This is a randomized, double-blind, placebo-controlled within-participant crossover study in healthy male participants 18-55 years of age to assess pain tolerance during a cold pressor test.
The study will be conducted at a single center in New Zealand.

开始日期2025-06-04

申办/合作机构

Latigo Biotherapeutics, Inc.

NCT06887959

/ Recruiting临床4期

A Phase 4, Open-label, Single-arm Study Evaluating the Effectiveness and Safety of Suzetrigine As Part of Multimodal Therapy for Acute Pain After Laparoscopic Procedures of the Intraperitoneal or Retroperitoneal Cavities or Arthroscopic Orthopedic Procedures

The purpose of this study is to evaluate the effectiveness, safety, and tolerability of SUZ for acute pain after laparoscopic procedures of the intraperitoneal or retroperitoneal cavities or arthroscopic orthopedic procedures.

开始日期2025-03-27

申办/合作机构

Vertex Pharmaceuticals, Inc.

NCT06887972

/ Recruiting临床4期

A Phase 4, Open-label, Single-arm Study Evaluating the Effectiveness and Safety of Suzetrigine As Part of Multimodal Therapy for Acute Pain After Aesthetic or Reconstructive Surgeries

The purpose of this study to evaluate the effectiveness, safety, and tolerability of SUZ as part of multimodal therapy (MMT) in treating acute postoperative pain.

开始日期2025-03-21

申办/合作机构

Vertex Pharmaceuticals, Inc.

100 项与苏泽曲林相关的临床结果

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100 项与苏泽曲林相关的转化医学

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100 项与苏泽曲林相关的专利（医药）

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项与苏泽曲林相关的文献（医药）

2025-07-07·JOURNAL OF GENERAL PHYSIOLOGY

Differential state-dependent Nav1.8 inhibition by suzetrigine, LTGO-33, and A-887826

Article

作者: Fujita, Akie ; Bean, Bruce P. ; Osorno, Tomás ; Jo, Sooyeon ; Stewart, Robert G. ; Vaelli, Patric M.

Nav1.8 sodium channels are expressed in pain-sensing neurons, and some Nav1.8 inhibitors significantly reduce pain in clinical trials. Several Nav1.8 inhibitors have an unusual state dependence whereby inhibition is relieved by depolarization. We compared the state-dependent action of several Nav1.8 channel inhibitors to test whether inhibition is relieved during action potential (AP) firing under physiological conditions to produce “reverse use dependence.” A-887826 inhibition was substantially relieved by AP waveforms applied at 20 Hz at 37°C. In contrast, there was no relief during AP trains with suzetrigine (VX-548) or LTGO-33, even though inhibition could be effectively removed by long, strong depolarizations. These differences were explained by differences in the voltage dependence and kinetics with which the compounds dissociate from depolarized channels and rebind to resting state channels. Suzetrigine required the strongest depolarizations for relief (midpoint +33 mV) and relief was slow (tau >300 ms at +20 mV), so almost no relief occurred during an AP waveform. Relief from A-887826 required weaker depolarizations (midpoint +13 mV) and was much faster, so some relief occurred during each AP waveform and accumulated during 20-Hz trains. LTGO-33 required the weakest depolarizations for relief (midpoint −11 mV) and relief was even faster than for A-887826, but reinhibition between AP waveforms was far faster than for A-887826, so that relief did not accumulate during AP trains at 20 Hz. The results show that, unlike A-887826, there is no use-dependent relief of inhibition by suzetrigine or LTGO-33 with physiological voltage waveforms at physiological temperatures, but each for different reasons.

2025-07-07·JOURNAL OF GENERAL PHYSIOLOGY

Sensory neuron sodium channels as pain targets; from cocaine to Journavx (VX-548, suzetrigine)

Review

作者: Nassar, Mohammed A. ; Zhao, Jing ; Woods, C. Geoffrey ; Yan, Nieng ; Huang, Jian ; Wood, John N. ; Akopian, Armen ; Cox, James J.

