MALVERN, PA — The U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy designation to Ocugen Inc.’s experimental OCU410 gene therapy for geographic atrophy, giving the blindness treatment access to a faster regulatory process as the company prepares a Phase 3 trial.
Ocugen Inc. (NASDAQ: OCGN) is developing OCU410 as a one-time treatment for geographic atrophy caused by dry age-related macular degeneration. Existing approved therapies require recurring injections into the eye.
The RMAT designation provides benefits available under the FDA’s Fast Track and Breakthrough Therapy programs, including more frequent agency interaction, potential rolling review of a biologics license application and eligibility for accelerated approval or Priority Review when applicable.
The designation does not establish that OCU410 is safe or effective or guarantee eventual approval.
Ocugen reported that the FDA based its decision on Phase 2 data showing preliminary clinical benefit and a favorable safety profile. The company reported no serious adverse events related to the therapy.
“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,” Chairman and Chief Executive Officer Shankar Musunuri stated.
Ocugen reached agreement with the FDA in early July on the design of a Phase 3 registrational trial. The company expects to begin the study during the third quarter of 2026 and anticipates filing a biologics license application in 2028.
OCU410 is delivered through a subretinal injection using an AAV5 viral vector. The therapy carries the RORA gene, which regulates biological pathways associated with oxidative stress, inflammation, complement activity and lipid metabolism in the retina.
Geographic atrophy is an advanced form of dry age-related macular degeneration that progressively damages the macula and causes irreversible central vision loss.
Ocugen estimated that 2 million to 3 million people in the United States and Europe have geographic atrophy. The company also cited estimates that dry age-related macular degeneration affects about 10 million Americans and more than 266 million people worldwide.
OCU410 has also received Advanced Therapy Medicinal Product classification from the European Medicines Agency.
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