Opus Genetics announces FDA acceptance of OPGx-LCA5 into RDEP program

Opus Genetics (IRD) announced that its investigational LCA5 gene therapy program, OPGx-LCA5, has been accepted into the FDA Rare Disease Evidence Principles, or RDEP, program. OPGx-LCA5 is a potential gene therapy for Leber congenital amaurosis type 5, a rare inherited retinal disease caused by mutations in the LCA5 gene. “RDEP eligibility represents an important element of our regulatory strategy as we seek alignment with the FDA on our pivotal Phase 3 program for OPGx-LCA5,” said George Magrath, CEO, Opus Genetics. “Given the rarity and severity of this disease, early engagement with the FDA alongside our RMAT designation will help inform a more efficient and streamlined development pathway. We look forward to collaborating with the FDA as we pursue a potential treatment option for patients affected by this devastating inherited retinal disease.”