Ocugen receives Rare Pediatric Disease Designation for OCU410ST

Ocugen (OCGN) announced that the United States Food and Drug Administration has granted Rare Pediatric 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 European Medicines Agency. With this designation for OCU410ST, Ocugen may be awarded a Priority Review Voucher, if the PRV program is reauthorized 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 $100M. 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 filing in 2027.