A Phase 2, Open-label, Fixed-dose Study to Assess the Efficacy of Sodium Phenylbutyrate (ACER-001) in Treating Pediatric and Adult Patients With Medium Chain Acyl-CoA Dehydrogenase (MCAD) Deficiency Resulting From the Prevalent ACADM c.985 A>G (K304E) Mutation

This is a medical research study to test a medication in patients 4 years of age and older with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD) caused by the common ACADM c.985 A>G (K304E) mutation. The medication is sodium phenylbutyrate (ACER-001), which is currently FDA approved for the treatment of Urea Cyle Disorders. Previous research suggests that sodium phenylbutyrate may also be effective in the treatment MCADD. This study will investigate the safety and efficacy (how well it works) of sodium phenylbutyrate in patients with MCADD.

开始日期2025-02-01

申办/合作机构

University of Pittsburgh

[+1]

NCT06645769

/ Not yet recruiting早期临床1期IIT

Na-Phenylbutyrate VAscular Trial

This study will investigate if the drug sodium phenylbutyrate (NaPB) impacts blood pressure and vascular function in healthy volunteers and in patients with diabetes.

开始日期2025-01-01

申办/合作机构

University of Pennsylvania

[+1]

ChiCTR2400086139

/ Suspended早期临床1期IIT

A clinical effectiveness study of Sodium phenylbutyrate in the treatment of polycystic ovary syndrome

开始日期2024-07-01

申办/合作机构

复旦大学附属中山医院

100 项与苯基丁酸钠相关的临床结果

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100 项与苯基丁酸钠相关的转化医学

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100 项与苯基丁酸钠相关的专利（医药）

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325

项与苯基丁酸钠相关的文献（医药）

2025-04-01·Neural Regeneration Research

Treating amyotrophic lateral sclerosis with allogeneic Schwann cell–derived exosomal vesicles: a case report

Article

作者: Pearse, Damien D. ; Dietrich, W. Dalton ; Levi, Allan D. ; Guest, James D. ; Silvera, Risset ; Goldschmidt, Alexander J.P. ; Brooks, Adriana ; Graham, Patricia ; Goldschmidt-Clermont, Pascal J. ; Jimsheleishvili, George ; Khan, Aisha

Schwann cells are essential for the maintenance and function of motor neurons, axonal networks, and the neuromuscular junction. In amyotrophic lateral sclerosis, where motor neuron function is progressively lost, Schwann cell function may also be impaired. Recently, important signaling and potential trophic activities of Schwann cell-derived exosomal vesicles have been reported. This case report describes the treatment of a patient with advanced amyotrophic lateral sclerosis using serial intravenous infusions of allogeneic Schwann cell-derived exosomal vesicles, marking, to our knowledge, the first instance of such treatment. An 81-year-old male patient presented with a 1.5-year history of rapidly progressive amyotrophic lateral sclerosis. After initial diagnosis, the patient underwent a combination of generic riluzole, sodium phenylbutyrate for the treatment of amyotrophic lateral sclerosis, and taurursodiol. The patient volunteered to participate in an FDA-approved single-patient expanded access treatment and received weekly intravenous infusions of allogeneic Schwann cell-derived exosomal vesicles to potentially restore impaired Schwann cell and motor neuron function. We confirmed that cultured Schwann cells obtained from the amyotrophic lateral sclerosis patient via sural nerve biopsy appeared impaired (senescent) and that exposure of the patient’s Schwann cells to allogeneic Schwann cell-derived exosomal vesicles, cultured expanded from a cadaver donor improved their growth capacity in vitro. After a period of observation lasting 10 weeks, during which amyotrophic lateral sclerosis Functional Rating Scale-Revised and pulmonary function were regularly monitored, the patient received weekly consecutive infusions of 1.54 × 1012(×2), and then consecutive infusions of 7.5 × 1012(×6) allogeneic Schwann cell-derived exosomal vesicles diluted in 40 mL of Dulbecco’s phosphate-buffered saline. None of the infusions were associated with adverse events such as infusion reactions (allergic or otherwise) or changes in vital signs. Clinical lab serum neurofilament and cytokine levels measured prior to each infusion varied somewhat without a clear trend. A more sensitive in-house assay suggested possible inflammasome activation during the disease course. A trend for clinical stabilization was observed during the infusion period. Our study provides a novel approach to address impaired Schwann cells and possibly motor neuron function in patients with amyotrophic lateral sclerosis using allogeneic Schwann cell-derived exosomal vesicles. Initial findings suggest that this approach is safe.

