MALVERN, Pa. — August 13, 2025 — Leads & Copy — Ocugen, Inc. (OCGN) announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has reviewed and accepted the design and analysis of the OCU410ST Phase 2/3 GARDian3 clinical trial for Stargardt disease. The EMA’s positive opinion supports the submission of a Marketing Authorization Application (MAA) based on a single U.S.-based trial.
The EMA’s opinion is based on the safety and tolerability shown in the Phase 1 GARDian trial, where OCU410ST showed a 48% slower lesion growth and a statistically significant improvement in visual acuity at 12 months. The Phase 2/3 study will involve 51 participants, with 34 receiving a one-time injection of OCU410ST and 17 assigned to a control group. The trial’s primary objective is to reduce atrophic lesion size, with secondary endpoints including improvements in visual acuity. Data from the one-year follow-up will support Ocugen’s planned Biologics License Application (BLA) and MAA in the EU.
Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen, said the positive opinion brings the company closer to providing a gene therapy to Stargardt patients in the U.S. and Europe. The company remains on track for a BLA filing in the first half of 2027.
OCU410ST uses an AAV delivery platform for the retinal delivery of the RORA gene, targeting pathways linked to Stargardt disease.
Stargardt disease is a genetic eye disorder that causes retinal degeneration and vision loss.
Contact: Tiffany Hamilton, AVP, Head of Communications, Tiffany.Hamilton@ocugen.com
Source: Ocugen, Inc.
