Guy Miller, Chairman and CEO of Edison Pharmaceuticals, said “Fast Track designation will facilitate Edison'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.”
EPI-743 is an orally bioavailable small molecule that belongs to the para-benzoquinone class of drugs. The company’s lead drug is currently being developed for pediatric and adult mitochondrial disease including Friedreich’s ataxia. EPI-743 has completed Phase I and Phase II studies, where it demonstrated safety and tolerability. A randomized, double blind, placebo controlled Phase II B trial in adults with Friedreich’s ataxia is fully enrolled and expected to be completed in the third quarter of this year.
Friedreich’s ataxia is a rare autosomal recessive nuclear DNA inherited mitochondrial disease. It affects approximately 1 in 50,000 individuals. The disease is caused by a defect in the gene frataxin and manifests itself in energy failure symptoms, leaving patients with ataxia, muscle weakness, heart failure, and diabetes. Visual, speech, and hearing deficiencies may also be observed. The disease is severely debilitating and shortens life of affected patients.
The federal regulator has previously granted EPI-743 Orphan Drug Status for the treatment of Friedreich’s ataxia. The Fast Track designation provides drug sponsors a range of benefits including eligibility for accepted approval and access to greater communication opportunities with the FDA to discuss drug development approval. The FDA may also grant ‘rolling reviews’ to drugs with Fast Track designation, allowing the company to submit completed portions of a NDA for immediate review before the full application is completed.
Mr. Ron Bartek, President of Friedreich's Ataxia Research Alliance (FARA) and Chairman of the Board of the National Organization for Rare Disorders, said “We greet with tremendous enthusiasm the FDA's issuance of Fast Track designation to EPI-743 for Edison's Friedreich's ataxia development program. This demonstrates the FDA's commitment to the accelerated development of promising drugs for diseases which have no treatments.”