Ocugen, Inc. announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has provided positive scientific advice regarding the approval pathway for OCU410ST. The EMA reviewed the study design, endpoints, and planned statistical analysis of the ongoing pivotal confirmatory OCU410ST Phase 2/3 GARDian3 clinical trial for Stargardt disease. This positive opinion confirms the acceptability of a single U.S.-based trial for submission of a Marketing Authorization Application (MAA).
This favorable outcome from the EMA is based on the safety and tolerability demonstrated by OCU410ST in the Phase 1 GARDian trial. That trial showed 48% slower lesion growth and a statistically significant (p=0.031) and clinically meaningful improvement of nearly a 2-line/9-letter gain in best corrected visual acuity (BCVA) at 12-month follow-up in evaluable treated eyes compared to untreated eyes. The EMA's endorsement is expected to significantly reduce the time and cost required to gain marketing authorization in the EU.
The Phase 2/3 study will enroll 51 participants, with data from the one-year follow-up supporting both the company’s planned Biologics License Application (BLA) in the U.S. and the MAA in the EU. This alignment with the EMA follows other important milestones for OCU410ST, including Rare Pediatric Disease Designation in May, IND clearance in June, and the first patient dosing in July. Ocugen remains on track for a BLA filing in the first half of 2027, aligning with its goal of three BLAs in the next three years.
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