FDA Accepts Opus Genetics Experimental Gene Therapy into Rare Disease Evidence Principles Program

Rare Daily Staff

The Food and Drug Administration has accepted Opus Genetics’ experimental gene therapy for a rare inherited eye disease into a new agency program meant to speed the development of treatments for ultra-rare conditions.

The company said the move could help shape the next phase of testing for a therapy aimed at children and adults with severe vision loss.

The treatment, called OPGx-LCA5, is being developed for Leber congenital amaurosis type 5, a disorder caused by mutations in the LCA5 gene. The disease can cause major vision problems early in life, and there are no approved treatments specifically for it.

The FDA’s Rare Disease Evidence Principles program is designed for very small patient populations, generally fewer than 1,000 people in the United States. It gives drug developers and regulators a framework to work together early on clinical trial design and the evidence needed for possible approval.

In a statement, Opus Genetics chief executive Dr. George Magrath said the FDA acceptance is an important part of the company’s regulatory plan for a planned pivotal Phase 3 study. He said the program could help create a more efficient development path for a disease that is both rare and severe.

Opus said OPGx-LCA5 is being studied in a phase 1/2 trial at the University of Pennsylvania. The company said earlier data in children showed large gains in cone-mediated vision, while adult participants showed durable improvements in visual function lasting up to 18 months, with no ocular serious adverse events or dose-limiting toxicities reported.

The program has already received other FDA designations, including Rare Pediatric Disease, Orphan Drug and Regenerative Medicine Advanced Therapy status.