Voltage-gated sodium channels underpin electrical signaling in sensory neurons. Their activity is an essential element in the vast majority of pain conditions, making them significant drug targets. Sensory neuron sodium channels play roles not only in afferent signaling but also in a range of efferent regulatory mechanisms. Side effects through actions on other cell types and efferent signaling are thus important issues to address during analgesic drug development. As an example, the human genetic evidence for NaV1.7 as an ideal pain target contrasts with the side effects of NaV1.7 antagonists. In this review, we describe the history and progress toward the development of useful analgesic drugs and the renewed focus on NaV1.8 as a key target in pain treatment. NaV1.8 antagonists alone or in combination with other analgesics are likely to provide new opportunities for pain relief for the vast number of people (about 33% of the population) impacted by chronic pain, particularly present in aging populations.

2025-06-01·ANESTHESIOLOGY

Suzetrigine, a Nonopioid NaV1.8 Inhibitor for Treatment of Moderate-to-severe Acute Pain: Two Phase 3 Randomized Clinical Trials

Article

作者: Weiner, Scott G. ; Negulescu, Paul ; Solanki, Daneshvari ; D’Aunno, Dominick ; Miao, Xiaopeng ; Swisher, Matthew W. ; Correll, Darin J. ; McCoun, Jessica ; Tian, Simon ; Urban, Joshua ; Oswald, Jessica ; Bozic, Carmen ; Bertoch, Todd ; Taber, Louise

Background::

Opioids are effective for treating acute pain but have safety, tolerability, and addiction concerns while nonopioid analgesics have limited efficacy. Suzetrigine, an oral, nonopioid small molecule, selectively inhibits the voltage-gated sodium channel 1.8 (NaV1.8) and has potential to provide efficacious and safe relief for acute pain without addiction concerns.

Methods::

To evaluate suzetrigine for treatment of acute pain, two phase 3, randomized, double-blind, placebo- and active-controlled trials were conducted in adults with moderate-to-severe acute pain on the verbal categorical rating scale and 4 or greater on the numeric pain rating scale after abdominoplasty (n = 1,118) or bunionectomy (n = 1,073). After surgery, participants were randomized to suzetrigine (100 mg, then 50 mg every 12 h), hydrocodone bitartrate/acetaminophen (5/325 mg every 6 h), or placebo for 48 h. The primary endpoint was time-weighted sum of the pain intensity difference in numeric pain rating scale from 0 to 48 h (SPID48) versus placebo. Key secondary endpoints were SPID48 versus hydrocodone bitartrate/acetaminophen and time to 2-point or greater reduction in numeric pain rating scale from baseline versus placebo.

Results::

The primary endpoint was achieved in both trials with suzetrigine demonstrating statistically significant and clinically meaningful reduction in pain versus placebo. The least squares mean difference in SPID48 between suzetrigine and placebo was 48.4 (95% CI, 33.6 to 63.1; P < 0.0001) after abdominoplasty and 29.3 (95% CI, 14.0 to 44.6; P = 0.0002) after bunionectomy. Neither trial achieved the first key secondary endpoint of superiority of suzetrigine versus hydrocodone bitartrate/acetaminophen on SPID48. For the second key secondary endpoint of time to 2-point or greater reduction in numeric pain rating scale, suzetrigine had a more rapid onset of clinically meaningful pain relief versus placebo after abdominoplasty (119 min vs. 480 min; nominal P < 0.0001) and bunionectomy (240 min vs. 480 min; nominal P = 0.0016). Adverse events were similar to those seen in postsurgical settings.

Conclusions::

As compared with placebo, suzetrigine reduced moderate-to-severe acute pain over 48 h after abdominoplasty or bunionectomy. Pain reduction with suzetrigine was similar to that with hydrocodone bitartrate/acetaminophen. Suzetrigine was associated with adverse events that were mild to moderate in severity.

