Ocugen Announces Rare Pediatric Disease Designation Granted for OCU410ST

Ocugen (NASDAQ: OCGN) has received Rare Pediatric Disease Designation (RPDD) from the FDA for OCU410ST, its gene therapy treatment for ABCA4-associated retinopathies, including Stargardt disease. This designation applies to serious diseases affecting children under 18 and fewer than 200,000 people in the U.S. The treatment already holds Orphan Drug designations from both FDA and European Medicines Agency. Approximately 100,000 people in the U.S. and Europe combined are affected by Stargardt disease. If the Priority Review Voucher (PRV) program is reauthorized, Ocugen could receive a voucher worth approximately $100 million. The company plans to begin Phase 2/3 pivotal confirmatory trials soon, targeting a Biologics License Application (BLA) filing in 2027.

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