Ocugen’s AAV Therapy OCU410ST Receives U.S. FDA Rare Pediatric Disease Designation for ABCA4-Associated Retinopathies

MALVERN, Pa., May 27, 2025 – Ocugen, Inc. , a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for OCU410ST. This designation is for the treatment of ABCA4-associated retinopathies, encompassing conditions such as Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3. Previously, OCU410ST received Orphan Drug designations for the treatment of ABCA4-associated retinopathies from the FDA and European Medicines Agency, leveraging its AAV delivery.

“This latest designation for OCU410ST reaffirms the urgency of providing a therapeutic option to Stargardt patients who currently have no FDA-approved treatment available,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “This inherited retinal disease often presents itself in childhood—making Stargardt disease a diagnosis that not only affects the patient but impacts the entire family. Our AAV-based approach offers significant promise.”

The FDA grants RPDD for serious and life-threatening diseases that primarily affect children aged 18 years or younger and fewer than 200,000 people in the U.S. Approximately 100,000 individuals in the U.S. and Europe combined are living with Stargardt disease. The development of new AAV therapies like OCU410ST is crucial for these rare pediatric conditions.

With this RPDD, Ocugen may be eligible for a Priority Review Voucher (PRV), provided the PRV program is reauthorized by the U.S. Congress. The PRV program incentivizes drug development for serious rare pediatric diseases. If awarded, a PRV can be redeemed for priority review of another product or sold, typically fetching around $100 million, further supporting innovative AAV gene therapy research.

Ocugen is committed to accelerating the OCU410ST program through clinical development. The Company plans to initiate the Phase 2/3 pivotal confirmatory trial within the next few weeks, targeting a Biologics License Application (BLA) filing in 2027 for this promising AAV-mediated gene therapy.

About OCU410ST

OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR-Related Orphan Receptor A) gene. It represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathophysiological pathways linked to Stargardt disease, such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks. The specific AAV vector used is designed for optimal retinal tropism.

About Stargardt Disease

Stargardt disease is the most common form of inherited macular degeneration, a genetic eye disorder that causes progressive retinal degeneration and vision loss. It is characterized by the degeneration of photoreceptor cells in the macula, the central part of the retina, leading to decreased central vision while typically preserving some peripheral vision. The disease usually develops during childhood or adolescence, though age of onset and progression rates vary. The retinal pigment epithelium (RPE), crucial for photoreceptor support, is also affected in patients with Stargardt disease, highlighting the need for targeted AAV therapies.