Ocugen Inc., a biotechnology company specializing in gene therapies for blindness diseases, has announced the Rare Pediatric Disease Designation (RPDD) granted by the U.S. Food and Drug Administration (FDA) for its product OCU410ST. This designation is targeted at treating ABCA4-associated retinopathies, which include Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3. The FDA's RPDD is awarded for serious and life-threatening diseases affecting children under 18 and with a prevalence of fewer than 200,000 people in the U.S. Ocugen plans to advance OCU410ST through clinical development, with the initiation of a Phase 2/3 pivotal confirmatory trial expected in the upcoming weeks and a target Biologics License Application $(BLA.AU)$ filing planned for 2027. This designation could potentially lead to a Priority Review Voucher if the program is reauthorized by U.S. Congress.
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