A Phase 3, Randomized, Crossover, Open-Label, Active-Controlled Study of Sepiapterin versus Sapropterin in Participants With Phenylketonuria ≥2 years of Age

开始日期2024-05-10

申办/合作机构

PTC Therapeutics, Inc.

NCT06302348

/ Recruiting临床3期

A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin

The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in early childhood.

开始日期2024-03-04

申办/合作机构

PTC Therapeutics, Inc.

100 项与塞匹蝶呤相关的临床结果

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100 项与塞匹蝶呤相关的转化医学

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100 项与塞匹蝶呤相关的专利（医药）

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436

项与塞匹蝶呤相关的文献（医药）

2026-07-01·BIOCHEMICAL PHARMACOLOGY

Targeted endothelial GTPCH1/BH4 pathway activation reverses vascular dysfunction in smoking-associated obesity

Article

作者: Zhang, Wei ; Bu, Wen-Jie ; Bai, Yun-Long ; Zheng, Dong-Jie ; Dong, Chao-Run ; Lian, Guang ; Fan, Zhe-Yu ; Yu, Yi ; Lu, Dong ; Li, Si-Si

Obesity and cigarette smoking are two major cardiovascular risk factors that frequently coexist, yet their combined impact on vascular function and the underlying mechanisms remains insufficiently defined. Here, we show that short-term cigarette smoke (CS) exposure induces persistent endothelial dysfunction exclusively in obese mice, while having minimal vascular effects in lean mice. Obese mice exposed to CS exhibited severely impaired acetylcholine-induced vasodilation, elevated blood pressure, sustained vascular oxidative stress, and accelerated endothelial senescence. Integrated metabolomic and transcriptomic analyses revealed that CS superimposed on obesity drives a distinct metabolic and transcriptional reprogramming characterized by disrupted redox homeostasis and inflammatory signaling. Mechanistically, CS selectively suppressed endothelial GTP cyclohydrolase 1 (GTPCH1) expression in obese mice, resulting in tetrahydrobiopterin (BH4) depletion, endothelial nitric oxide synthase (eNOS) uncoupling, reduced nitric oxide bioavailability, and increased superoxide production. Endothelial-specific restoration of GTPCH1 expression or pharmacological treatment with sepiapterin restored eNOS coupling, normalized endothelial redox balance, and rescued vascular function. In summary, we demonstrate that CS leads to sustained endothelial dysfunction in the setting of obesity, a phenomenon that is associated with disruption of the GTPCH1/BH4/eNOS axis. This provides a mechanistic explanation for the heightened cardiovascular risk in obese smokers and identifies actionable molecular targets for intervention.

2026-05-01·METABOLISM-CLINICAL AND EXPERIMENTAL

Efficacy and safety of sepiapterin versus sapropterin in patients with phenylketonuria: Results from the Phase 3, randomized, crossover, open-label, active-controlled AMPLIPHY trial

Article

作者: van Spronsen, Francjan ; Peters, Heidi ; Hughes, Catalina ; Bélanger-Quintana, Amaya ; Potter, Murray ; Maillot, François ; Ingalls, Kim ; Smith, Neil ; Tchan, Michel ; Burlina, Alberto ; Inwood, Anita ; Tyčová, Renáta ; Grošelj, Urh ; Vijay, Suresh ; Chakrapani, Anupam ; Rutsch, Frank ; Guilder, Laura ; Venegas-Moreno, Eva Maria ; Giżewska, Maria ; Fjellbirkeland, Olivia ; Zhao, Zhenming ; Muntau, Ania C ; Arnoux, Jean-Baptiste

AIM:

AMPLIPHY is the first Phase 3 study comparing sepiapterin versus sapropterin in children and adults with phenylketonuria (PKU).

METHODS:

AMPLIPHY was an international, Phase 3, two-part, open-label study in participants with PKU aged ≥2 years. Participants responsive to sepiapterin (60 mg/kg/day) in Part 1 (≥20% reduction in blood phenylalanine [Phe]) entered Part 2, a crossover treatment period, and were randomized 1:1 to alternative treatment sequences of sepiapterin (60 mg/kg/day, licensed dosage) and sapropterin (20 mg/kg/day, maximum licensed dosage) for 4 weeks each, with a 14-day washout between treatments. The primary endpoint was mean change in blood Phe from baseline to Weeks 3-4 of each treatment period (Part 2).

