“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.” “Fast Track designation will facilitate Edison’s clinical development of EPI-743 for patients with Friedreich’s ataxia,” said Guy Miller, MD, PhD, chairman and chief executive of Edison Pharmaceuticals. In addition, EPI-743 is being evaluated in a rare Friedreich’s ataxia genetic subtype – patients with a point mutation in the gene encoding frataxin.
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A phase 2B trial in adults with Friedreich’s ataxia is fully enrolled and is expected to be completed in the third quarter of 2014. Two phase 2 trials in patients with Friedreich’s ataxia are ongoing.
In these studies, EPI-743 has been demonstrated to be safe and well tolerated. Friedreich’s ataxia is a highly debilitating and life-shortening disease.ĮPI-743 has successfully completed phase 1 and multiple phase 2 studies. Patients have “energy failure” symptoms, including ataxia, muscle weakness, heart failure, diabetes, and visual, speech and hearing deficiencies. Friedreich’s ataxia is caused by a defect in the gene frataxin.
If Edison can gain approval, EPI-743 will be the first ever treatment for Friedreich’s ataxia, and already has orphan status in the US for the condition.ĭifferent types of mitochondrial disease all share common biochemical defects in the mitochondrion, special ‘power generators’ found in the body’s cells.įriedreich’s ataxia is an autosomal recessive nuclear DNA inherited mitochondrial disease, and affects an estimated 1 in 50,000 individuals in the United States and Europe. A new treatment for the rare disease Friedreich’s ataxia has been given ‘fast track’ status by the FDA.Įdison’s EPI-743 is being developed for a range of paediatric and adult mitochondrial disease, of which Friedreich’s ataxia is just one.
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