2024-12-01·AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Missense variant in PIGM associated with severe cystic encephalomalacia and portal vein thrombosis: Phenotypic and genotypic expansion of the glycosylphosphatidylinositol biosynthesis defect‐1 (GPIBD1)

Article

作者: Yadav, Rashmi ; Edmondson, Andrew C. ; Tarnopolsky, Mark ; Brady, Lauren

Abstract:

Glycosylphosphatidylinositols (GPIs) are a type of glycolipid responsible for anchoring many important proteins to the cell membrane surface. Defects in the synthesis of GPIs can lead to a group of multisystem disorders known as the inherited GPI deficiencies (IGDs). Homozygosity for the c.‐270C > G variant in the promoter of PIGM has been associated with a IGD subtype known as glycosylphosphatidylinositol biosynthesis defect‐1 (GPIBD1). The several cases reported in the literature have been described to have a milder neurologic phenotype in comparison to the other IGDs and have been treated with sodium phenylbutyrate with some degree of success. These patients typically present with portal and hepatic vein thrombosis and mostly develop absence seizures. Here we describe a patient homozygous for a nonsynonymous variant in PIGM who deceased at 9 weeks of life and had multiple physical dysmorphisms (rocker bottom feet, midline cleft palate, thickened and lichenified skin), portal vein thrombosis, CNS structural anomalies (progressive multicystic encephalomalacia and ventriculomegaly), and a neurological phenotype of a diffuse encephalopathy. This is the first known case report of a PIGM‐related IGD/CDG due to a coding variant.

2024-11-01·JOURNAL OF APPLIED TOXICOLOGY

Hepatic enzyme induction and its potential effect on thyroid hormone metabolism in the metamorphosing tadpole of Xenopus laevis (African clawed frog)

Article

作者: Fujisawa, Takuo ; Tanaka, Hitoshi ; Wada, Kohei ; Yamaguchi, Takafumi

Abstract:

Hepatic enzyme induction, an inherent defense system against xenobiotics, is known to simultaneously affect endocrine system functions in mammals under specific conditions, particularly thyroid hormone (TH) regulation. While this phenomenon has been studied extensively, the pathway leading to this indirect thyroid effect in mammals has unclear applicability to amphibians, despite the importance of amphibian species in assessing thyroid‐disruptive chemicals. Here, we investigated the effects of three well‐known mammalian enzyme inducers—β‐naphthoflavone (BNF), pregnenolone carbonitrile (PCN), and sodium phenobarbital (NaPB)—on the gene expression of phase‐I and phase‐II metabolizing enzymes in Xenopus laevis tadpoles. Waterborne exposure to BNF and PCN significantly induced the expression of both phase‐I (cytochrome P450, CYP) and phase‐II enzymes (UDP‐glucuronosyltransferase, UGT and sulfotransferase, SULT), but in different patterns, while NaPB exposure induced CYP2B expression without affecting phase‐II enzymes in tadpoles, in contrast to mammals. Furthermore, an ex vivo hepatic enzyme activity assay confirmed that BNF treatment significantly increased phase‐II metabolic activity (glucuronidation and sulfation) toward TH. These results suggest the potential for certain mammalian enzyme inducers to influence TH clearance in X. laevis tadpoles. Our findings provide insights into the profiles of xenosensing activity and enzyme induction in amphibians, which can facilitate a better understanding of the mechanisms of indirect effects on the thyroid system via hepatic enzyme induction in nonmammalian species.