RESULTS:

Of 82 participants enrolled, 67 (81.7%) and 62 (75.6%) had reductions in blood Phe ≥20% and ≥30%, respectively, in Part 1. Sixty-two participants were randomized in Part 2 (mean [SD] age, 15.8 [10.8] years). In the primary analysis set (≥30% reduction in blood Phe in Part 1, n = 58), mean (SD) baseline blood Phe before sepiapterin and sapropterin treatment was 725.8 (302.1) and 790.4 (370.0) μmol/L, respectively. Least-squares mean (SE) reduction in blood Phe from baseline was -437.0 (28.0) and -256.6 (28.2) μmol/L, respectively, representing a least-squares mean difference of -180.4 μmol/L (95% CI: -229.5, -131.4; p < 0.0001) and a relative 70% greater reduction with sepiapterin versus sapropterin. Both treatments were well tolerated, with safety profiles consistent with previous reports.

CONCLUSIONS:

Sepiapterin was superior to the highest approved dose of sapropterin in lowering blood Phe. No new safety signals were observed. The trial was registered in the UK Clinical Study Registry, ISRCTN, on January 29, 2024 (ID number, ISRCTN79102999; https://www.isrctn.com/ISRCTN79102999).

2026-04-01·GENETICS IN MEDICINE

Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY Extension Study

Article

作者: Vijay, Suresh ; Thomas, Janet A ; Giżewska, Maria ; Fjellbirkeland, Olivia ; Gao, Lan ; Lund, Allan ; Mungan, Halise ; Guimas, Arlindo ; Ingalls, Kimberly ; Sacharow, Stephanie ; Burlina, Alberto ; Canda, Ebru ; Peters, Heidi ; Rutsch, Frank ; Minami, Maria ; Opladen, Thomas ; Hughes, Catalina ; Plana, Jaume Campistol ; Oliveira, Anabela ; Hamazaki, Takashi ; Margvelashvili, Lali ; van Spronsen, Francjan ; Vockley, Jerry ; Smith, Neil ; Tchan, Michel ; Muntau, Ania C ; Manti, Filippo ; Bélanger-Quintana, Amaya ; Bratkovic, Drago ; Chakrapani, Anupam ; Kiykim, Ertuğrul ; Inwood, Anita ; Liu, Emelline ; Longo, Nicola ; Larkin, Alex ; Yıldız, Yılmaz ; Ito, Tetsuya ; Lah, Melissa ; MacDonald, Anita ; Ezgü, Fatih ; Schwartz, Ida Vanessa D ; Janeiro, Patricia ; Zori, Roberto ; Ishige, Mika ; Agladze, Dodo ; Guerra, Ixiu-Cabrales ; Breilyn, Margo Sheck ; Guilder, Laura ; Khan, Aneal

PURPOSE:

To report interim results from the ongoing, open-label, phase 3 APHENITY Extension Study (NCT05166161), evaluating long-term treatment with sepiapterin in patients with phenylketonuria.

METHODS:

Participants received an age-based dose of oral sepiapterin daily; those with mean blood phenylalanine (Phe) levels <360 μmol/L (<5.95 mg/dL) after 2 weeks underwent a 26-week dietary Phe tolerance assessment, wherein dietary Phe intake was adjusted and blood Phe levels monitored. Other participants continued treatment with optional diet liberalization. Primary endpoints included change from baseline to week 26 in dietary Phe intake and treatment-emergent adverse events (TEAEs).

