Ocugen’s OCU400 granted Orphan Drug designations for two diseases

Ocugen’s OCU400 – human nuclear hormone receptor subfamily 2 group E member 3 – has been granted orphan drug designations by the FDA for the treatment of retinitis pigmentosa and Leber congenital amaurosis. Currently, RP is associated with mutations in more than 100 genes, affecting approximately 110,000 people in the U.S. LCA is associated with mutations in more than 25 genes, affecting approximately 10,000 people in the U.S. There are currently no treatment options available for patients living with RP and LCA, and OCU400 has the potential to treat both with a single product.