Edison Pharmaceuticals announced that the FDA has granted Fast Track designation to EPI-743, a study drug for the treatment of Friedreich’s ataxia.
In one Phase 1 and multiple Phase 2 studies, EPI-743 was demonstrated to be safe and well tolerated. Currently, two Phase 2 trials are underway with complete enrollment in a Phase 2b randomized, double-blind, placebo-controlled trial. EPI-743 is also being studied in a single-arm, subject-controlled trial for a rare Friedreich’s ataxia genetic subtype where patients have a point mutation in the gene encoding frataxin.
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EPI-743 is an orally bioavailable small molecule being developed for the treatment of Friedreich’s ataxia and other inherited mitochondrial diseases. It is a part of the para-benzoquinone class that augments endogenous glutathione biosynthesis, which is essential for the control of oxidative stress.
The FDA has previously granted Orphan Drug designation status to EPI-743 for the treatment of Friedreich’s ataxia.
For more information call (650) 641-9200 or visit EdisonPharma.com.