RESULTS:

As of September 2, 2024, 169 participants received sepiapterin (median [minimum, maximum] age: 14.0 [0.2, 55.0] years, median exposure: 72.9 weeks); 102 participants underwent dietary Phe tolerance assessments. Mean (SD) dietary Phe intake increased from 27.6 (18.0) mg/kg/day at baseline to 62.5 (41.5) mg/kg/day at week 26 (least-squares mean change [SE]: 36.4 [2.8] mg/kg/day from baseline) (P < .0001 from post hoc analysis). The incidence of treatment-related TEAEs was 29.0%; 3 participants (1.8%) discontinued treatment owing to treatment-related TEAEs. There were no treatment-related serious TEAEs or deaths.

CONCLUSION:

Interim results support the long-term safety of sepiapterin and demonstrate the potential for diet liberalization in adults and children with phenylketonuria.

CLINICALTRIALS:

gov/study/NCT05166161; date of registration, December 8, 2021).

GOV IDENTIFIER:

NCT05166161 (https://www.

133

项与塞匹蝶呤相关的新闻（医药）

2026-04-22

·酷炫z

2025年美国FDA批准新药简析

前言：这几天美国加州宜居城市圣地亚哥正在热闹召开美国癌症研究协会（AACR）2026年度会议，各路大咖纷纷到场。AACR每年发布的前沿成果，尤其是关于新靶点发现、临床前研究数据和早期临床数据，预示了未来癌症治疗的发展方向。AACR年会不仅是全球肿瘤研究领域的权威学术盛会，更是新药审批和临床研发的风向标。圣地亚哥是全球三大生命科学中心之一，在生物技术集中度、资金投入和人才储备方面，与波士顿、旧金山湾区并列为全球生命科学高地。尽管近几年资本市场下滑导致行业普遍裁员，但圣地亚哥仍然是新药研发重镇。 2025年，美国 FDA 药品评价与研究中心（CDER）批准了 46 种此前从未在美国获批或上市的新药，即所谓的创新药物。尽管较2024年的 50 种和2023年的 55 种新药获批数略有下降，但仍延续了高获批数量的趋势。2025年获批的药物包括 34 种新分子实体（NME）和 12 种生物制剂，其中肿瘤（占获批药物总数的35%）和罕见病领域尤为突出。小分子药物仍然占据主导地位（67%），许多小分子药物具有全新的作用机制。主要审批趋势：肿瘤领域领先地位：精准肿瘤治疗，尤其是在非小细胞肺癌 (NSCLC) 领域，是重点关注方向。罕见病领域推进：FDA 批准了 23 种疗法（占总数的 50%）获得孤儿药资格认定，用于治疗罕见且特殊的疾病。生物制剂和先进疗法：生物制剂占获批药物的 26%，其中包括 7 种单克隆抗体 (mAb)，2 种抗体药物偶联物 (ADC)，以及 1 种双特异性抗体。 TIDES药物创新：4 种创新TIDES药物（寡核苷酸和肽）获得批准，占获批药物总数的 9%。审批速度：尽管内部人员流动以及政府停摆对各机构造成影响，FDA 仍保持了高效及时的审批速度，70% 的新药快于其他国家获批上市。 2025年美国新药审批监管分类： 21种（46%）新获批的疗法获得了优先审评资格，这是 FDA 授予那些预期疗效优于现有标准疗法的药物的监管认定。这一比例低于过去五年平均水平57%。获得优先审评资格的药物通常在提交申请后6个月内完成审评，而获得标准审评资格的药物则需要10个月。 23种（50%）新获批的药物获得了孤儿药资格认定，该资格适用于美国境内患病人数少于20万的罕见病。这些药物的申请、资助单位可获得各种激励措施，包括部分临床试验的税收抵免、免除用户费用以及潜在的市场独占权。 15种（33%）新获批的药物获得了突破性疗法资格认定，该资格适用于那些疗效优于现有疗法的药物。这些药物的申请、资助单位可获得与 FDA 更频繁的会面机会，以及在整个药物研发过程中更深入的监管指导。 11种（24%）新药获得了加速审批，其依据是替代终点指标的改善，FDA 认为这些指标“极有可能”预测临床获益。加速审批缩短了新药的整体研发周期。药企需要开展验证性试验，以提供实际临床获益的证据。 