Opus Genetics’ LCA5 Gene Therapy Accepted into FDA Rare Disease Evidence Program
The FDA has accepted Opus Genetics’ OPGx-LCA5 gene therapy into its Rare Disease Evidence Principles program, granting early collaboration on Phase 3 trial design and regulatory strategy. Data show AAV8-delivered treatment produced large pediatric cone-mediated vision gains and durable adult improvements to 18 months with no ocular safety issues.
1. FDA Acceptance into RDEP Program
Opus Genetics’ OPGx-LCA5 gene therapy for Leber congenital amaurosis type 5 has been accepted into the FDA’s Rare Disease Evidence Principles program. This enrollment provides structured early interaction on regulatory strategy, trial design, evidence generation and the potential use of a single pivotal study for approval.
2. Clinical Trial Results
In its ongoing Phase 1/2 trial, the AAV8-based therapy produced substantial pediatric improvements in cone-mediated vision and durable adult sensitivity gains lasting up to 18 months. The treatment remains well tolerated, with no ocular serious adverse events or dose-limiting toxicities reported across cohorts.
3. Future Development and Strategy
Opus plans to leverage RDEP guidance alongside its existing RMAT designation to finalize a pivotal Phase 3 study protocol. The company will focus on trial endpoints tailored to ultra-rare populations, aiming to support approval for a disease affecting fewer than 1,000 U.S. patients.