Ocugen Receives FDA RMAT Designation for OCU410 in Geographic Atrophy
Thursday, July 30, 2026
Ocugen has received Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration (FDA) for its investigational gene therapy, OCU410, for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). The designation supports the accelerated development and regulatory review of the therapy for a condition with significant unmet medical need.
The RMAT designation is based on Phase 2 clinical data showing clinically meaningful efficacy and a favourable safety profile. No serious treatment-related adverse events were reported during the study. The FDA concluded that OCU410 met the requirements for RMAT status by demonstrating the potential to address a serious disease through preliminary clinical evidence.
Earlier in July 2026, Ocugen reached agreement with the FDA on the design of its Phase 3 registrational trial for OCU410. The company plans to begin the study in the third quarter of 2026 and expects to submit a Biologics License Application (BLA) in 2028.
OCU410 is an investigational AAV5-based gene therapy delivered through a subretinal injection. It is designed as a one-time treatment by delivering the RORA gene, which regulates several biological pathways linked to retinal health, including oxidative stress, inflammation, complement activity and lipid metabolism. The therapy aims to provide a long-lasting treatment option for patients with GA caused by dAMD.
Geographic atrophy is an advanced form of dry age-related macular degeneration that causes progressive and irreversible loss of central vision. Although treatment options have recently expanded, existing therapies require ongoing intravitreal injections, highlighting the need for more durable approaches. OCU410 has also received Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency, supporting its continued global development.
Source: globenewswire.com
