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news.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.
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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.
Image: Friedreich’s ataxia is an autosomal recessive disorder that occurs when the FXN gene contains amplified intronic GAA repeats. Photo: courtesy of Emw