Opus Genetics recently announced that its ocular gene therapy OPGx-LCA5 has received the rare pediatric disease (RPD) designation from the US Food and Drug Administration (FDA) for the treatment of a form of Leber congenital amaurosis (LCA) that results from biallelic mutations in the LCA5 gene.

OPGx-LCA5 is an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina. OPGx-LCA5 is currently being studied in an open-label, dose-escalation phase 1/2 clinical trial (NCT05616793) 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 LCA5gene.

The FDA grants RPD designation to therapeutics intended to treat serious or life-threatening rare diseases that primarily affect individuals under the age of 18. The RPD program aims to incentivize drug development for these unusual conditions. Upon FDA approval of OPGx-LCA5, the company will receive a priority review voucher that can be used to expedite the review of another therapeutic or sold to another company.