323

项与苯基丁酸钠相关的新闻（医药）

2024-12-29

·药渡

盘点：2024年值得关注的5项失败的临床试验

来源：药渡撰文：幻目编辑：维他命药物开发向来都是具有挑战性的。由于各种原因（包括候选药物本身、剂量和不良事件），大约90%的研究性药物都会失败。中枢神经系统（CNS）疾病的药物的II期和III期失败率约为85%。一项研究发现，在1995年至2007年期间，CNS药物获得FDA批准的比率不到非CNS药物的一半。在过去的一年里，我们再次看到神经科学领域太多令人失望的中后期临床试验结果，如肌萎缩侧索硬化症（ALS）、亨廷顿舞蹈症和精神分裂症。这反映了药物开发尤其是在推进精神疾病和神经退行性疾病的新疗法开发，仍然是生物技术和制药行业最具挑战性的领域之一。 BioSpace盘点了2024年五项失败的临床试验，我们或许可以从中受到一些启发。 01 Emraclidine 研发企业：艾伯维适应症：精神分裂症 2023年12月，艾伯维向Cerevel Therapeutics押注87亿美元，对这家生物技术公司的主导产品Emraclidine寄予厚望。内容回顾：艾伯维年终大手笔：200亿美元两笔并购，将掀起2024药企并购潮？ Emraclidine是一种毒蕈碱M4受体的正变构调节剂（PAM），是一种潜在“best-in-class”的下一代抗精神病药物，可用于精神分裂症的治疗。在一项1b期研究中，Emraclidine在精神分裂症患者治疗中显示出良好的疗效和安全性。今年9月，FDA批准Cobenfy作为35年来第一个治疗精神分裂症的新类别药物。（内容回顾：35年来首款！FDA批准BMS新型精神分裂症药物，5款新药紧随其后……)与Emraclidine一样，Cobenfy靶向毒蕈碱受体——这一事实可能预示着Emraclidine会有比较好的结果。然而，2024年11月，Emraclidine在两项II期试验中未能显著改善精神分裂症症状。在EMPOWER-1和EMPOWER-2中，与安慰剂相比，Emraclidine在第6周时未达到阳性和阴性综合征量表基线变化的主要终点。在EMPOWER-2试验中，较低剂量导致症状恶化。随着Emraclidine的失败，Cobenfy成为唯一的毒蕈碱赢家。 02 Dalzanemdor 研发企业：Sage Therapeutics 适应症：亨廷顿病、阿尔茨海默病和帕金森病 2024年对Sage Therapeutics来说并不是好年头。这家总部位于马萨诸塞州剑桥的生物技术公司的股票在过去18个月中暴跌了90%以上，这在很大程度上是由于主要项目Dalzanemdor的临床试验失败。 Dalzanemdor是N-甲基-D-天冬氨酸受体（NMDA）阳性变构调节剂，可用于研究亨廷顿舞蹈病、阿尔茨海默病和认知功能障碍。 2024年4月，Sage Therapeutics宣布Dalzanemdor未达到II期PRECEDENT研究的主要终点，与安慰剂相比，帕金森病轻度认知障碍患者并不存在统计学上的显著差异。 10月，该候选药物在阿尔茨海默病的II期LIGHTWAVE研究中也未成功，与安慰剂相比未能显著提高智力。此外，Dalzanemdor在亨廷顿病的II期DIMENSION试验中，再次未能达到主要终点。接连的失败后，Sage宣布将停止该药物的开发。 03 莫哌达基研发企业：辉瑞适应症：杜氏肌营养不良症 Sarepta的Elevidys于2023年6月获得批准后，人们普遍认为fordadistrogene movaparvovec（莫哌达基）是神经肌肉疾病的下一个基因疗法。 Fordadistrogene movaparvovec是一种静脉注射基因疗法。它将由人类肌肉特异性启动子控制的“迷你”抗肌萎缩蛋白（mini-dystrophin）转基因装在腺相关病毒9（AAV9）载体中，用于治疗杜氏肌营养不良症（DMD）。 