Edison Pharma gets another orphan drug designation for vatiquinone
- Edison Pharmaceuticals has been granted Orphan Drug status by FDA for its investigational treatment for Leigh Syndrome---EPI-743 (vatiquinone).
- Leigh Syndrome is a fatal, genetic neurometabolic disease that affects one of every 40,000 newborns—except in a distinct region of central Quebec where the incidence is one in 2,000 live births.
- Edison Pharmaceuticals has already received orphan drug designation for Leigh Syndrome in the EU and Japan.
According to the National Institutes of Health, Leigh Syndrome is a rare but devastating disease, which starts during infancy with signs of general weakness and poor development. It progresses to vomiting, seizures, lactic acidosis and loss of respiratory and kidney function. On average, affected children die by age 6 or 7. The current standard of care is a low-carbohydrate/high-fat diet and thiamine, as well as treatment for lactic acidosis. The pronounced unmet medical need allowed California-based Edison Pharmaceuticals, which specializes in developing drugs for mitochondrial disorders, to pursue orphan drug designation and the expedited timeline and additional support that comes with this status.
EPI-743 is a para-benzoquinone and is currently in phase IIb/III development. As a company that focuses on developing treatments for mitochondrial diseases, Edison’s strategy has been to pursue orphan drug designation for numerous indications, as well as from numerous regulatory bodies outside of the US, for EPI-743. In February, EPI-743 was granted orphan drug status for the treatment of Friedreich’s ataxia in adults—a mitochondrial disorder that affects 1 in 30,000 people in the US and Europe. Trial enrollment for the Leigh Syndrome IIb/III study is underway.
- Pharmaceutical Business Review Edison's EPI-743 gets FDA orphan drug status for Leigh syndrome
- NIH NINDS Leigh's Disease Information Page