Edison制药3月18日宣布，FDA已授予EPI-743用于治疗弗里德赖希共济失调（Friedreich's Ataxia）的快速通道地位。此前，FDA已授予EPI-743治疗弗里德赖希共济失调的孤儿药地位。
目前，该公司正开发EPI-743用于儿童和成人线粒体疾病，包括弗里德赖希共济失调。在最近完成的一项I期和II期临床试验中，EPI-743已被证明具有良好的安全性和耐受性。此外，该公司正在开展2项II期试验，另一项IIb期试验也已完成患者招募，该项研究预计将于2014年第三季度完成。同时，该公司也正在一种罕见的弗里德赖希共济失调遗传亚型患者群体中调查EPI-743，该亚型患者群体的frataxin编码基因存在点突变。
关于
EPI-743是一种处于临床开发阶段的候选药物，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。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。
关于
弗里德赖希共济失调（Friedreich/s Ataxia， FRDA）属脊髓型共济失调的一种，遗传方式主要为常染色体隐性遗传，致病基因为X25基因，蛋白产物为frataxin，亚细胞定位研究发现frataxin定位于线粒体，FRDA发病与线粒体功能失调相关。
US-based Edison Pharmaceuticals has received Fast Track designation from the US Food and Drug Administration (FDA) for its lead drug EPI-743 to treat patients with Friedreich's ataxia.
The company is developing EPI-743 for pediatric and adult mitochondrial disease, including Friedreich's ataxia.
In the recently completed Phase I and multiple Phase II trials, EPI-743 has been demonstrated to be safe and well tolerated.
Currently, the company is conducting two Phase II trials in patients with Friedreich's ataxia and has completed patient enrollement in a Phase IIb randomized double-blind placebo-controlled trial, which is expected to be completed in the third quarter of 2014.
Additionally, EPI-743 is being assessed in a rare Friedreich's ataxia genetic subtype- patients with a point mutation in the gene encoding frataxin.
The company said that this single-arm subject-controlled trial is also fully enrolled.
Edison Pharmaceuticals chairman and CEO Guy Miller said fast track designation will facilitate the company's clinical development of EPI-743 for patients with Friedreich's ataxia.
"We are fully committed to delivering the first approved drug for this highly debilitating and lethal disease for which there are no FDA-approved therapies," Miller said.
The company has already received FDA Orphan designation status to EPI-743 for the treatment of Friedreich's ataxia.
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