The FDA has granted its Rare Pediatric Disease designation to Opus Genetics’s OPGx-LCA5 for the treatment of inherited retinal disease LCA5, an early onset retinal degeneration. Currently, no approved treatments exist for people with LCA5-related vision loss.

Studies in LCA5 patients have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation, Opus says. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. It is currently being studied in an open-label, dose-escalation Phase 1/2 clinical trial at the University of Pennsylvania designed to evaluate its safety and preliminary efficacy in 15 patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene.

By obtaining the Rare Pediatric Disease designation, Opus has access to valuable incentives and support from the FDA during its development program. Additionally, OPGx-LCA5 will be eligible to receive a priority review voucher upon approval for any subsequent marketing application that can be sold or transferred to other companies.