2025年美国新药列表: Drug (brand name) Sponsor Properties Indication Datopotamab deruxtecan (Datroway) Daiichi Sankyo TROP2-targeted ADC; topoisomerase inhibitor payload HR-positive, HER2-negative breast cancer Treosulfan (Grafapex) Medexus DNA alkylating drug Preparative regimen for haematopoietic stem cell transplantation for cancers Suzetrigine (Journavx) Vertex NaV1.8 inhibitor Acute pain Mirdametinib (Gomekli) SpringWorks/Merck KGaA MEK1/2 kinase inhibitor Neurofibromatosis type 1 Vimseltinib (Romvimza) Deciphera/Ono CSF1R kinase inhibitor Tenosynovial giant cell tumours Gepotidacin (Blujepa) GSK Triazaacenaphthylene bacterial type II topoisomerase inhibitor Uncomplicated urinary tract infections Fitusiran (Qfitlia) Genzyme/Sanofi Antithrombin-targeted siRNA Bleeding episodes in haemophilia A or B Atrasentan (Vanrafia) Novartis Endothelin receptor antagonist Proteinuria in adults with primary IgAN Penpulimab (Penpulimab) Akeso Biopharma PD1-targeted mAb Nasopharyngeal carcinoma Nipocalimab (Imaavy) Janssen/J&J Neonatal Fc receptor-targeted mAb Generalized myasthenia gravis Avutometinib plus defactinib (Avmapki Fakzynja Co-Pack) Verastem MEK1 inhibitor plus FAK kinase inhibitor KRAS-mutated recurrent low-grade serous ovarian cancer Telisotuzumab vedotin (Emrelis) AbbVie c-Met-targeted ADC; microtubule inhibitor payload Non-squamous NSCLC with high c-Met protein overexpression Acoltremon (Tryptyr) Alcon Labs TRPM8 thermoreceptor agonist Dry eye disease Clesrovimab (Enflonsia) Merck & Co. RSV fusion protein-targeted mAb RSV lower respiratory tract disease prevention in neonates and infants Taletrectinib (Ibtrozi) Nuvation ROS1 kinase inhibitor ROS1-positive NSCLC Garadacimab (Andembry) CSL Behring Activated factor XII-targeted mAb Hereditary angioedema prevention Linvoseltamab (Lynozyfic) Regeneron BCMA × CD3 bispecific T cell engager Multiple myeloma Sunvozertinib (Zegfrovy) Dizal Jiangsu EGFR kinase inhibitor NSCLC with EGFR exon 20 mutations Sebetralstat (Ekterly) KalVista Plasma kallikrein inhibitor Hereditary angioedema attacks Delgocitinib (Anzupgo) Leo Pharma Janus kinase inhibitor Hand eczema Sepiapterin (Sephience) PTC Phenylalanine hydroxylase activator Hyperphenylalaninemia Aceclidine (Vizz) Lenz Cholinergic agonist Presbyopia Dordaviprone (Modeyso) Chimerix/Jazz ClpP protease activator Diffuse midline glioma with H3 K27M mutations Zongertinib (Hernexeos) Boehringer Ingelheim HER2 kinase inhibitor Non-squamous NSCLC with HER2 activating mutations Brensocatib (Brinsupri) Insmed DPP1 inhibitor Non-cystic fibrosis