1b期临床试验结果显示，治疗12个月后，患者显示出持久和具有统计学意义的显著改善，包括微型抗肌萎缩蛋白表达的持续水平，以及NorthStar门诊评估量表（NSAA）得分的改善。然而，今年6月，辉瑞（Pfizer）的fordadistrogene movaparvovec在III期CIFFREO试验中未能显著改善4至7岁男孩的运动功能。此外，fordadistrogene movaparvovec临床试验进程也是一波三折。2021年12月，在一名患者意外死亡后，辉瑞被迫暂停Ib期患者的筛选和给药。2024年5月，2期DAYLIGHT研究中的一名参与者突然死亡，辉瑞暂停了与3期CIFFREO试验交叉部分相关的给药。辉瑞表示，尽管没有显示出明显的疗效信号，但fordadistrogene movaparvovec的安全性可控，大多数不良事件的严重程度为轻度至中度。 04 Relyvrio 研发企业：Amylyx 适应症：肌萎缩侧索硬化症 Relyvrio是两种药物苯丁酸钠和牛磺酸二醇的复方制剂。它们可以改善细胞内线粒体和内质网的健康状态，从而延缓神经细胞的死亡。Relvyrio于2022年9月获得批准，成为有史以来第三种治疗肌萎缩侧索硬化症的药物。 FDA根据II期CENTAUR试验的结果批准了Relvyrio，该药物（苯丁酸钠和牛磺酸二醇的组合）被证明可以减缓ALS的进展并将患者的寿命延长数月。然而，在PHOENIXIII期试验，与安慰剂相比，第48周时ALSFRS-R总分相对于基线的变化没有显著差异。在Relyvrio获得批准之前，Amylyx联合首席执行官JustinKlee和JoshCohen承诺，如果III期数据不乐观，他们将从市场上撤下该药物。在PHOENIXIII期试验失败后，Relyvrio于今年4月从美国和加拿大市场撤出。 05 Trodelvy 研发企业：Gilead Sciences 适应症：非小细胞肺癌 Trodelvy是吉利德开发的一款靶向TROP2的抗体-药物偶联物（ADC），于2020年4月首次在美国获批上市，该药物也因此成为了全球首款获批的TROP2 ADC。截至目前，已获批3项适应症： 1）已接受过至少两种系统治疗的转移性三阴性乳腺癌（mTNBC）成年患者； 2）既往接受过含铂化疗和PD-(L)1抑制剂治疗的局部晚期或转移性尿路上皮癌（mUC）成年患者； 3）接受过内分泌治疗和至少两种系统治疗的不可切除的局部晚期或转移性HR+/HER2-（IHC0、IHC1+或IHC2+/ISH–）乳腺癌成年患者。今年1月，吉利德宣布Trodelvy在III期EVOKE-01研究中未达到总生存期这一主要终点。EVOKE-01研究是一项全球性、多中心、开放标签III期临床试验（N=603），评估了Trodelvy对比多西他赛治疗接受铂类化疗和检查点抑制剂治疗期间或之后进展的转移性或晚期NSCLC患者的疗效和安全性。结果显示，相比于多西他赛组，Trodelvy组患者的OS有所延长，虽然二者并无显著性差异。不过，在接受过PD-(L)1抑制剂治疗但无应答的亚组患者中，Trodelvy组患者的OS显著延长了3个月以上；而这种差异不存在于对PD-(L)1抑制剂治疗应答的亚组患者中。鉴于此，吉利德决定继续探索Trodelvy的目标人群。除了EVOKE-01研究以外，吉利德还开展了EVOKE-03研究，以评估Trodelvy+帕博利珠单抗对比帕博利珠单抗一线治疗PD-L1 TPS≥50%的转移性NSCLC患者的有效性和安全性。参考资料： https://www.biospace.com/drug-development/5-clinical-assets-that-flopped-in-2024 *声明：本文仅是介绍医药疾病领域研究进展或简述研究概况或分享医药相关讯息，并非也不会进行治疗或诊断方案推荐，也不对相关投资构成任何建议。内容如有疏漏，欢迎沟通指出！ 2024年最值得关注的糖尿病药物TOP10 国内CXO无投资价值？CXO行业五条破局之道大盘点 7亿美元！济民可信抗IgE单抗成功出海，授权于RAPT