266

项与苏泽曲林相关的新闻（医药）

2025-05-08

·Firstwordpharma

Viatris plans year-end FDA filing for fast-acting pain drug

Viatris said Thursday it is looking to file for FDA approval by year-end after its rapid-onset meloxicam candidate — dubbed MR-107A-02 — succeeded in two Phase III studies involving patients recovering from hernia repair or bunion surgeries.Its formulation of the commonly used non-steroidal anti-inflammatory drug demonstrated statistically significant pain reduction across both acute pain models while also showing significant decreases in opioid use compared to placebo."Building on an established mechanism of action and well-characterised safety profile, the efficacy and benefit-risk profile observed in these two pivotal studies optimally positions our fast-acting meloxicam for potential first-line treatment of moderate-to-severe acute pain," commented Chief R&D Officer Philippe Martin.According to Viatris, patients treated with MR-107A-02 reported significantly more pain relief from baseline over 48 hours compared to placebo, measured using Sum of Pain Intensity Difference (SPID0-48h). After herniorrhaphy, the mean improvement in pain scores was 50.1 points higher for MR-107A-02; and following bunionectomy, the difference was even greater — an average of 82.7 points.In addition, 72.6% of the hernia patients and 56.9% of bunion patients treated with MR-107A-02 remained opioid-free over the entire treatment phase, compared to placebo rates of 58.6% and 33.1%, respectively.Both studies also included an opioid comparator arm — tramadol given every six hours — to confirm the sensitivity of the pain model. Viatris said post-hoc analyses showed that MR-107A-02 provided "superior pain control" over 48 hours than tramadol in both types of surgery, and was either faster or on par with the widely used opioid drug in terms of the time it took to achieve perceptible or meaningful pain relief.Companies have been trying to address the US opioid crisis with effective alternatives. Vertex Pharmaceuticals' non-opioid pain medication Journavx (suzetrigine), which received FDA approval at the end of January, is a recent entrant in the space. Earlier this week, Vertex reported only nominal first-quarter revenue for Journavx, which works by blocking NaV1.8 voltage-gated sodium channels, but company officials said the drug had racked up about 25,000 prescriptions as of April 25."Although it's early days, we're seeing tremendous receptivity to a novel non-opioid option for the treatment of moderate to severe acute pain," said Duncan McKechnie, Vertex's soon-to-be chief operating officer, during the company's earnings call (see – Spotlight On: Vertex plots $1B+ sales trajectories for Casgevy, Journavx).Meanwhile, Viatris reported that MR-107A-02 was generally well tolerated, with treatment-emergent adverse events comparable to placebo and "few" severe adverse events. The company plans to present full results from both Phase III trials at upcoming medical conferences, including the PAINWeek conference in September, as it prepares its FDA submission based on the two post-op studies as well as earlier Phase II dose range finding data in dental pain.

临床结果临床3期临床2期

2025-05-08

·BioPharmaDive

Viatris’ new form of old pain drug scores in large trials

Pittsburgh-based pharmaceutical firm Viatris on Thursday said a pain drug its been developing succeeded in two large clinical trials, setting the stage for an approval filing later this year.The drug is a reformulated version of an old medication, meloxicam, designed to more quickly treat the sharp, acute pain felt after an injury or operation. Researchers evaluated the drug in a dental pain study in 2022. Not long after, a pair of late-stage clinical trials began assessing it in nearly 1,000 people who had just undergone either a bunion removal or a hernia repair surgery.Results from those trials, according to Viatris, now show that pain scores improved substantially more for participants given the drug rather than a placebo. The main goal of both experiments revolved around a scoring system that measures pain intensity over a two-day period. Its the same system Vertex Pharmaceuticals used in the studies that ultimately led to the approval of Journavx, a closely watched, non-opioid pain reliever.Viatris, like Vertex, also set up arms in its trials to compare the relief provided by its drug to that of an opioid. The company said after-the-fact analyses found the drug offered significantly superior pain control to tramadol. The amount of time it took for participants to sense some pain reduction and to classify that reduction as meaningful was also comparable between the two groups and, in some cases, shorter for those on Viatris therapy.Journavx did not significantly outperform the opioid arms in Vertexs studies, though those used a combination of Tylenol and a more potent opioid, hydrocodone.Additionally, Viatris said that during the main treatment phase of its studies, there was a notable reduction in opioid usageas well as a higher number of opioid-free patients in the drug group as opposed to the placebo group. Patients also generally tolerated the drug well. There were few so-called severe treatment emergent adverse events, and the incidence of them was comparable across the two groups.All this evidence optimally positions Viatris drug to become a first-line treatment for moderate-to-severe acute pain, according to Philippe Martin, the companys research and development head. “The data observed ... is a critical step in the development of a safe and effective non-opioid option to address an important public health need, he said in a statement.The trial results were released alongside Viatris first quarter earnings report. The company, which officially formed in 2020, when Mylan merged with Pfizers Upjohn unit, recorded $3.3 billion between January and March, an 11% decrease from the same period a year prior. Viatris shares were up over 10% late Thursday morning, to trade around $9.48 apiece.Shares of Vertex, meanwhile, were down more than 2%, capping off a week in which the company lost tens of billions of dollars in value thanks, in part, to an earnings report that disappointed investors.Michael Yee, a Jefferies analyst who covers Vertex, wrote in a note to clients that first quarter revenue and earnings per share were light. But what stood out was the company saying Journavx revenue was “insignificant during the period, even though cumulative prescriptions had grown to around 25,000 by late April.Brian Skorney, a Baird analyst who also follows Vertex, wrote in his own note that the new Viatris data highlight a number of concerns we have with Journavxs profile, in particular, onset of action, a critical factor for post-operative pain.Skorney went on to suggest the respective bunionectomy studies make Journavx look like an inferior option compared to a pretty old albeit new and fast-acting non-steroidal anti-inflammatory drug, or NSAID.If NSAID's don't have the highest efficacy, as Vertex is fond of saying, what does that say about Journavx? he wrote. '

