Press release
Ocugen Announces Rare Pediatric Disease Designation Granted for OCU410ST—Modifier Gene Therapy for the Treatment of Stargardt Disease
MALVERN, Pa., May 27, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for
About this update from Ocugen, Inc.
[{"type":"text","content":"MALVERN, Pa., May 27, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the United States Food and Drug Administration (U.S. FDA) has granted Rare Pediatric Disease Designation (RPDD) for OCU410ST for the treatment of ABCA4-associated retinopathies including 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. “This latest designation for OCU410ST reaffirms the urgency of providing a therapeutic option to Stargardt patients who have no FDA-approved treatment available,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “This inherited retinal disease presents itself most often in childhood—making Stargardt disease a diagnosis that not only affects the patient but impacts the entire family.” The FDA grants RPDD for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the U.S. There are approximately 100,000 people in the U.S. and Europe combined living with Stargardt disease. With this designation for OCU410ST, Ocugen may be awarded a Priority Review Voucher (PRV), if the PRV program is reauthorized by the U.S. Congress. The PRV program is designed to incentivize drug development for serious rare pediatric diseases. If awarded, a PRV can be redeemed to receive priority review for a different product or sold to another sponsor and typically sells for about $100 million. Ocugen is committed to advancing the OCU410ST program through clinical development and plans to initiate the Phase 2/3 pivotal confirmatory trial in the next few weeks with a target Biologics License Application (BLA) filing in 2027. About OCU410STOCU410ST 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. About Stargardt DiseaseStargardt dise...