Ocugen Gains FDA Rare Pediatric Disease Designation for OCU410ST To Treat Stargardt And Related Retinopathies

Ocugen, Inc. has received Rare Pediatric Disease Designation (RPDD) from the U.S. FDA for its gene therapy candidate OCU410ST, which targets ABCA4-associated retinopathies such as Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3. This designation follows earlier Orphan Drug designations that the FDA and the European Medicines Agency granted. RPDD is awarded to therapies for severe, life-threatening pediatric conditions affecting fewer than 200,000 patients in the U.S. Stargardt disease alone affects about 100,000 people in the U.S. and Europe combined. 

“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 affects the patient and the entire family.”

If the U.S. Congress reauthorises the Priority Review Voucher (PRV) program, Ocugen could be eligible for a PRV upon FDA approval of OCU410ST. This highly valuable asset can be redeemed for expedited review or sold, often for around $100 million. Ocugen is advancing OCU410ST toward a Phase 2/3 pivotal trial, expected to begin in the coming weeks, to file a Biologics License Application (BLA) in 2027.