bronchiectasis Donidalorsen (Dawnzera) Ionis Prekallikrein-targeted ASO Hereditary angioedema prevention Rilzabrutinib (Wayrilz) Genzyme/Sanofi BTK kinase inhibitor Immune thrombocytopenia Pembrolizumab plus berahyaluronidase alfa (Keytruda Qlex)a Merck & Co. PD1-targeted mAb and an endoglycosidase to enable subcutaneous delivery Solid tumours approved for the intravenous formulation of pembrolizumab Elamipretide (Forzinity) Stealth Mitochondrial cardiolipin binder Muscle strength in Barth syndrome Imlunestrant (Inluriyo) Eli Lilly Selective oestrogen receptor degrader ER-positive, EGFR2-negative, ER1-mutated breast cancer Paltusotine (Palsonify) Crinetics Somatostatin receptor agonist Acromegaly Remibrutinib (Rhapsido) Novartis BTK kinase inhibitor Spontaneous urticaria Nerandomilast (Jascayd) Boehringer Ingelheim PDE4 inhibitor Idiopathic pulmonary fibrosis Elinzanetant (Lynkuet) Bayer Healthcare NK1 and NK3 receptor antagonist Vasomotor symptoms due to menopause Doxecitine plus doxribtimine (Kygevvi) UCB Pyrimidine nucleosides Thymidine kinase 2 deficiency Ziftomenib (Komzifti) Kura Menin inhibitor AML with a susceptible NPM1 mutation Plozasiran (Redemplo) Arrowhead ApoC-III-targeted siRNA Triglycerides in familial chylomicronemia syndrome Sevabertinib (Hyrnuo) Bayer Healthcare HER2 kinase inhibitor Non-squamous NSCLC with HER2 activating mutations Sibeprenlimab (Voyxact) Otsuka APRIL-targeted mAb Proteinuria in primary IgAN Lerodalcibep (Lerochol) LIB Therapeutics PCSK9-targeted adnectin LDL-C-lowering in hypercholesterolaemia Etripamil (Cardamyst) Milestone Calcium channel blocker Paroxysmal supraventricular tachycardia Zoliflodacin (Nuzolvence) Entasis/Innoviva Spiropyrimidinetrione bacterial type II topoisomerase inhibitor Urogenital gonorrhea due to Neisseria gonorrhoeae Depemokimab (Exdensur) GSK IL-5-targeted mAb Severe asthma, eosinophilic phenotype Aficamten (Myqorzo) Cytokinetics Cardiac myosin inhibitor Obstructive hypertrophic cardiomyopathy Narsoplimab (Yartemlea) Omeros MASP2-directed mAb Haematopoietic stem cell transplant-associated thrombotic microangiopathy Tradipitant (Nereus) Vanda NK1 receptor antagonist Vomiting induced by motion 主要参考资料： 1) Nature Reviews Drug Discovery 25, 163 (2026) 2) Nature Reviews Drug Discovery 25, 81-87 (2026) 3) fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025 4) AACR.org

