OCU410ST Granted Rare Pediatric Disease Designation by FDA

The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to Ocugen’s OCU410ST for the treatment of ABCA4-associated retinopathies. These include Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.

Building on Previous Regulatory Milestones

OCU410ST had previously received Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA) for the treatment of ABCA4-associated retinopathies. These recognitions emphasize the therapy’s potential in addressing rare retinal diseases that currently lack FDA-approved treatments.

Mechanism of Action: AAV-Mediated RORA Gene Delivery

OCU410ST is based on an adeno-associated virus (AAV) delivery platform specifically designed for retinal gene therapy. It delivers the RORA (RAR-Related Orphan Receptor A) gene, which regulates key pathways involved in Stargardt disease, such as:

• Lipofuscin formation

• Oxidative stress

• Complement activation

• Inflammation

• Cell survival mechanisms

Company Leadership Emphasizes Urgency for Treatment

Shankar Musunuri, PhD, MBA, Chairman, CEO, and Co-Founder of Ocugen, commented in a press release:

“This latest designation for OCU410ST reaffirms the urgency of providing a therapeutic option to Stargardt patients who have no FDA-approved treatment available.”

Implications of the Rare Pediatric Disease Designation

With this designation, Ocugen becomes eligible for a Priority Review Voucher (PRV) — if the PRV program is reauthorized by the U.S. Congress. A PRV can either be used to accelerate FDA review for another product or sold to another pharmaceutical company. Historically, these vouchers sell for $100 million or more.

Next Steps in Clinical Development

Ocugen plans to initiate a Phase 2/3 pivotal confirmatory trial in the coming weeks. The company also aims to submit a Biologics License Application (BLA) for OCU410ST in 2027, marking a significant step toward bringing the therapy to market.