临床2期临床结果引进/卖出临床3期

2024-12-27

·immedica.com

Application to the Ministry of Health in Japan for Ravicti® (Glycerol Phenylbutyrate) submitted

Stockholm, December 27, 2024 – Immedica is pleased to announce that its partner OrphanPacific, Inc has submitted an application to the Ministry of Health, Labour and Welfare in Japan for the approval of Ravicti® (glycerol phenylbutyrate) for the treatment of Urea Cycle Disorders (UCD). The submission follows the approval of the orphan drug designation of Ravicti on December 25. "This submission marks a significant step toward bringing this important treatment addition to patients with UCD in Japan. We are pleased to see this progress together with our partner OrphanPacific, as we continue our commitment to addressing unmet medical needs in rare diseases” says Anders Edvell, CEO of Immedica. An orphan drug designation is handled by the Ministry of Health, Labour and Welfare based on Article 77-2 of the Pharmaceuticals and Medical Devices Act. The designation is given to products that meet conditions such as having fewer than 50,000 patients in Japan and being particularly necessary from a medical standpoint, following an opinion of the Pharmaceutical Affairs and Food Sanitation Council. Orphan Pacific has been a valued partner to Immedica in Japan, holding exclusive rights to Buphenyl® (sodium phenylbutyrate). This collaboration was further strengthened in May 2022 through an exclusive license agreement, adding Ravicti to their portfolio for the Japanese market. About Urea Cycle Disorders (UCD) Urea cycle disorders are a group of metabolic diseases that affect a specific enzyme or transporter in the urea cycle leading to elevated ammonia or glutamine levels in the circulation. Symptoms of the disorder can begin at any age, where more severe defects are seen with an onset of the disease early in life. UCD patients may experience episodes, called hyperammonemic crises, when ammonia levels in the blood become excessively high, which can result in irreversible brain damage, coma, or death. Beyond hyperammonemic crises there are also more subtle symptoms including vomiting, refusal to feed, irritability, muscular hypotonia as well as delayed motor and psychointellectual development. As a group, these disorders occur in 1 in 35,000 newborns. About Ravicti® (glycerol phenylbutyrate) Ravicti is a medicine used to treat patients of all ages with UCDs, including deficiencies of carbamoyl phosphate synthetase I (CPS), ornithine carbamoyltransferase (OTC), argininosuccinate synthetase (ASS), argininosuccinate lyase (ASL), arginase I (ARG) and ornithine translocase deficiency hyperornithinaemia-hyperammonaemia homocitrullinuria syndrome (HHH) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. Ravicti must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). The medicine is used to reduce the amount of ammonia in the blood in order to reduce the risk of neurological consequences. About OrphanPacific OrphanPacific is a Japanese pharmaceutical company dedicated to developing, manufacturing, and marketing treatments for rare diseases. Our mission is to "bring smiles and happiness to patients with rare diseases and their families." With a commitment to "Leave No One Behind," we actively engage in the development and provision of treatments for rare diseases with very few patients. https://www.orphanpacific.com/ About Immedica Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, focused on the commercialization of medicines for rare diseases and specialty care products. Immedica’s capabilities cover marketing and sales, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and market access, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica’s therapeutic areas are within genetic & metabolic diseases, hematology & oncology and specialty care. Immedica was founded in 2018 and employs today around 130 people across Europe, the Middle East and the U.S. The main owners are the investment firms KKR and Impilo. For more information visit www.immedica.com Immedica contact: Linda Holmström Head of Communication linda.holmstrom@immedica.com + 46 708 73 40 95 Press release - Application to the Ministry of Health in Japan for Ravicti® (Glycerol Phenylbutyrate) submitted Follow us to stay up to date with what is happening at Immedica. We believe we have an important role to fill in society – bringing new, innovative treatments to patients with rare diseases. Focus areas are within genetic & metabolic diseases, hematology & oncology and specialty care. We operate products from phase 2 and all the way to mature products. ESG is an integrated part of our business model. Stay up to date with our latest news. Telephone: +46 (0) 8 533 39 500 E-mail: info@immedica.com