2026-04-18

·罕见病

从“这不能吃”到“终于能吃了”—PKU治疗新进展，给家庭的新希望

每一个小进步，都是向希望迈出的一步有一种苦，不是尝不到糖，而是明明就在嘴边，却不得不咽下口水说“不要”。这就是很多PKU家庭的日常。但我想告诉你的是——2026年，情况正在您您变化。先说个简单的比喻。你的身体里住着一群“维修工”，专门负责把一种叫苯丙氛歖的东西转化成另一种有用的物质。但PKU孩子的身体里，这些维修工要么数量不够，要么干不了活。结果呢？苯丙氛歖越堆越多，跑到了血液里，跑到了脑子里，开始搞破坏。所以，从出生那一刻起，这些孩子就要开始一场漫长的“饮食保卫战”——每一顿饭，都是一道计算题。以前怎么办？两条路。第一条：严格忌口。肉、蛋、奶、豆制品……这些普通孩子习以为常的美食，对PKU孩子来说全是禁区。第二条：喝特殊的配方粉。一喝就是一辈子，很多孩子直到长大成人，都没怎么尝过正常食物的味道。难吗？太难了。更难的是，随着孩子长大，社交场合越来越多——生日派对、学校餐厅、亲戚聚餐……每次看着别人吃，自己只能咽下口水，那种感觉，没经历过的人真的不懂。重点来了——2025年7月，美国FDA批准了一款新药，叫Sephience（墨蝶蛳）。这药牛在哪儿？它让很多PKU患者真的实现了“饮食自由”。看这组数据：平均血苯丙氛歖水平下降了63% 97%的患者增加了天然蛋白进食部分患者甚至可以吃到和普通人一样的蛋白质量更让人挥苻的是——之前对另一种药Kuvan没反应的患者里，有43%对这款新药有效。这意味着更多家庭看到了希望。每天口服一次，随餐服用，适用年龄从1个月起。目前美国、欧盟、日本都已批准，中国的审批也在推进中。新药研发给PKU患者带来了更多选择但这还不是全部。如果你家孩子的血苯丙氛歖一直控制不好，还有一个选择——Palynziq（培伐利酶）。这是全球唯一批准的PKU酶替代疗法。通俗点说，就是直接往身体里补充缺少的那种“维修工”，帮你把苯丙氛歖分解掉。 2026年2月，FDA把它适用年龄从16岁以上扩展到了12岁以上。数据说话：治疗72周后，血苯丙氛歖平均下降473 μmol/L 近半数患者血值降低超过50% 很多患者的反馈是：“终于能和家人一起吃饭了” 但要注意：这款药有过敏风险，需要在医生指导下使用，患者需要随身带着肺上腾素应急。治疗方案一定要和主治医生充分沟通。眼光放远一点——基因治疗正在路上。 PKU的“病根”是PAH基因出了问题。如果能把这个问题基因修好，是不是就能从根上解决问题？科学家们正在探索几条路： AAV基因治疗用特殊载体把正常的基因送进肝细胞，让它开始工作。目前有候选药物在临床试验阶段。基因编辑用CRISPR技术直接修正错误的基因序列。宾汉法尼亚大学的研究已经在小鼠实验中成功把血苯丙氛歖水平正常化。 mRNA替代疗法把“生产维修工的说明书”送进细胞里，让细胞自己造出需要的酶。这个方法还有个好处——可以重复给药。这些研究还在路上，距离真正用到人身上还需要时间。但方向已经明确，未来可期。还有一个好消息——还有新药在临床试验中。 JNT-517是一种口服小分子药，原理是阻断肾脏对苯丙氛歖的“回收”，让它多排出体外。II期临床试验显示，用药后血苯丙氛歖下降60%，目前已经进入III期临床。医学进步让每一个家庭都看到希望说给PKU家庭的话看完这些，你可能会想：我家孩子什么时候能用上？好消息是：治疗选择真的在变多。从单纯的饮食控制，到药物治疗，再到基因治疗的探索，每一步都在往前走。但也要理性看待：新药需要时间进入中国市场每种治疗都有适用人群和注意事项个体差异很大，需要在医生指导下选择最重要的事：定期监测血苯丙氛歖水平，坚持小诺，关注新药进展。这些听起来是老生常谈，但真的非常重要。饮食控制确实很难，你不是一个人在战斗。这条路不容易，但每一步都算数。我们一起，向前走。参考资料 [1] FDA批准Sephience (sepiapterin)用于PKU. 2025年7月 [2] FDA批准Palynziq扩龄至12岁以上. 2026年2月 [3] JNT-517 III期临床试验进展. 2025年12月 [4]宾汉法尼亚大学PKU基因编辑研究. 2023年 [5] Orphanet Journal of Rare Diseases. PKU治疗现状. 2025年免费声明：本文仅供参考，不能替代专业医疗建议。如有疑问，请咨询主治医生。

