Ocugen Receives Positive Advice from EMA for OCU410ST Clinical Trial

Ocugen has received positive advice from the European Medicines Agency for the acceptability of OCU410ST in its ongoing Phase 2/3 GARDian3 clinical trial for Stargardt disease. The trial aims to evaluate the reduction in atrophic lesion size, with key secondary endpoints including improvements in visual acuity. The EMA's opinion is expected to reduce the time and cost to gain marketing authorization in the EU.

Malvern, Pa., Aug. 13, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (NASDAQ: OCGN), a leading biotechnology company focused on gene therapies for blindness diseases, has received positive scientific advice from the European Medicines Agency (EMA) regarding the acceptability of its OCU410ST gene therapy in the ongoing Phase 2/3 GARDian3 clinical trial for Stargardt disease. The EMA's Committee for Medicinal Products for Human Use (CHMP) has accepted a single U.S.-based trial for the Marketing Authorization Application (MAA) submission, significantly streamlining the regulatory pathway for the EU market.

The GARDian3 trial, which aims to evaluate the reduction in atrophic lesion size, will enroll 51 participants, with 34 receiving OCU410ST treatment and 17 serving as controls. The therapy has shown promising Phase 1 results, including a 48% slower lesion growth rate and a statistically significant 2-line/9-letter gain in visual acuity (p=0.031) at 12 months compared to untreated eyes. The trial employs an adaptive design with an interim analysis of 24 subjects at 8 months, focusing on reducing lesion size and improving visual acuity as key endpoints.

The EMA's positive opinion is expected to reduce the time and cost associated with gaining marketing authorization in the EU. This regulatory advancement follows several recent milestones for the OCU410ST program, including Rare Pediatric Disease Designation (RPDD) in May, IND clearance in June, and first patient dosing in July. Ocugen plans to complete enrollment by the first quarter of 2026 and submit a Biologics License Application (BLA) in the first half of 2027.

Stargardt disease is a genetic eye disorder that affects approximately 100,000 patients in the U.S. and Europe combined, with no currently approved treatments. OCU410ST represents a one-time subretinal injection approach, offering a transformative treatment modality for these patients. The company's strategic commitment to advancing multiple candidates through its pipeline simultaneously is evident in its goal of filing three BLAs within the next three years.

References:
[1] https://ir.ocugen.com/news-releases/news-release-details/ocugen-inc-announces-positive-scientific-advice-european-0
[2] https://www.stocktitan.net/news/OCGN/ocugen-inc-announces-positive-scientific-advice-from-the-european-ihrqhaqewa8p.html