The U.S. Food and Drug Administration (FDA) recently granted Malvern-based Ocugen’s treatment for geographic atrophy (GA), secondary to dry age-related macular degeneration (dAMD) with a Regenerative Medicine Advanced Therapy (RMAT) Designation.
The RMAT designation is granted to investigational regenerative medicine therapies supported by preliminary clinical evidence indicating the potential to treat, modify, reverse, or cure serious or life-threatening diseases or conditions. The designation expedites the development and review of these therapies.
Ocugen’s investigational product OCU410 is being developed as a one-time gene therapy for patients with GA secondary to dAMD, a disease characterized by progressive degeneration of the macula, leading to irreversible central vision loss.
“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,” Dr. Shankar Musunuri, Ocugen chairman, CEO and co-founder, said. “With an estimated 2 to 3 million people in the U.S. and Europe affected, a number expected to grow as the population ages, geographic atrophy is a leading cause of irreversible blindness in older adults, highlighting the urgent need for new treatment options.”
Phase two clinical data of OCU410 demonstrated clinically meaningful efficacy and a favorable safety profile.