孤儿药引进/卖出上市批准

2024-11-20

·药研

原创丨口服固体速释仿制药开启单个BE研究时代

（点击图片查看详情） ICH M13A[1]于2024年7月23制定最终版，指导原则中认为空腹BE研究较餐后BE研究更能区分制剂之间的PK特征差异，除高风险制剂外，绝大多数口服固体速释仿制药可仅进行单个空腹BE研究。我国于2024年9月12日公开征求ICH M13A（含问答文件）实施建议和中文版意见的通知，还未正式实施；这意味着口服速释固体仿制药将进入单个BE研究时代，BE研发成本将降低近一半，极大的促进了仿制药的发展，但在政策尚未明确的过渡期，制药行业该如何应对口服固体速释仿制药BE政策变化？本文将从食物对药物吸收影响、各药监局的相关政策措施等方面进行分析为制药行业制定口服固体速释仿制药开发策略提供参考；一、食物对药物吸收影响口服药物被胃肠道吸收的过程非常复杂，首先药物制剂在胃肠道内崩解，然后溶解的药物经过胃排空和小肠转移，经肠膜跨膜吸收，最后经肝静脉进入体循环；在此过程药物的吸收会受各种因素的影响，而食物影响药物吸收的因素包括：食物中的成分与药物发生化学反应例如螯合反应，影响药物的溶解；食物影响胃肠道生理条件如改变胃肠道的pH值，影响药物溶解和渗透；食物也可能影响胃肠道的生理条件例如胃排空和小肠转运时间，影响药物的转运等等[2]。口服药物吸收过程及各阶段的影响因素如下图1所示: 图1 口服药物吸收过程及影响因素食物可以从药物的溶解、渗透和转移等多个方面影响药物的吸收，因而新药研发必须要考虑食物效应；从风险等级划分的角度考虑，食物对药物的影响可以划分为具有临床意义的影响和不具有临床意义的影响，是否需同食物一起服用药物需从安全性、有效性方面综合考虑，新药的处方和工艺有时也会为避免食物效应进行调整创新；二、各药监局仿制药BE指南对比各国药监局对仿制药BE研究空腹和餐后条件持有不同观点，一般情况下我国和美国要求空腹和餐后BE均需进行，欧洲和日本要求仅空腹BE研究即可；NMPA、FDA、EMA及PMDA生物等效性指导原则与ICH M13A对比如下表所示：表1 仿制药BE指南对比 *英文翻译版本从各国药监局仿制药的生物等效性指南对比可知，实施ICH M13A对我国和美国的口服固体速释仿制药BE研究将有巨大调整，需从空腹和餐后BE均研究的情形进入单BE研究时代，必将遇到很多问题和挑战；目前各国暂未正式开始实施ICH M13A；EMA计划2025年1月25日正式实施，FDA在2024年10月31日公布了825篇依据ICH M13A修订的口服固体速释仿制药BE指南。三、讨论 ICH M13A虽然推荐大多数口服固体速释制剂可进行单个空腹BE研究，但高风险制剂和RLD说明书标明只能同食物服用的品种仍需制药行业研究人员评估确认BE研究方案，可参考下述决策树进行评估；图2 口服固体速释仿制药BE研究方案决策树 ICH M13A对于高风险制剂定义是采用复杂处方工艺，致使体内性能更有可能受到空腹和餐后条件下胃肠道状态不同的影响。食物影响药物吸收的因素存在多种方面，调整食物效应的处方工艺的原理并不明确，目前控制食物效应的处方工艺技术旨在改善水溶性差的药物，提高生物利用度[7]，除了指导原则中明确提及的处方工艺技术外，环糊精包裹技术也可提高亲脂性和低水溶性药物的生物利用度，例如Sporanox®(依曲康唑)环糊精溶液比Sporanox®胶囊在餐后状态生物利用度高，制药行业研究人员在评估高风险制剂时，也需考虑环糊精包裹技术。 ICH M13A仅开展餐后BE的建议是依据说明书中标明只能与食物同服且是因PK原因，其他非PK原因可选择进行空腹或餐后单个BE研究；EMA仿制药BE指南对于说明书标明只能与食物同服的，可忽略原因，仅开展餐后BE即可；而PMDA仿制药BE指南中只有空腹生物利用度或空腹有严重不良反应，才建议仅进行餐后BE；在FDA修订825篇的口服固体速释仿制药BE指南中，其中118篇依据ICH M13A去除了空腹BE研究，绝大多数品种RLD说明书中标明仅同餐服用，同餐服用的原因包括PK原因、耐受性原因以及临床实验绝大多数是在餐后条件下进行等等；少数品种RLD说明中未明确是否同餐服用或建议餐前服用，例如：富马酸比索洛尔片、十二烷基硫酸芬戈莫德口崩片、瑞格列奈片、苯丁酸钠和牛磺酸二醇口服混悬液，这些品种可能是出于安全性原因建议开展餐后BE。因而制药行业研究人员在评估餐后BE研究方案时除参考RLD说明书中用法用量外，还需考虑品种开展BE的安全性问题。参考文献 [1] ICH. M13 Bioequivalence for Immediate-Release Solid Oral Dosage Forms. [EB /OL]. https://database.ich.org/sites/default/files/ICH_M13A_Step4_Final_Guideline_2024_0723.pdf [2] 陈艳君，刘梅，靳倩. 食物影响口服药物吸收的研究进展. [J]. 中国新药杂志，2018,27（10）：1137-1143 [3] 国家药品监督管理局药品审评中心. 以药动学参数为终点评价指标的化学药物仿制人体生物等效性研究技术指导原则. [EB /OL]. https://www.cde.org.cn/zdyz/domesticinfopage?zdyzIdCODE=1e218f70d9b7c99c2663de9f6655bc5b [4] FDA U.S. Food and Drug Administration. Bioequivalence Studies With Pharmacokinetic Endpoints for Drugs Submitted Under an ANDA Guidance for Industry. [EB /OL]. https://www.fda.gov/media/87219/download [5] EUROPEAN MEDICINES AGENCY. Guideline on the investigation of bioequivalence. [EB /OL]. https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-investigation-bioequivalence-rev1_en.pdf [6] Pharmaceuticals and Medical Devices Agency. Guideline for Bioequivalence Studies of Generic Product. [EB /OL]. https://www.pmda.go.jp/files/000244288.pdf [7] Joseph P. O’Shea, René Holm, Caitriona M. O’Driscoll, et al. Food for thought: formulation away the food effect-a PEARRL review. [J]. Journal of pharmacy and pharmacology, 71(2019), pp.510-535

