Stargardt Disease Gene Therapy: FDA Designation
- The FDA has granted rare pediatric disease designation to Ocugen's OCU410ST, a gene therapy intended for ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3,...
- this designation may allow Ocugen to receive a priority review voucher if Congress reauthorizes the program.
- The FDA and European Medicines Agency previously granted OCU410ST orphan drug designations for the same retinopathies.
Ocugen’s OCU410ST gene therapy, designed for ABCA4-associated retinopathies like Stargardt disease, just secured a Rare Pediatric Disease Designation from the FDA. This is a major win, potentially paving the way for a priority review voucher—a significant development for patients without existing FDA-approved treatments. The company is set to initiate a phase 2/3 pivotal trial in the coming weeks, with a biologics license request planned for 2027. This designation from the FDA underscores the pressing need for a Stargardt disease treatment and offers a beacon of hope. News Directory 3 is following the progress, reporting on the breakthroughs. Eager to learn more about the forthcoming trial results?
OcugenS OCU410ST Gene Therapy Receives FDA Rare Pediatric Disease Designation
Updated May 28, 2025
The FDA has granted rare pediatric disease designation to Ocugen’s OCU410ST, a gene therapy intended for ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3, Ocugen announced.
this designation may allow Ocugen to receive a priority review voucher if Congress reauthorizes the program. The voucher could expedite review of another product or be sold.
The FDA and European Medicines Agency previously granted OCU410ST orphan drug designations for the same retinopathies.
Shankar Musunuri, PhD, MBA, chairman, CEO, and co-founder of Ocugen, said the designation underscores the urgency of providing a therapeutic option for Stargardt patients, for whom there is no FDA-approved treatment. He added that Stargardt disease frequently enough presents in childhood, impacting both the patient and their family.
Ocugen plans to begin a phase 2/3 pivotal confirmatory trial for the OCU410ST gene therapy in the coming weeks. The company anticipates filing a biologics license application in 2027.
“This latest designation for OCU410ST reaffirms the urgency of providing a therapeutic option to Stargardt patients who have no FDA-approved treatment available,”
what’s next
Ocugen is set to initiate a phase 2/3 trial soon, with hopes of filing for a biologics license application in 2027, marking a critically important step toward a potential treatment for inherited retinal diseases.