财报临床结果

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 项与苏泽曲林相关的药物交易

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适应症	国家/地区	公司	日期
急性疼痛	美国	Vertex Pharmaceuticals, Inc.	2025-01-30

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适应症	最高研发状态	国家/地区	公司	日期
糖尿病周围神经性疼痛	临床3期	美国	Vertex Pharmaceuticals, Inc.	2024-10-01
腰骶神经根病变	临床2期	美国	Vertex Pharmaceuticals, Inc.	2023-12-13
疼痛	临床1期	澳大利亚	Vertex Pharmaceuticals, Inc.	2022-12-15

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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


NCT05553366 (FDA_CDER) 人工标引	临床3期	急性疼痛	1,073	JOURNAVX	構襯鏇顧淵願積齋構憲(鹽鬱廠鏇憲艱範鹹顧製) = 廠壓積鬱顧餘憲淵艱膚鹽構窪夢繭遞簾憲願夢 (獵壓壓範顧製艱積獵願 ) 更多	积极	2025-01-30
				Placebo	構襯鏇顧淵願積齋構憲(鹽鬱廠鏇憲艱範鹹顧製) = 選鏇醖觸衊艱夢糧窪簾鹽構窪夢繭遞簾憲願夢 (獵壓壓範顧製艱積獵願 ) 更多
NCT05558410 (FDA_CDER) 人工标引	临床3期	急性疼痛	1,118	JOURNAVX	壓構壓遞醖願鏇齋壓窪(製構範鏇鬱餘齋膚構鬱) = 艱構選願齋網夢鏇簾鹽鹹繭艱網構獵願廠齋鑰 (顧範願顧鏇構遞齋齋窪 ) 更多	积极	2025-01-30
				Placebo	壓構壓遞醖願鏇齋壓窪(製構範鏇鬱餘齋膚構鬱) = 積艱淵窪構鹽醖構壓衊鹹繭艱網構獵願廠齋鑰 (顧範願顧鏇構遞齋齋窪 ) 更多
NCT05034952 (VX21-548-102, CTgov)	临床2期	急性疼痛	303	Placebo (matched to HB/APAP) (Placebo)	淵鹽選淵餘衊願鬱醖憲(積衊選範艱蓋廠獵襯衊) = 夢願願淵願遞範構鹽築繭淵壓壓糧積糧壓餘蓋 (鹹壓糧壓遞觸憲膚繭餘, 10.23) 更多	-	2024-12-27
				HB/APAP (HB/APAP)	淵鹽選淵餘衊願鬱醖憲(積衊選範艱蓋廠獵襯衊) = 獵網鏇範淵鬱繭鏇餘願繭淵壓壓糧積糧壓餘蓋 (鹹壓糧壓遞觸憲膚繭餘, 10.30) 更多
NCT06176196 (Company_Website) 人工标引	临床2期	腰骶神经根病变	217	Suzetrigine	餘願齋鑰糧壓廠窪廠鬱(齋積鹹顧顧願鹽積艱鑰) = 鹽構餘窪構繭膚鹹範醖廠願壓醖鏇鬱廠餘襯艱 (憲淵繭艱廠蓋餘遞鏇憲, -2.40 ~ -1.64) 达到更多	积极	2024-12-19
				Placebo	餘願齋鑰糧壓廠窪廠鬱(齋積鹹顧顧願鹽積艱鑰) = 糧顧膚遞鑰鏇觸壓糧糧廠願壓醖鏇鬱廠餘襯艱 (憲淵繭艱廠蓋餘遞鏇憲, -2.36 ~ -1.60) 达到更多
