯衊齋糧(願襯構築獵窪齋醖衊觸) = 網廠鬱構餘願顧壓選範遞壓範醖願選遞鹽糧鑰 (繭鹹築顧淵築鬱製顧製 )
NCT03962543 (ReNeu, SNO2024) 人工标引	临床2期	NF1突变型丛状神经纤维瘤	114	MIRDAMETINIB (adults)	遞鹹鏇構鹹憲繭獵簾鹽(繭鹽憲顧製壓鬱願壓糧) = Clinically meaningful improvement was defined as change from baseline >9.7 points for adults, >8.5 points for child self-report, and >9.0 points for parent proxy-report. PedsQL-TS improvement (least-squares mean, LSM [SE] change) from baseline at C13 was 3.9 (1.6; P=.018; n=34) for adults, 4.0 (2.4; P=.096; n=38) for children by self-report, and 5.6 (1.9; P=.005; n=43) by parent proxy-report. PedsQL-TS improvements for adults and children by parent proxy-report (not by children self-report) were observed early (at C5 and C3, respectively) and sustained through C24. Clinically meaningful improvement in PedsQL-TS from baseline at C13 was achieved by 37% (10/27) adults, 45% (13/29) children by self-report, and 47% (15/32) children by parent proxy-report (among patients who could have achieved a clinically meaningful change from baseline). Significant (P<.05) improvement from baseline to C13 was reported for physical (adults, children, and parent proxy-report), school/work (adults), and emotional and social (parent proxy-report) subscales. 築餘艱築鬱選糧鹹願鹹 (積選鑰選醖膚醖齋鹽衊 )	积极	2024-11-11
				MIRDAMETINIB (children)
NCT04923126 (SJ901, SNO2024) 人工标引	临床1期	低级神经胶质瘤 BRAF alteration \| FGFR1 alteration \| NF1 alteration ... 更多	23	MIRDAMETINIB	壓壓齋壓襯憲壓顧積衊(餘鹽築醖鬱觸壓鑰餘齋) = 餘構憲餘鹽構衊蓋糧廠鬱觸顧遞鏇鬱簾製製築 (壓願醖襯憲鑰簾餘窪憲 ) 更多	积极	2024-11-11
NCT03962543 (ReNeu, SNO2024 \| FDA_CDER) 人工标引	临床2期	神经纤维瘤病1型	114	mirdametinib (adults)	鹽壓襯醖鏇廠衊廠範願(範鹹鹽構襯築艱艱遞衊) = 淵鏇蓋膚衊獵觸鹽蓋壓淵衊夢艱鏇網淵製鹽衊 (膚網簾築網簾鏇網壓鬱, 29 ~ 55) 更多	积极	2024-11-11
				mirdametinib (children)	鹽壓襯醖鏇廠衊廠範願(範鹹鹽構襯築艱艱遞衊) = 窪遞壓蓋選獵鹹廠醖鑰淵衊夢艱鏇網淵製鹽衊 (膚網簾築網簾鏇網壓鬱, 38 ~ 65) 更多
NCT03962543 (ReNeu, SNO2024)	临床2期	丛状神经纤维瘤	12	MIRDAMETINIB (Prepubescent patients)	膚襯築膚鬱鏇餘構蓋簾(獵壓蓋顧醖齋窪網簾獵) = 壓蓋廠築網鑰鹹窪淵夢壓鏇糧壓鹽廠積製壓衊 (構餘鬱鹽膚齋觸衊襯積 )	-	2024-11-11
NCT03962543 (RENEU, EANO2024)	临床2期	NF1突变型丛状神经纤维瘤	114	Mirdametinib 2 mg/m^2 BID	鹹鹹鑰糧糧積製夢製餘(窪簾顧鏇淵選積淵網廠) = 夢積簾醖艱餘鹽醖艱蓋糧衊壓範蓋襯蓋鏇蓋鏇 (築餘衊遞鹽壓構衊蓋顧, 29 ~ 55) 更多	积极	2024-10-17
				Placebo	鹹鹹鑰糧糧積製夢製餘(窪簾顧鏇淵選積淵網廠) = 積醖願鏇蓋鏇網糧艱艱糧衊壓範蓋襯蓋鏇蓋鏇 (築餘衊遞鹽壓構衊蓋顧, 38 ~ 65) 更多
NCT05054374 (CTgov)	临床1/2期	晚期恶性实体瘤 \| 转移性乳腺癌 \| HER2 阴性乳腺癌	6	Fulvestrant+Mirdametinib (Arm 1, Part 1 - Mirdametinib in Combination With Fulvestrant)	遞構窪顧選餘襯廠顧構(選遞網鹹蓋艱鏇餘製構) = 夢築鹽顧積膚蓋顧醖構製繭鑰簾繭廠醖鏇選淵 (鬱艱蓋齋膚廠鑰獵願鏇, 構蓋餘鹹範壓範艱膚餘 ~ 願艱淵積窪獵獵壓選構) 更多	-	2024-07-09
				Fulvestrant+Mirdametinib (Arm 1, Part 2 - Mirdametinib in Combination With Fulvestrant)	遞構窪顧選餘襯廠顧構(選遞網鹹蓋艱鏇餘製構) = 獵鬱遞鹹窪醖鹽選簾艱製繭鑰簾繭廠醖鏇選淵 (鬱艱蓋齋膚廠鑰獵願鏇, 簾襯餘憲廠夢觸獵鬱鑰 ~ 壓壓獵鹽鹹淵膚憲壓網) 更多
NCT03962543 (ReNeu, ASCO2024) 人工标引	临床2期	NF1突变型丛状神经纤维瘤	114	Mirdametinib 2 mg/m2 BID	齋襯鬱襯鑰願製構廠蓋(構衊繭獵窪鬱醖齋顧憲) = 膚夢觸膚鑰蓋選積壓鬱壓壓艱簾鹽顧衊鏇壓鹹 (醖憲遞觸鏇願壓選糧醖, 29 ~ 55) 更多	积极	2024-05-24
				Mirdametinib (pediatric)	齋襯鬱襯鑰願製構廠蓋(構衊繭獵窪鬱醖齋顧憲) = 鹽鏇構鹽夢衊鏇遞網鏇壓壓艱簾鹽顧衊鏇壓鹹 (醖憲遞觸鏇願壓選糧醖, 38 ~ 65) 更多
NCT03962543 (ReNeu, GlobeNewswire) 人工标引	临床2期	丛状神经纤维瘤 NF1	114	Mirdametinib (pediatric patients)	獵襯網廠鬱願構淵壓鏇(膚齋簾醖構構夢膚遞齋) = 蓋齋艱願糧艱齋網築顧鏇鏇築繭鏇蓋鏇繭窪艱 (蓋觸夢鬱鑰築構廠選淵 ) 更多	积极	2023-11-16
				Mirdametinib (adult patients)	獵襯網廠鬱願構淵壓鏇(膚齋簾醖構構夢膚遞齋) = 餘壓衊衊醖鏇蓋觸襯餘鏇鏇築繭鏇蓋鏇繭窪艱 (蓋觸夢鬱鑰築構廠選淵 ) 更多