Ocugen, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for OCU410ST. This designation is for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3. This latest designation reaffirms the urgent need for a therapeutic option for Stargardt patients, who currently have no FDA-approved treatment available.
The FDA grants RPDD for serious and life-threatening diseases that primarily affect children aged 18 years or younger and fewer than 200,000 people in the U.S. Stargardt disease often presents in childhood and affects approximately 100,000 people in the U.S. and Europe combined. This designation highlights the significant impact of the disease on pediatric populations.
With this RPDD for OCU410ST, Ocugen may be awarded a Priority Review Voucher (PRV) if the PRV program is reauthorized by the U.S. Congress. A PRV can be redeemed to receive priority review for a different product or sold to another sponsor, typically for about $100 million. Ocugen plans to initiate the Phase 2/3 pivotal confirmatory trial for OCU410ST in the next few weeks, targeting a Biologics License Application (BLA) filing in 2027.
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