Ocugen’s Gene Therapies For Vision Loss Receive EMA Approval For OCU410 And OCU410ST, Advancing Toward Late-Stage Trials

Ocugen, Inc., a biotechnology leader specialising in gene therapies for blindness-related diseases, has received a positive opinion from the European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT) for classifying OCU410 and OCU410ST as Advanced Therapy Medicinal Products (ATMPs). OCU410 is being developed to treat geographic atrophy (GA), a severe form of dry age-related macular degeneration (dAMD), while OCU410ST targets Stargardt disease caused by ABCA4-related retinopathies. The ATMP classification is a significant regulatory milestone facilitating accelerated review and enhanced engagement with the EMA.

“Receiving ATMP classification for OCU410 and OCU410ST is a critical step to potentially address these severely unmet medical needs very shortly. Dosing of Phase 2 in the ongoing OCU410 ArMaDa clinical trial is complete, and we are on track to initiate the Phase 3 clinical trial next year to pursue potential Marketing Authorization Application (MAA) and Biologics License Application (BLA) filings in 2028. Last week, the U.S. Food and Drug Administration (FDA) endorsed Ocugen’s plan to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST, which can be the basis of BLA and potential MAA submissions in 2027,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen.

GA affects 2-3 million people in the U.S. and Europe, with current therapies requiring frequent dosing and none yet approved in Europe. Stargardt disease has no approved treatments globally, affecting 100,000 individuals in these regions. Preliminary data from the OCU410 Phase 1 trial in GA patients showed a 44% slower lesion growth rate and a clinically meaningful improvement in vision. Similarly, the Phase 1 OCU410ST GARDian trial demonstrated a 52% reduction in lesion growth and statistically significant vision improvement, supporting the therapy’s potential effectiveness.

Dr. Huma Qamar, Chief Medical Officer at Ocugen, commented, “The novel modifier gene in OCU410 and OCU410ST targets all four pathways linked with dAMD and Stargardt and is delivered through a single subretinal injection as a one-and-done treatment. We are very pleased with the structural and functional outcomes both candidates demonstrated, along with a stellar safety profile.”

Both OCU410 and OCU410ST use an adeno-associated virus (AAV) platform to deliver the RORA gene, which plays a key role in reducing oxidative stress, inflammation, and lipofuscin deposits while modulating the complement system. These promising results reinforce Ocugen’s commitment to advancing gene therapies for blinding diseases, with plans to launch a Phase 2/3 pivotal trial for OCU410ST by mid-2025.