FDA授予Edison制药EPI-743治疗Leigh综合征孤儿药地位

FDA授予Edison制药EPI-743治疗Leigh综合征孤儿药地位

                               
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发布日期：2014-06-10  来源：新药汇  
FDA授予Edison制药EPI-743治疗Leigh综合征的孤儿药地位。此前，欧盟和日本也已授予EPI-743治疗Leigh综合征的孤儿药地位。

Edison制药6月9日宣布，FDA已授予vatiquinone治疗Leigh综合征(Leigh syndrome,LS)的孤儿药地位。vatiquinone是该公司实验性药物EPI-743的国际非专利名称（INN），又名通用名称（generic name）。

目前，EPI-743正处于IIb/III期开发，用于Leigh综合征的治疗。在美国，儿科Leigh综合征患者中开展的IIb期随机双盲安慰剂对照试验已完成患者招募工作。此外，Edison与日本住友制药联合开展的一项IIb/III期试验正在日本开展。
此前，FDA已授予EPI-743用于治疗线粒体遗传性呼吸链疾病和弗里德赖希共济失调（Friedreich's Ataxia）的快速通道地位及孤儿药地位，同时欧洲药品管理局（EMA）和日本劳动卫生福利部（MHLW）已授予EPI-743治疗Leith综合征的孤儿药地位。
关于Leigh综合征：
Leigh综合征是一种主要发生于儿科患者的遗传性、致命性、进行性神经系统变性病，目前无治疗药物。该病是一种最常见形式的儿科遗传性线粒体病，主要特点是负责呼吸控制和其他神经系统功能的中枢神经系统区域双侧坏死（死亡）。Leigh综合征属于线粒体病家族，共有的生化特征为细胞能量代谢存在缺陷。
关于EPI-743：
EPI-743是一种口服生物可利用小分子药物，属于苯醌类药物，目前正处于临床开发阶段，主要集中于遗传性线粒体疾病的治疗。EPI-743通过口服给药传递到大脑，通过调节涉及能量代谢合成和调控的关键酶发挥作用。EPI-743的作用机制包括：1）提高谷胱甘肽的合成，2）优化代谢控制，3）增强细胞氧化应激管理中关键遗传元件的表达，4）作用于线粒体，调节电子传递。
目前，Edison也正在探索EPI-743用于其他遗传性呼吸链疾病的治疗。通过扩展获取协议和前瞻性临床试验，EPI-743已累计给药1.3万例患者，涉及15种疾病，并已记录到了良好的安全性。
英文原文：FDA Grants Edison Pharmaceuticals' EPI-743 Orphan Status for Leigh Syndrome
EPI-743 in phase 2B development for Leigh syndrome
MOUNTAIN VIEW, Calif., June 9, 2014 /PRNewswire/ -- Edison Pharmaceuticals today announced that the FDA has granted Orphan Drug status to vatiquinone for the treatment of Leigh syndrome.
Vatiquinone is the International Nonproprietary Name (INN) for Edison's EPI-743. The INN is a unique international name issued by the World Health Organization, which is used to identify the active pharmacological ingredient in a drug.  This is also known as the generic name.
EPI-743 is currently in phase 2B/3 development for the treatment of Leigh syndrome. A phase 2B randomized double-blind placebo-controlled trial in children with Leigh syndrome is fully enrolled in the United States, and a phase 2B/3 trial of EPI-743, which is being conducted in conjunction with Dainippon Sumitomo Pharma Co, Ltd, is ongoing in Japan.
Orphan designation was established as part of the Orphan Drug Act which was passed by the US Congress in 1983 to encourage the development of drugs for the treatment of rare, orphan diseases. The FDA grants orphan status to drugs that are being developed specifically to treat a rare condition and have shown potential benefit for the indication. Orphan designation affords several advantages including a more expedited drug approval process and an extended period of market exclusivity.
Edison Pharmaceuticals has previously received Orphan Designation from the FDA for the treatment of inherited respiratory chain diseases of the mitochondria and Friedreich's ataxia, and from the European Medicines Agency Committee on Orphan Products and Japanese Ministry of Health, Labor and Welfare for the treatment of Leigh syndrome.
Leigh Syndrome
Leigh syndrome is an inherited lethal, progressive, predominately pediatric, neuromuscular disorder for which there are no approved treatments. It is the most common pediatric inherited mitochondrial disease. Initially described in 1951, the hallmarks of the disease include bilateral necrosis (death) of central nervous system regions responsible for the control of breathing and other neurologic functions. Leigh syndrome belongs to a large family of disorders identified as "mitochondrial disease." The disorders share as a common biochemical feature defects in cellular energy metabolism.
EPI-743
EPI-743 is an orally bioavailable small molecule being developed by Edison Pharmaceuticals for inherited mitochondrial diseases, and is a member of the para-benzoquinone class of drugs. The mechanism of action of EPI-743 involves: i) augmenting the synthesis of glutathione; ii) optimizing metabolic control; iii) enhancing the expression of genetic elements critical for cellular management of oxidative stress; and iv) acting at the mitochondria to regulate electron transport. The utility of EPI-743 is also being explored in other inherited respiratory chain disorders.
Edison Pharmaceuticals
Edison Pharmaceuticals is a specialty pharmaceutical company dedicated to developing treatments for children and adults with orphan mitochondrial diseases.

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