2026-04-16

·药研苑

先通医药前列腺检测用氟[18F]妥司特注射液申请上市

药品营销避坑”试读：药品导入期管理视频课（试听）药品生命周期管理（案例）药品立项需要注意什么（案例） 2024年4月15日先通医药宣布，前列腺癌诊断的放射性药物氟[18F]妥司特注射液（XTR020）新药上市申请（NDA）已获国家药品监督管理局（NMPA）受理。氟[18F]妥司特（Flotufolastat F 18）为氟[18F标记的前列腺特异性膜抗原（PSMA）配体，作为PET示踪剂能够与前列腺癌细胞PSMA结合，并内化进入细胞，实现诊断前列腺癌的原发性和转移性病灶。18F标记放射性药物的放射性半衰期为109.7分钟，比68Ga标记剂放射性药（放射性半衰期：67.7分钟），在物流和分销方面更具有优势。氟[18F]妥司特（Posluma，XTR020）由Blue Earth Diagnostics开发，2023年基于项3期临床试验LIGHTHOUSE的研究结果，获得FDA批准用于前列腺癌患者前列腺特异性膜抗原（PSMA）阳性病灶的正电子发射断层扫描（PET）检查。 LIGHTHOUSE研究为多中心、开放标签、单臂3期临床试验，共纳入352例新诊断前列腺癌患者，在接受氟[18F]妥司特PET/CT后接受外科手术治疗。接受氟[18F]妥司特PET/CT独立阅片显示，7.8-13%的患者为盆腔淋巴结（PLN）阳性；组织学检查显示24%的患者为PLN阳性。氟[18F]妥司特PET/CT未达到预设的敏感性阈值。氟[18F]妥司特PET/CT三名独立阅片者的特异性分别93%、94%和97%，高于预测的特异性阈值。氟[18F]妥司特PET/CT检查高危/极高危前列腺癌的敏感性为24-33%，高于中危前列腺癌（16-21%）。在安全性方面，未观察到严重不良事件。352例患者中有61例患者，氟[18F]妥司特PET/CT检测显示存在盆腔外转移（M1期）。与其他影像学检查或组织检查的匹配度为56%（Devaki Shilpa Surasi., 2023）。推荐指数：★★★ 推荐原因：在前列腺癌诊断中，氟[18F]妥司特具有积极的意义，能够为临床分期和治疗决策提供了更可靠的依据。相关阅读：拜耳HER2酪氨酸激酶抑制剂塞伐艾替尼国内上市一品制药申请上市Lambert-Eaton综合征治疗药物二氨基吡啶百济神州HER2双特异性抗体泽尼达妥申请用于胃食管腺癌泽璟制药吉卡昔替尼中重度特应性皮炎3期临床达到主要终点百济神州T细胞衔接器抗体塔拉妥获批治疗广泛期小细胞肺癌首家申请上市，四环科宝艾地骨化醇片上市申请获得受理罗氏申请奥妥珠单抗新增自身免疫性相关肾病适应症第二个CTLA-4单抗，阿斯利康曲麦利尤单抗获批转移性NSCLC 首仿，迪赛诺抗HIV药物拉米夫定多替拉韦片获批首仿，苏州盛达药业盐酸头孢卡品酯颗粒获批上市独家，海融度骨化醇注射液获批治疗继发性甲状旁腺功能亢进荣昌生物HER2 ADC维迪西妥单抗获批一线治疗晚期尿路上皮癌独家儿童抗过敏药物，四环科宝盐酸奥洛他定口服溶液获得映恩生物HER2 ADC帕康曲妥珠单抗申请国内新药上市高苯丙氨酸血症治疗药物塞匹蝶呤已在国内申请上市相关内容只作为药物研发的参信息。不构成任何与用药咨询有关的建议。如需发稿，您可通过在药研苑公众号下以发送信息的方式将官网或官方公众号的稿件链接和标题发送给我们。我们将择优确定是否发布。

100 项与塞匹蝶呤相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	塞匹蝶呤	-	DB16326

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
苯丙酮尿症	欧盟	PTC Therapeutics, Inc.	2025-06-24
苯丙酮尿症	冰岛	PTC Therapeutics, Inc.	2025-06-24
苯丙酮尿症	列支敦士登	PTC Therapeutics, Inc.	2025-06-24
苯丙酮尿症	挪威	PTC Therapeutics, Inc.	2025-06-24
高苯丙氨酸血症	欧盟	PTC Therapeutics International Ltd.	2025-06-19
高苯丙氨酸血症	冰岛	PTC Therapeutics International Ltd.	2025-06-19
高苯丙氨酸血症	列支敦士登	PTC Therapeutics International Ltd.	2025-06-19
高苯丙氨酸血症	挪威	PTC Therapeutics International Ltd.	2025-06-19

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
糖尿病性胃轻瘫	临床2期	美国	PTC Therapeutics, Inc.	2019-02-27
胃轻瘫	临床2期	美国	PTC Therapeutics, Inc.	2019-02-27
四氢生物蝶呤缺乏症	临床2期	美国	PTC Therapeutics, Inc.	2019-01-03