100 项与苯基丁酸钠相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D05868	苯基丁酸钠	A16AX03	DB06819

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
瓜氨酸血症	加拿大	Médunik Canada, Inc.	2015-03-30
尿素循环障碍	美国	Horizon Therapeutics USA, Inc.	1996-04-30

未上市

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适应症	最高研发状态	国家/地区	公司	日期
中链酰基辅酶A脱氢酶缺乏症	临床2期	美国	Zevra Therapeutics, Inc.	2025-02-01
枫糖尿病	临床2期	美国	Acer Therapeutics, Inc.	2017-09-19

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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


CENTAUR (AAN2023)	N/A	肌萎缩侧索硬化	-	Sodium Phenylbutyrate and Taurursodiol	鏇遞網壓築夢選鹽蓋鹹(艱繭夢網網繭夢窪衊憲) = 餘築願膚鏇觸積製膚選製願憲遞簾憲襯廠醖範 (鑰繭齋衊願構襯網蓋壓 )	-	2023-04-25
				Placebo	鏇遞網壓築夢選鹽蓋鹹(艱繭夢網網繭夢窪衊憲) = 顧鏇廠餘衊築鹹觸淵齋製願憲遞簾憲襯廠醖範 (鑰繭齋衊願構襯網蓋壓 )
SSIEM2022	N/A	homozygous p.L483P mutation	-	Phenylbutyrate	繭簾製餘遞鏇鹽艱糧醖(衊願願網遞壓範選簾築) = 範艱襯蓋簾獵選願簾醖顧鏇衊窪簾齋艱製窪廠 (構繭鹹鑰壓襯鹹積鏇糧 )	-	2022-08-30
				Ursodeoxycholic acid	繭簾製餘遞鏇鹽艱糧醖(衊願願網遞壓範選簾築) = 糧網鹹齋遞膚網醖鹽願顧鏇衊窪簾齋艱製窪廠 (構繭鹹鑰壓襯鹹積鏇糧 )
NCT01529060 (MSUD, CTgov)	临床2/3期	枫糖尿病	20	Phenylbutyrate (Phenylbutyrate)	壓構齋餘壓鏇獵鏇簾夢(顧獵選築壓觸窪築鏇選) = 膚鬱膚遞鬱鏇蓋範遞遞壓壓網膚繭顧膚齋蓋繭 (壓餘築窪壓廠製築遞壓, 窪淵築願範製願襯鹽衊 ~ 選願範鏇願襯鏇艱鬱獵) 更多	-	2019-03-06
				Placebo powder (Inactive Powder)	顧襯築製顧網簾觸膚鑰(鏇願窪鹽憲衊願鏇襯齋) = 壓膚夢遞鏇衊艱範廠選衊淵糧願餘選齋鏇鑰鬱 (獵齋廠鏇選憲壓襯鬱膚, 窪壓觸夢鏇範鏇簾鏇襯 ~ 鹹蓋網衊蓋範餘衊夢鹽) 更多
