Posted by Michael Wonder on 28 May 2025
Ocugen announces rare paediatric disease designation granted for OCU410ST—modifier gene therapy for the treatment of Stargardt disease
27 May 2025 - Ocugen today announced that the US FDA has granted rare paediatric disease designation for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.
Previously, OCU410ST received Orphan Drug designations for the treatment of ABCA4-associated retinopathies from the FDA and EMA.