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临床结果

适应症

分期

评价

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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


NCT05099640 (FDA_CDER) 人工标引	临床3期	高苯丙氨酸血症 \| 苯丙酮尿症	98	SEPHIENCE	網鹹窪淵簾憲簾鹽範積(壓願願範築夢窪衊選顧) = 願繭積構齋鹹艱鏇獵獵繭積簾餘夢願選網鏇齋 (製襯繭廠遞壓憲壓壓製, 24.1) 更多	积极	2025-07-28
				Placebo	網鹹窪淵簾憲簾鹽範積(壓願願範築夢窪衊選顧) = 鹽製壓糧遞窪淵廠膚範繭積簾餘夢願選網鏇齋 (製襯繭廠遞壓憲壓壓製, 24.2) 更多
NCT05099640 (APHENITY, Literature) 人工标引	临床3期	苯丙酮尿症	157	Placebo (part 2)	獵願夢膚憲憲齋製鬱糧(窪顧繭餘壓廠顧築網構) = 觸積選鹹醖製壓簾觸簾餘網築網壓鹹製簾襯廠 (顧壓築淵窪製鹹構繭蓋, 29)	积极	2024-10-05
				Placebo	繭鑰積餘鹹願製醖觸繭(衊廠糧襯繭壓積膚繭襯) = 鏇醖糧壓鬱製網糧糧餘網餘醖觸衊糧觸選觸糧 (積齋顧膚廠顧網鏇選衊 ) 更多
NCT05099640 (APHENITY, CTgov)	临床3期	苯丙酮尿症	157	Placebo	醖淵蓋簾範襯鏇鑰淵夢(鑰獵膚鹽網簾艱廠選觸) = 範憲構夢蓋繭簾鏇製鏇壓構鏇鹹鑰顧鬱獵糧窪 (淵襯鑰憲鬱鑰蓋鑰鹽鬱, 24.223) 更多	-	2024-01-10
NCT03519711 (CTgov)	临床1/2期	四氢生物蝶呤缺乏症 \| 高苯丙氨酸血症	8	PTC923 (Cohort 1: PTC923 2.5 mg/kg/Day)	鹽膚鏇簾獵艱鹽積積願 = 觸醖範鏇積願簾糧遞網構蓋鬱壓築膚遞衊膚廠 (淵艱繭顧膚觸繭鑰積製, 鬱蓋窪鏇衊憲糧網憲製 ~ 製糧製壓獵觸糧鏇衊衊) 更多	-	2023-11-14
				PTC923 (Cohort 1: PTC923 10 mg/kg/Day)	鹽膚鏇簾獵艱鹽積積願 = 夢鏇鏇選醖艱構襯廠鑰構蓋鬱壓築膚遞衊膚廠 (淵艱繭顧膚觸繭鑰積製, 簾衊繭窪膚鏇齋鬱鹽糧 ~ 鬱壓繭顧膚廠積夢膚餘) 更多
NCT03712124 (CTgov)	临床2期	胃轻瘫 \| 糖尿病性胃轻瘫	21	CNSA-001 (CNSA-001)	觸膚鹽願網簾積廠衊醖(願鬱膚積襯鹹鬱繭鏇壓) = 糧淵齋鑰繭壓夢壓廠壓獵窪窪膚齋遞夢築蓋築 (積膚淵廠獵醖構鹽鏇築, 124.52) 更多	-	2022-01-05
				Placebo (Placebo)	觸膚鹽願網簾積廠衊醖(願鬱膚積襯鹹鬱繭鏇壓) = 壓糧齋衊餘鏇糧膚鏇壓獵窪窪膚齋遞夢築蓋築 (積膚淵廠獵醖構鹽鏇築, 99.06) 更多
NCT03712124 (Pubmed) 人工标引	临床2期	糖尿病性胃轻瘫	-	PTC923	壓製壓網獵艱構顧築簾(餘積築獵壓網觸網糧網): difference = 98 (95% CI, 36 ~ 161), P-Value = 0.0042	积极	2021-09-01
				Placebo