Ocugen’s OCU410ST gene therapy lands a rare badge of honor in the fight against IRDs.
The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to Ocugen’s (Pennsylvania, USA) gene therapy candidate OCU410ST for ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19 and cone-rod dystrophy 3. In short, the treatment just earned a place on a very short and serious list.
Ocugen announces the news on its LinkedIn page.
This adds another feather to the cap of OCU410ST, which has already secured Orphan Drug status from both 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, Ocugen’s chairman, CEO and co-founder, in a 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.”
READ MORE: FDA Grants Breakthrough Therapy Designation to Belite Bio’s Tinlarebant for Stargardt Disease
What RPDD means and why it matters
The FDA’s RPDD is reserved for serious and life-threatening diseases that primarily affect children under 18 and fewer than 200,000 people in the U.S. An estimated 100,000 individuals in the United States and Europe are living with Stargardt disease, according to Ocugen.
The designation could come with a rare perk: a Priority Review Voucher (PRV). If Congress reauthorizes the PRV program (which it has so far let sunset in late 2024), Ocugen could use the voucher to speed up the FDA review of another product—or sell it. Historically PRVs have fetched upwards of $100 million.1
READ MORE: One Day, Three IRD Gene Therapy Triumphs
The therapy and the target
OCU410ST is delivered via AAV vector and introduces the RORA (RAR-Related Orphan Receptor A) gene directly to the retina. The approach targets multiple disease pathways—think lipofuscin buildup, oxidative stress and inflammation—rather than chasing a single gene mutation.
A clinical study investigator and participant speak about their experience with OCUS410ST.
Stargardt disease is the most common inherited macular degeneration. It causes progressive vision loss due to the death of light-sensing cells in the macula. Most patients are diagnosed in childhood or adolescence, though severity and timeline can vary.
READ MORE: The Promise of Gene Therapy in Retinal Disorders
What’s next
Ocugen aims to kick off a Phase II/III pivotal confirmatory trial in the coming weeks and has circled 2027 for a potential Biologics License Application (BLA) filing.
As for the PRV program? Time may be running out. Unless Congress steps in, only drugs that received RPDD before December 2024 will be eligible—and only until 2026. The clock, as they say, is ticking.1
References
Rare pediatric disease designation and priority review voucher programs. US FDA. Updated September 27, 2024. Available at: https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/rare-pediatric-disease-designation-and-priority-review-voucher-programs#2024update. Accessed on May 28, 2025.
