Ocugen's OCU410ST trial for Stargardt disease meets EMA's acceptability criteria.

Ocugen's OCU410ST Phase 2/3 GARDian3 clinical trial for Stargardt disease has been reviewed by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP). The CHMP has accepted a single U.S.-based trial for a Marketing Authorization Application (MAA), based on the safety and tolerability demonstrated in the Phase 1 GARDian trial. The Phase 2/3 study aims to enroll 51 participants and evaluate the efficacy of OCU410ST in slowing lesion growth and improving visual acuity.

Ocugen, Inc. (NASDAQ: OCGN), a pioneering biotechnology company focused on gene therapies for blindness diseases, has received positive scientific advice from the European Medicines Agency (EMA) regarding the approval pathway for OCU410ST, its gene therapy treatment for Stargardt disease. The EMA's Committee for Medicinal Products for Human Use (CHMP) has accepted a single U.S.-based trial for Marketing Authorization Application (MAA) submission [1].

The Phase 2/3 GARDian3 clinical trial, which aims to enroll 51 participants, will evaluate the efficacy of OCU410ST in slowing lesion growth and improving visual acuity. Of the 51 participants, 34 will receive a one-time subretinal injection of OCU410ST, while 17 will serve as controls. The trial employs a sophisticated adaptive design with an interim analysis of 24 subjects at 8 months. The primary endpoint focuses on reducing atrophic lesion size, with visual acuity measures as key secondary endpoints [1].

The EMA's decision is based on compelling Phase 1 data showing 48% slower lesion growth and a statistically significant 9-letter gain in visual acuity at 12 months compared to untreated eyes (p=0.031). These improvements represent clinically meaningful benefits for patients with this progressive retinal degeneration disorder [1].

Ocugen plans to complete enrollment by Q1 2026 and submit a Biologics License Application (BLA) in H1 2027. The treatment targets approximately 100,000 Stargardt patients in the U.S. and Europe combined, who currently have no approved treatment options [1].

This favorable opinion from the EMA could significantly reduce the time and cost to gain marketing authorization in the EU. Ocugen's recent milestones, including Rare Pediatric Disease Designation (RPDD), IND clearance, and first patient dosing, further demonstrate the company's momentum towards its goal of three BLAs in the next three years [1].

References:
[1] https://www.stocktitan.net/news/OCGN/ocugen-inc-announces-positive-scientific-advice-from-the-european-ihrqhaqewa8p.html