MOUNTAIN VIEW, Calif., June 8, 2011 /PRNewswire/ -- Edison Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has allowed an Expanded Access program to provide EPI-743 to seriously ill patients diagnosed with inherited respiratory chain diseases of the mitochondria. Patients with genetically confirmed disease and patients who meet specific clinical criteria, absent genetic confirmation are both eligible.
As of June 1, 2011, 40 seriously ill subjects diagnosed with genetically confirmed inherited mitochondrial disease have been treated with EPI-743 worldwide for a cumulative exposure of over 7,436 days. No significant drug-related adverse events have been observed. Initial clinical, biomarker, and brain-imaging data are encouraging. The company cautions that the data obtained in these initial studies is preliminary and needs verification in controlled prospective trials. Edison and its clinical investigator team are working closely with FDA and the European Medicines Agency to expedite prospective phase 2B/3 clinical trials. EPI-743 has received orphan designation from FDA.
Clinical Trial Sites
Clinical trial enrollment sites have been established in North America at Lucile Packard Children's Hospital– Stanford University Medical Center; Akron Children's Hospital; Columbia University; Seattle Children's Hospital; Medical University of South Carolina; Children's Hospital of Philadelphia; and University of California, Los Angeles.
Additional trial sites are being established in Europe, Japan, and in North America.
United Mitochondrial Disease Foundation
Friedreich's Ataxia Research Alliance
United States Food and Drug A