Edison's EPI-743 granted orphan drug designation
MOUNTAIN VIEW, Calif. — The Food and Drug Administration has granted orphan drug designation to an investigational treatment made by Edison Pharmaceuticals for rare diseases, Edison said Wednesday.
The drug maker announced that the FDA had granted the designation to EPI-743, a drug for inherited mitochondrial respiratory chain diseases, which affect an estimated 60,000 people. The agency grants orphan drug designation to drugs for diseases that affect fewer than 200,000 people in the United States, providing accelerated review of approval applications and market exclusivity periods of seven years, compared with the five years given to most drugs.
Inherited mitochondrial diseases cause defects in how cells make and regulate energy and can affect virtually any organ system in the body, causing such symptoms as central nervous system disorders, diabetes, heart failure, liver failure, deafness, blindness and others.