Ocugen, Inc. announced that it has reached alignment with the U.S. Food and Drug Administration (FDA) to proceed with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST. This modifier gene therapy candidate is being developed for Stargardt disease. This alignment is a significant development, as a positive outcome from this trial can serve as the basis for a Biologics License Application (BLA) submission.
The FDA's decision accelerates the regulatory pathway for OCU410ST by two to three years, enabling Ocugen to initiate the pivotal confirmatory trial in the coming months. The company is now preparing for a potential BLA filing by 2027. Stargardt disease affects approximately 100,000 people in the U.S. and Europe combined, with no currently approved treatments.
Data from the Phase 1 GARDian trial for OCU410ST demonstrated 52% slower lesion growth and a statistically significant (p=0.02) 2-line (10-letter) improvement in best-corrected visual acuity (BCVA). The planned Phase 2/3 clinical trial will randomize 51 subjects, with a primary endpoint of change in atrophic lesion size. This expedited pathway offers new hope for patients suffering from this debilitating inherited retinal disease.
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