The FDA granted rare pediatric disease designation for OCU410ST, a gene therapy designed to treat ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 and cone-rod dystrophy 3, according to Ocugen.
Ocugen may receive a priority review voucher (PRV), which is designed to incentivize drug development for serious rare pediatric diseases, if Congress reauthorizes the PRV program, according to a press release. The company would then be able to redeem the PRV to receive priority review for a different product or sell it to another sponsor.
The FDA and European Medicines