MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for its investigational therapy OCU410ST, aimed at treating ABCA4-associated retinopathies. These include Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3. The therapy previously received Orphan Drug designations from both the FDA and the 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 RPDD is granted for serious, life-threatening diseases primarily affecting children under 18, with fewer than 200,000 cases in the U.S. Stargardt disease alone affects roughly 100,000 individuals across the U.S. and Europe combined. The designation allows Ocugen to qualify for a Priority Review Voucher (PRV) upon FDA approval of OCU410ST, pending reauthorization of the PRV program by Congress. PRVs are designed to encourage drug development for rare pediatric diseases and have an estimated market value of $100 million, as they can be sold or redeemed for priority reviews of other products.
OCU410ST employs an innovative AAV delivery platform to deliver the RORA (RAR-Related Orphan Receptor A) gene, targeting cellular pathways linked to Stargardt disease, such as lipofuscin formation, oxidative stress, inflammation, and cell survival. The company plans to move forward with a Phase 2/3 pivotal confirmatory trial in the coming weeks, with a Biologics License Application (BLA) submission anticipated by 2027.
Stargardt disease, the most common form of inherited macular degeneration, causes central vision loss due to the degeneration of photoreceptors in the macula. While peripheral vision is often maintained, the progressive condition, which commonly arises in childhood or adolescence, leads to degenerative changes in the retinal pigment epithelium (RPE), further impacting vision.
With this milestone, Ocugen is advancing its commitment to address the unmet needs of patients with Stargardt disease and other debilitating retinal conditions.
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