Ocugen, Inc. announced today that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) amendment to initiate a Phase 2/3 pivotal confirmatory trial of OCU410ST. This modifier gene therapy candidate is being developed for all Stargardt disease (ABCA4-associated retinopathies). This clearance is a result of highly productive engagement with the FDA’s Center for Biologics Evaluation and Research (CBER).
The FDA previously granted Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation for OCU410ST, underscoring the urgent need for treatment options. This clearance expedites the clinical development of OCU410ST by two to three years, moving the company closer to potentially providing an innovative gene therapy to patients who currently have no available treatments. The Phase 2/3 clinical trial will enroll 51 participants diagnosed with Stargardt disease.
Positive data from the Phase 1 GARDian trial for OCU410ST demonstrated 48% slower lesion growth at 12 months and a 2-line/10-letter gain in best corrected visual acuity (BCVA). The primary objective of the Phase 2/3 trial is to evaluate the reduction in atrophic lesion size, with one-year follow-up data supporting the company’s Biologics License Application (BLA). This represents Ocugen’s second late-stage clinical program, with a BLA submission planned for 2027.
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