FDA grants RMAT designation to Ocugen's OCU410 for dry AMD

Ocugen, Inc. (NASDAQ: OCGN) announced that the U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OCU410, its investigational gene therapy for geographic atrophy secondary to dry age-related macular degeneration.

The designation was supported by Phase 2 clinical data showing efficacy and a safety profile with no reported serious adverse events related to the drug. The FDA determined OCU410 met RMAT criteria by providing preliminary clinical evidence of potential to address a serious condition with significant unmet medical need.

RMAT designation makes OCU410 eligible for benefits associated with the Fast Track and Breakthrough Therapy programs, including increased FDA interactions, eligibility for rolling Biologics License Application (BLA) review, and potential for accelerated approval and Priority Review.

OCU410 is an AAV5-based gene therapy administered via subretinal injection that delivers RORA, a nuclear receptor involved in retinal homeostasis. Ocugen is developing it as a one-time treatment for geographic atrophy, in contrast to currently available therapies that require chronic intravitreal injections.

In early July 2026, Ocugen reached alignment with the FDA on the design of a Phase 3 registrational trial for OCU410, with study initiation expected in the third quarter of 2026 and a BLA filing anticipated in 2028.

"RMAT designation for OCU410 is a significant accomplishment that recognizes both the potential of our 'one treatment for life' novel gene therapy platform and the substantial unmet medical need for geographic atrophy," said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen.

Geographic atrophy affects an estimated 2 to 3 million people in the U.S. and Europe. Dry age-related macular degeneration affects approximately 10 million Americans and more than 266 million people worldwide, accounting for 85 to 90 percent of all AMD cases. OCU410 has also received Advanced Therapy Medicinal Product classification from the European Medicines Agency.

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