Ocugen, Inc. has announced that the FDA has granted Rare Pediatric Disease Designation (RPDD) to OCU410ST for the treatment of ABCA4-associated retinopathy, including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3. Previously, OCU410ST received Orphan Drug designations for the treatment of ABCA4-associated retinopathy from the FDA and European Medicines Agency, the company said in a press release.
“This latest designation for OCU410ST reaffirms the urgency of providing a therapeutic option to Stargardt patients who have no FDA-approved treatment available,” said chairman, CEO, and co-founder of Ocugen, Shankar Musunuri, in the press 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.”
With this designation for OCU410ST, Ocugen said it may be awarded a Priority Review Voucher (PRV) if the PRV program is reauthorized by the US Congress. The program is designed to incentivize drug development for serious rare pediatric diseases.
Ocugen said it is committed to advancing OCU410ST through clinical development and plans to initiate the Phase 2/3 pivotal confirmatory trial in the next few weeks, with a target Biologics License Application (BLA) filing in 2027.
from Retinal Physician