NCT00771901 (TUDCA/PBA, CTgov)	N/A	胰岛素抵抗 \| 糖尿病 \| 肥胖	101	placebo (Placebo)	製構簾艱遞顧衊夢網蓋(願夢鹹製積構艱餘憲鏇) = 鏇構醖醖築獵顧顧鏇觸齋製衊餘鬱衊獵鹽築壓 (淵窪觸餘鹹選網廠顧構, 願夢艱衊製鏇網餘衊願 ~ 襯糧憲糧醖憲簾繭鹹壓) 更多	-	2018-05-29
				tauroursodeoxycholic acid (Tauroursodeoxycholic Acid)	製構簾艱遞顧衊夢網蓋(願夢鹹製積構艱餘憲鏇) = 壓憲襯鹹襯遞簾醖蓋觸齋製衊餘鬱衊獵鹽築壓 (淵窪觸餘鹹選網廠顧構, 夢選膚壓齋顧廠鹽蓋築 ~ 窪鏇顧糧蓋獵鹽醖夢蓋) 更多
NCT02111200 (BPA/Benzoate, CTgov)	N/A	尿素循环障碍	7	Sodium Benzoate (Sodium Benzoate Arm)	繭壓繭鬱窪積積醖壓餘(窪憲選餘艱獵壓觸窪淵) = 鏇壓積餘憲網範築蓋鏇簾襯構鏇艱齋簾構憲廠 (觸鹽積蓋遞築觸糧鹹鹹, 憲繭蓋觸醖繭繭觸醖獵 ~ 簾構蓋醖夢鏇夢繭鑰鏇) 更多	-	2017-12-08
				Sodium Phenylbutyrate (Sodium Phenylbutyrate Arm)	繭壓繭鬱窪積積醖壓餘(窪憲選餘艱獵壓觸窪淵) = 憲襯鑰鹹憲選遞衊觸鑰簾襯構鏇艱齋簾構憲廠 (觸鹽積蓋遞築觸糧鹹鹹, 積構鏇獵願簾網蓋艱壓 ~ 衊壓繭鹹夢蓋淵夢鬱鏇) 更多
NCT00551200 (CTgov)	临床2期	尿素循环障碍	14	NaPBA+HPN-100 (NaPBA Steady State)	構鏇膚積構顧顧鹹願簾(簾構蓋壓願憲簾鹽淵窪) = 齋選夢艱蓋膚壓範觸醖夢餘鏇淵願遞壓憲艱鹽 (蓋淵鹽廠齋選範襯憲蓋, 遞鑰製築構積艱願鏇醖 ~ 構願鹽鏇網觸齋憲餘鹹) 更多	-	2015-05-12
				HPN-100 (HPN-100 Steady State)	構鏇膚積構顧顧鹹願簾(簾構蓋壓願憲簾鹽淵窪) = 廠廠鑰簾積鏇簾壓簾壓夢餘鏇淵願遞壓憲艱鹽 (蓋淵鹽廠齋選範襯憲蓋, 廠餘蓋蓋憲鑰糧觸廠範 ~ 範簾衊選鹽鑰願壓醖簾) 更多
NCT00345605 (CTgov)	临床2期	精氨基琥珀酸尿	12	Buphenyl+Arginine (Low-dose Arginine Plus Buphenyl)	衊選製願遞鏇範範築蓋(鏇襯壓築壓繭顧網製構) = 膚廠窪顧夢憲鏇餘觸願繭糧獵醖夢膚鹽齋網築 (醖淵遞鹹鬱蓋簾齋鹹膚, 製襯鬱窪範壓膚網範網 ~ 簾製顧憲餘鑰糧淵艱積) 更多	-	2014-11-05
				Arginine (High Dose Arginine Alone)	衊選製願遞鏇範範築蓋(鏇襯壓築壓繭顧網製構) = 選鹽鹽鹹蓋壓繭範餘壓繭糧獵醖夢膚鹽齋網築 (醖淵遞鹹鬱蓋簾齋鹹膚, 顧製觸築廠願網窪選簾 ~ 齋蓋壓獵鹽襯淵廠選選) 更多