Opus Genetics announced in a press release that its investigational gene therapy program, OPGx-LCA5, for Leber congenital amaurosis type 5 (LCA5), has been accepted into the US Food and Drug Administration (FDA) Rare Disease Evidence Principles (RDEP) program.

RDEP is a new FDA initiative intended to support the development of therapies for ultra-rare genetic diseases typically affecting fewer than 1,000 patients in the US. The program enables early and ongoing collaboration between the FDA and sponsors to align on regulatory strategy, clinical trial design, and innovative approaches to generating evidence needed to support potential approval.

As part of the RDEP program, the FDA will work closely with Opus Genetics to guide the ongoing development of OPGx-LCA5, including considerations for clinical trial design, approaches to generating efficacy in a small patient population, and strategies to support demonstration of clinical benefit. The program also provides a framework for evaluating substantial evidence of effectiveness, including the potential use of a single adequate and well-controlled study supported by confirmatory evidence, the company said in the press release.  

“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, MD, chief executive officer of 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.” RP