USFDAJul 6, 2026, 07:01 AM
Opus Genetics FDA Aligns on OPGx-LCA5 Phase 3 Design; BLA Possible
AI Summary
Opus Genetics announced successful alignment with the FDA on the pivotal Phase 3 study design for OPGx-LCA5, a gene therapy for LCA5-associated inherited retinal disease. The FDA indicated that a Biologics License Application (BLA) may be submitted based on compelling 6-month efficacy data, with 12-month durability data provided during the review process. The company expects to initiate Phase 3 dosing in the fourth quarter of 2026, with 7 of 8 planned participants already enrolled and completing the run-in period. The primary efficacy endpoint is a mean improvement of at least 7 decibels in retinal sensitivity, supported by Phase 1/2 data showing an average improvement of approximately 10.5 dB.
Key Highlights
- FDA aligned on pivotal Phase 3 study design for OPGx-LCA5.
- BLA submission for OPGx-LCA5 possible based on 6-month efficacy data.
- Phase 3 dosing for OPGx-LCA5 expected to initiate in Q4 2026.
- Seven of eight planned Phase 3 participants already enrolled.
- Primary efficacy endpoint is 7 dB mean improvement in retinal sensitivity.
- Phase 1/2 data showed average 10.5 dB improvement in retinal sensitivity.
- OPGx-LCA5 holds Rare Pediatric Disease, Orphan Drug, and RMAT designations.
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