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news.The FDA has granted orphan drug designation for Edison Pharmaceuticals' investigational product EPI-A0001 for treatment of inherited mitochondrial respiratory chain diseases which affect an estimated 60,000 individuals in the US.
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The 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 preclinical 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 mitochondrial 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.