Washington, D.C. — The Friedreich's Ataxia Research Alliance announced today that the National Institutes of Health has accepted the University of Pennsylvania and Edison Pharmaceuticals into the Rapid Access to Interventional Development (RAID) Pilot Program for the development of Edison Pharmaceuticals EPI-A0001 for Friedreich's ataxia.
Under the NIH Roadmap initiative, the RAID Pilot Program has assembled an inter-institute team consisting of the National Institute of Neurological Disorders and Stroke, the National Institute of Child Health and Human Development and the Developmental Therapeutics Program of the National Cancer Institute that will combine resources and expertise to translate EPI-A0001 into the clinic. This NIH RAID project will provide drug development guidance and resources to enable an Investigative New Drug application to be filed with the Food and Drug Administration- the first step in its regulatory submission process. The Orphan Products Division of the Food and Drug Administration recently awarded EPI-A0001, Orphan Designation status for inherited respiratory chain diseases of the mitochondria.
"Our acceptance into the NIH RAID Pilot Program heralds the Friedreich's Ataxia Research Alliance (FARA) entering the era of therapeutic development," stated Ron Bartek, President of FARA. "The benefits of the last 8 years of discovery and clinical research funded by FARA are now beginning to be realized. This translational initiative brings us one step closer to the clinical evaluation of EPI-A0001, and evaluating its potential for arresting the damage done by Friedreich's ataxia. Our goal is to develop a therapy that will stop the devastating progression of Friedreich's ataxia in its tracks. We are grateful to all of the NIH institutes and their personnel who have provided invaluable support for this goal since FARA's inception."
Friedreich's ataxia is an inherited disease. It is caused by a defect in the gene encoding the protein frataxin, whose function is essential for biological energy production. In the last few years, medical and research communities have shown growing interest in Friedreich's ataxia. This attention is based on a renewed interest in the role that mitochondria play in disease, and an emerging body of literature suggesting that Friedreich's ataxia may share a common mechanism with other neurodegenerative diseases and movement disorders, as well as the aging process.
Today, there is no effective treatment or cure available for Friedreich's ataxia. It often first becomes noticeable as a balance and coordination problem in children of elementary school age. Further loss of strength and coordination in all four extremities usually forces those with the disease into wheelchairs by their teens. It is progressive and can lead to impaired vision, speech and hearing. Friedreich's ataxia is also commonly associated with severe heart disease, spinal abnormalities and diabetes. In the later stages of the disorder, patients are frequently incapacitated with cardiac failure leading to death. Friedreich's ataxia, although in relative terms rare, is the most prevalent inherited ataxia, affecting about 1 in every 50,000 people in the United States.
The Friedreich's Ataxia Research Alliance (FARA) is a 501(c)(3), non-profit, charitable organization dedicated to accelerating research leading to treatments and a cure for Friedreich's ataxia. Since its founding in 1998 by Friedreich's ataxia-affected families and scientists, FARA has provided more than $7 million in grants to scientists around the world who are pursuing treatments. http://www.FAResearchAlliance.org
President, Friedreich’s Ataxia Research Alliance