Orphan Drug Designation for Inherited Mitochondrial Respiratory Chain Diseases
Edison Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to EPI-A0001 for treatment of inherited mitochondrial respiratory chain diseases. These mitochondrial diseases today affect an estimated
The Orphan Drug Act, passed in 1982, provides incentives for companies to pursue treatments for diseases affecting fewer than 200,000 individuals in the United States. Such designation provides an accelerated review and a seven-year period of exclusivity upon FDA approval.
The FDA granted orphan drug designation to Edison’s EPI-A0001 based on review of the application, which included pre-clinical data demonstrating a favorable efficacy and safety profile. Current data is consistent with EPI-A0001 targeting electron shuttling and energy production– two processes that are impaired by genetic defects in the (mitochondrial) respiratory chain.
The respiratory chain is located within the inner mitochondrial membrane and is comprised of numerous proteins encoded for by both the nuclear and mitoch ondrial genome. Genetic errors in the synthesis of these proteins results in a variety of clinical conditions that have disruptions in energy production as a common biochemical feature. These diseases frequently affect skeletal and cardiac muscle, as well as the nervous system, and thus are often classified as mitochondrial encephalomyopathies. They are highly debilitating, and life shortening. Edison is partnered with leading academic centers and foundations to develop first-in-class therapeutics for the treatment of these diseases.
About Edison Pharmaceuticals
Edison Pharmaceuticals, Inc. is focused on the development of drugs to treat energy impairment diseases, also referred to as mitochondrial diseases. The company is advancing into clinical development EPI-A0001 for the treatment of inherited mitochondrial diseases predominately affecting children. Edison’s technology platform consists of a specialized knowledge in redox medicinal chemistry, which the company believes is essential to drugging key targets contained within mitochondria. Edison will leverage clinical and laboratory data obtained in rare, or orphan, diseases to pursue other conditions that share common mechanisms. The company has obtained substantial non-dilutive peer-reviewed grant support to advance both its pre-clinical and clinical initiatives from foundations including the Muscular Dystrophy Association, Friedreich’s Ataxia Research Alliance, and Seek A Miracle.
Ms. Lorraine Gilmore