Opus Genetics’ emerging LCA5 gene therapy, OPGx-LCA5, has been accepted into the US Food & Drug Administration’s (FDA) new Rare Diseases Evidence Principles (RDEP) process for providing greater speed and predictability for the review of therapies intended for diseases with a significant unmet need affecting fewer than 1,000 people.
LCA5 is a rare inherited retinal disease causing blindness or severe vision loss in childhood.
The RDEP process can allow potential FDA approval based on one adequate and well-controlled study plus additional robust confirmatory evidence, which may include: strong mechanistic or biomarker evidence; evidence from relevant non-clinical models; clinical pharmacodynamic data; and case reports, expanded access data, or natural history studies.
OPGx-LCA5 is currently being evaluated in a Phase 1/2 clinical trial at the University of Pennsylvania. Vision gains and positive safety results for the treatment for adult and pediatric trial participants have been reported. Enrollment for an adaptive Phase 3 clinical trial is ongoing. OPGx-LCA5 has also received Rare Pediatric Disease, Orphan Drug, Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA.
Opus Genetics is a Hope in Focus partner.