英文药名：Vyndaqel capsules（Tafamidis Meglumine） 中文药名：氯苯唑酸胶囊 生产厂家：辉瑞公司 ビンダケルカプセル20mg 药物分类名称 TTR型淀粉样变性治疗药物 批准日期：2013年11月 商標名 Vyndaqel capsules 20mg 一般名 タファミジスメグルミン（Tafamidis Meglumine） 化学名 2-（3,5-Dichlorophenyl）-1,3-benzoxazole-6-carboxylic acid mono（1-deoxy-1-methylamino-D-glucitol） 分子式 C14H7Cl2NO3・C7H17NO5 分子量 503.33 構造式 性状 本品为白色至粉红色红色粉末。 该产品难溶于水和甲醇，非常不溶于乙醇（95）。 处理注意事项 避免在高温下储存。 批准条件 由于日本的临床试验案例数量极为有限，在复审期间，通过针对所有病例进行使用结果调查，掌握了使用该药物的患者的背景信息，在早期阶段收集有关安全性和有效性的数据，并采取必要措施正确使用该药物。 药用药理学 作用机制 Tafamidis通过结合四聚体的两个甲状腺素结合位点之一来稳定四聚体，四聚体是TTR的天然结构并抑制其解离和变性。 （1）在体外试验中，与TTR结合的解离常数为2至3nmol/L（Kd 1）和154至278nmol/L（Kd 2）。 （2）体外试验在野生型，V30M和V122I突变体TTR（3.6μmol/L）的酸性条件下抑制原纤维形成，EC 50值分别为2.7,3.2和4.1μmol/L。 （3）在体外试验中，用野生型V30M，V122I突变体TTR向人血浆中加入贝伐珠单抗3.6或7.2μmol/ L，以浓度依赖性方式抑制尿素对四聚体的解离。 （4）将7.2μmol/L的阿利吉仑加入到从具有除V30M以外的突变的受试者收集的血浆中，并且在25种突变型中抑制尿素的解离。 适应症 抑制转甲状腺素蛋白型家族性淀粉样多发性神经病周围神经病变的进展 用法与用量 成人，每天口服一次，每次服用20mg。 包装 胶囊 20毫克：14粒（PTP） 存储方法 存储 室温 制造销售 辉瑞公司 注：以上中文资料不够完整，使用者以原处方资料为准。 完整说明书附件：http://www.info.pmda.go.jp/go/pack/1290001M1022_1_03/ New release of transthyretin-type familial amyloid polyneuropathy (TTR-FAP) therapeutic agent for peripheral nerve disorder "Bindakel capsule 20 mg" ~ First rare treatment medicine for rare refractory neurological diseases ~ Inhibition of peripheral neuropathy progression in transthyretin-type familial amyloid polyneuropathy (TTR-FAP), a rare refractory neurological disease. New drug is Japan's first therapeutic drug "Bindakel® Capsule 20mg" (Common name: tamhanzis meglumine). TTR - FAP is a progressive, fatal neurological disease, estimated to be about 130 in the country, and it is designated as a target disease of overcoming intractable disease research project. In patients with TTR-FAP, the transthyretin tetramer, which is a precursor protein, dissociates and degenerates due to genetic mutation of transthyretin, and as a result, it is deposited on organs such as nerve, heart and kidney as amyloid, Various functions are hindered. In many cases, the disease is progressive, eventually becoming bedridden, leading to death in an average of 10 years from the onset. Traditionally, the only treatment that can be expected to suppress disease progression for patients was liver transplantation, but due to lack of donor, problems of complications accompanying surgery and necessity of administration of immunosuppressant over a long period after surgery, The current situation is that patients are very limited. "Bindakel capsule 20 mg" is a drug that stabilizes transthyretin by suppressing dissociation of transthyretin tetramer and has a mechanism to suppress accumulation of amyloid causing peripheral nerve degeneration . In November 2011, it was approved in Europe as the world's first treatment for suppressing the progression of peripheral neuropathy in TTR-FAP. In Japan, we started Phase 1 trial in August 2011 and applied for manufacturing and marketing approval in February 2013. In addition, Bindaquel received designation of rare disease medicine from the Ministry of Health, Labor and Welfare and was reviewed as a priority review, and in a very short period of 7 months from the application, it was manufactured from the Ministry of Health, Labor and Welfare on September 20 this year We obtained sales approval. Professor Sangjiang, Professor of Neurosurgery at Nagoya University Graduate School of Medicine, said, "There are only treatments for TTR-FAP in the liver, but many patients do not receive organs, symptomatic treatment It was the present situation that I will send my hard days only and will pass away. Japan is a gathering place of TTR-FAP globally, but being able to intervene from early treatment with internal medicine due to the release of Bindaker will be a big gospel for all TTR-FAP patients, and future diagnosis and treatment of TTR-FAP Development is expected. " What is transthyretin-type familial amyloid polyneuropathy (TTR-FAP) Transthyretin-type familial amyloid polyneuropathy (TTR-FAP) is a disorder caused by transthyretin having a genetic mutation, resulting in dysfunction due to amyloid deposits on whole body organs such as nerves, heart and eyes. It is said that there are about 10,000 patients in the world. Japan is one of the collection areas for patients lined with Portuguese and Sweden, and the estimated number of patients in Japan is estimated at about 130 people. Symptoms such as polyneuropathy characterized by sensory loss of lower limbs and pain muscle strength, erectile dysfunction, diarrhea constipation alternation, unintentional weight loss, orthostatic hypotension, urinary incontinence, urinary retention, delay of gastric emptying etc. Because of autonomic nervous system injury, the quality of life is greatly reduced. TTR-FAP is progressive, leading to deaths on average 10 years after onset. Bindakel capsule 20 mg " Product name: Bindakel capsule 20 mg Common name (JAN) Tafamisis meglumine (Tafamidis Meglumine) Manufacture and sale approval date 20th September 2013 Drug price listing date November 19, 2013 Release date 20 November 2013 Manufacture and sale: Pfizer Inc. Efficacy/efficacy: Suppressing the progression of peripheral neuropathy in transthyretin-type familial amyloid polyneuropathy DOSAGE AND ADMINISTRATION: Usually, adults are orally administered once a day 20 mg once a day as a tamerminis meglumine