Friedreich's Ataxia Research Alliance (FARA) has announced in a press release that the FDA has granted orphan drug status to Edison Pharmaceutical’s EPI-743 (vatiquinone). The drug treats Friedreich’s ataxia, a rare nervous system disorder. Guy Miller, MD, PhD, Chairman & CEO, Edison Pharmaceuticals said, “The FDA’s granting of orphan designation underscores the promise that EPI-743 has for the treatment of Friedreich’s ataxia.” Three sites in the Friedreich's Ataxia Research Alliance (FARA) Collaborative Clinical Research Network are conducting clinical trials with EPI-743.
Orphan Drug Designation
Orphan drug status confers incentives to drug companies working on pharmaceutical interventions for rare diseases and conditions. The Orphan Drug Act of 1983 established the Orphan Drug Designation. The FDA grants this special designation to drugs for rare conditions that demonstrate safety and efficacy. Incentives for companies include extended market exclusivity, development cost assistance, waiver of some fees, and review of applications within six months.
Friedreich’s ataxia is a neuro-muscular disorder. It affects adults and children with loss of coordination, vision, speech, and hearing deficits, scoliosis, diabetes, and heart problems. The disease usually degenerates until the patient is using a wheelchair and it shortens the life span. No FDA approved treatments are currently available.
Early in 2013, phase 2b clinical trials of EPI-743 began at the University of California in Los Angeles, the University of South Florida, and the Children's Hospital of Philadelphia. Participants are enrolled and further details about the trial are found at the Clinical Trials website. Study participants have genetically confirmed Friedreich's ataxia and have agreed to abstain from dietary supplements and non-approved drugs. If using hormone replacement therapy, they must maintain treatment consistently. Women in the reproductive years have agreed to use contraception.
Study authors have indicated the primary and secondary outcome measures in the research. Primary outcomes are an improvement in visual function and low contrast visual acuity. Secondary measures include improvements in color vision, neurologic function on the Friedreich's ataxia rating scale, neuromuscular function with a 25-foot walking test and a 9-hole peg test, cardiac function, blood biomarkers, and quality of life. Sixty people will participate in the studies expected to finish by April 2014.