FDA Clears Amended Ocugen Trial for Stargardt Disease

Ocugen, Inc. received FDA clearance of an amendment to begin a Phase 2/3 pivotal confirmatory trial of its gene therapy candidate OCU410ST for Stargardt disease, a genetic disorder causing retinal degeneration and vision loss. The trial will enroll 51 participants, with 34 receiving a one-time subretinal injection and 17 as untreated controls, focusing on reducing atrophic lesion size and improving visual acuity. Ocugen plans to use one-year follow-up data from the trial to support a Biologics License Application in 2027 and aims to file three BLAs over the next three years. The FDA has previously granted OCU410ST Rare Pediatric Disease and Orphan Drug Designations for ABCA4-associated retinopathies. Ocugen's engagement with the FDA is expected to accelerate its clinical development by two to three years. The company's stock is up 40% year-to-date, reflecting investor confidence.