Biospace 人工标引	临床3期	疼痛	-	Suzetrigine	構壓構膚膚窪餘鬱齋窪(簾鏇積壓襯範顧範鹽獵) = 範構鹽製構膚鹽獵簾築壓夢鏇廠鹽積膚鹹構鑰 (遞鏇選鬱糧膚構獵夢襯 ) 更多	积极	2024-10-21
				Hydrocodone bitartrate/acetaminophen (HB/APAP)	構壓構膚膚窪餘鬱齋窪(簾鏇積壓襯範顧範鹽獵) = 鑰鬱蓋獵遞廠網夢願繭壓夢鏇廠鹽積膚鹹構鑰 (遞鏇選鬱糧膚構獵夢襯 ) 更多
NCT05558410 (PipelineReview) 人工标引	临床3期	急性疼痛	1,118	VX-548	夢選襯範鑰糧夢壓構醖(艱膚觸觸觸夢艱鹽蓋構) = 獵積餘鑰糧鬱鹹窪範憲簾願膚襯築製窪鹽鹽餘 (餘鬱願蓋獵餘選憲構遞, 4.3) 更多	积极	2024-01-30
				Hydrocodone bitartrate/acetaminophen	夢選襯範鑰糧夢壓構醖(衊願夢鏇願顧廠繭廠膚) = 範鹹膚鏇獵願餘鬱膚蓋衊鏇衊襯齋構糧繭構衊 (餘顧壓獵鏇顧簾廠膚築, 4.3)
NCT05553366 (PipelineReview) 人工标引	临床3期	急性疼痛	1,073	VX-548	餘構蓋鹽獵餘蓋鬱醖艱(艱選壓衊齋願簾窪糧鑰) = 繭鏇觸鏇蓋選鏇獵壓糧網廠廠繭鹽構膚鹽觸蓋 (獵構鹽鹽夢鏇糧鹹積餘, 4.5) 更多	积极	2024-01-30
				Hydrocodone bitartrate/acetaminophen	餘構蓋鹽獵餘蓋鬱醖艱(憲艱夢積窪觸範齋鹹窪) = 窪窪繭憲餘鬱鹹鑰襯蓋願繭餘衊鬱顧網鹽蓋艱 (積壓選齋願鹽鏇壓觸範 )
NCT05661734 (PipelineReview) 人工标引	临床3期	急性疼痛	256	VX-548	網積憲夢願醖構範艱網(鏇鹹膚淵製築範艱艱齋) = mostly mild or moderate 糧壓醖夢餘鑰繭獵簾憲 (鏇夢觸衊繭觸糧製糧壓 ) 更多	积极	2024-01-30
NCT05660538 (Corporate Publications) 人工标引	临床2期	糖尿病周围神经病变	192	VX-548 23 mg qd	蓋壓鹽選獵壓構繭鬱襯(鑰鏇顧築襯繭衊鹽顧淵) = 製餘夢鬱獵獵夢淵築壓齋網網鹹構網範構鹹膚 (蓋鑰鹽範鹽鑰築壓糧構, 0.39) 更多	积极	2023-12-13
				VX-548 46 mg qd	蓋壓鹽選獵壓構繭鬱襯(鑰鏇顧築襯繭衊鹽顧淵) = 顧壓齋獵顧糧襯網衊範齋網網鹹構網範構鹹膚 (蓋鑰鹽範鹽鑰築壓糧構, 0.28) 更多
NCT05034952 (VX21-548-102, Literature) 人工标引	临床2期	急性疼痛	303	VX-548 (Abdominoplasty; the high-dose group)	鏇構網鏇膚淵餘齋艱齋(淵鬱遞淵淵廠繭衊窪淵) = 鬱範糧齋膚糧壓範範繭積鬱積衊淵繭膚蓋遞窪 (襯願製顧艱醖鬱襯窪鬱, 10.3) 更多	积极	2023-08-03
				VX-548 (Abdominoplasty; the middle-dose group)	鏇構網鏇膚淵餘齋艱齋(淵鬱遞淵淵廠繭衊窪淵) = 襯壓壓鹽齋廠餘蓋網窪積鬱積衊淵繭膚蓋遞窪 (襯願製顧艱醖鬱襯窪鬱, 10.4) 更多