Ocugen's Retina Gene Therapy Drug Gets Rare Disease Designation From FDA

Published: 2025-05-27 08:34:08 ET OCGN

Published on 05/27/2025 at 08:34

By Adriano Marchese

Ocugen's drug to treat retina diseases received a rare pediatric disease designation from U.S. regulators.

The biotechnology said Tuesday that the Food and Drug Administration granted the designation to OCU410ST, a gene therapy for the treatment of ABCA4-associated retinopathies, or genetic retinal disorders.

Previously, OCU410ST received orphan drug designations for the treatment of these retinopathies from the FDA and European Medicines Agency.

With the new designation, Ocugen may be awarded a priority review voucher, if the program is reauthorized by the U.S. Congress, the company said, which is a system designed to encourage drug development for rare diseases.

Write to Adriano Marchese at [email protected]

(END) Dow Jones Newswires

05-27-25 0833ET