Ocugen, Inc. Announces Rare Pediatric Disease Designation Granted for OC410ST--Modifier Gene Therapy for the Treatment of Stargardt Disease

Published: 2025-05-27 08:05:30 ET OCGN

Published on 05/27/2025 at 08:05

Ocugen, Inc. announced that the United States Food and Drug Administration (U.S. FDA) has granted Rare Pediatric Disease Designation (RPDD) for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19, andcone-rod dystrophy 3. Previously, OCU410ST received Orphan Drug designations for the treatment of ABCA 4-associated retinopathies from the FDA and European Medicines Agency. The FDA grants RPDD for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the U.S. There are approximately 100,000 people in the U.,S. and Europe combined living with Stargardt disease. With this designation for OCU410ST, Ocugen may be awarded a Priority Review Voucher (PRV), if the PRV program is authorized by the U.S. Congress.

The PRV program is designed to incentivize drug development for serious rare pediatric diseases. If awarded, a PRV can be redeemed to receive priority review for a different product or sold to another sponsor and typically sells for about $100 million. Ocugen is committed to advancing the OCU410ST program through clinical development and plans to initiate the Phase 2/3 pivotal confirmatory trial in the next few weeks with a target Biologics License Application (BLA) filing in 2027.