Ocugen, Inc., a biotechnology leader in gene therapies for blindness diseases, has announced that the US FDA has granted Rare Pediatric Disease Designation (RPDD) for its treatment OCU410ST. The designation is given to serious and life-threatening diseases primarily affecting children under 18 years old and fewer than 200,000 people in the US.
The RPDD grants Ocugen priority review for its product if the program is reauthorized by Congress. This can lead to a Priority Review Voucher (PRV) worth approximately $100 million. The treatment OCU410ST uses an AAV delivery platform to deliver the RORA gene, aiming to address Stargardt disease, a genetic eye disorder causing retinal degeneration and vision loss.
Stargardt disease affects around 100,000 people in the US and Europe combined, with most cases developing during childhood. Ocugen plans to initiate its Phase 2/3 pivotal confirmatory trial soon, targeting a Biologics License Application (BLA) filing in 2027. The company believes this designation reaffirms the urgency of providing a treatment option for Stargardt patients who currently have no FDA-approved treatment available.
Source: https://ir.ocugen.com/news-releases/news-release-details/ocugen-announces-rare-pediatric-disease-designation-granted