Rare Pediatric Disease Designation Granted for Ocugen's Gene Therapy for the Treatment of Stargardt Disease

Ocugen announced that the FDA has granted Rare Pediatric Disease Designation 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 (EMA).

“This latest designation for OCU410ST reaffirms the urgency of providing a therapeutic option to Stargardt patients who have no FDA-approved treatment available,” Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen, said in a company news release. “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 Rare Pediatric Disease Designation 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 US 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 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.