Rare Paediatric Disease designation (RPD) has been granted by the US Food and Drug Administration (FDA) for OPGx-LCA5, an ocular gene therapy developed by Opus Genetics to treat patients with the inherited retinal disease Leber congenital amaurosis (LCA).
OPGx-LCA5 is an adenoassociated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with LCA resulting from biallelic mutations in the LCA5 gene.
LCA5-LCA is a form of earlyonset retinal degeneration that affects approximately one in 1.7 million people in the United States. Currently, there are no approved treatments for individuals with LCA5-related vision loss. OPGx-LCA5 is currently being studied in an open-label, dose-escalation Phase 1/2 clinical trial (NCT05616793) at the University of Pennsylvania. The trial is designed to evaluate the therapy’s safety and preliminary efficacy in 15 patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene.
The FDA grants Rare Paediatric Disease designation to therapeutics intended to treat serious or life-threatening rare diseases that primarily affect individuals under the age of 18. By obtaining this designation, Opus has access to valuable incentives and support from the FDA during its development program. With this designation, 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.
