Opus Genetics announced May 4 that the US FDA had accepted OPGx-LCA5, a gene therapy candidate targeting Leber congenital amaurosis type 5, into the new Rare Disease Evidence Principles (RDEP) program.

The FDA unveiled the RDEP initiative in September 2025. It aims to support the development of therapies for ultra-rare genetic diseases. Approval under RDEP may be based on one adequate and well-controlled study plus robust confirmatory evidence, which may include:

—Strong mechanistic or biomarker evidence.

—Evidence from relevant non-clinical models.

—Clinical pharmacodynamic data.

—Case reports, expanded access data, or natural history studies.

Sponsors may apply to the RDEP process any time prior to the launch of a pivotal trial, the FDA said. To be eligible, investigative therapies must specifically address the genetic defect in question and target a very small, rare disease population or subpopulation (generally fewer than a thousand patients in the US) facing rapid deterioration in function leading to disability or death, for whom no adequate alternative therapies exist.

LCA5 leads to early-onset, progressive vision loss and often results in severe visual impairment or blindness in childhood. Opus, of Durham, North Carolina, said OPGx-LCA5 uses an AAV8 vector to deliver a functional LCA5 gene to the outer retina. Six-month data from the first cohort in a Phase I/II trial of OPGx-LCA5 demonstrated significant visual improvement in all three of the treated adult subjects.

Opus said that, under the RDEP program, it will work closely with the FDA to guide clinical trial design; approaches to generating efficacy in a small patient population; and strategies to support demonstration of clinical benefit.

OPGx-LCA5 has also received rare pediatric disease, orphan drug, and regenerative medicine advanced therapy (RMAT) designations from the FDA.

Drug Farm’s ROSAH Candidate

Drug Farm, of Shanghai, China, announced in March that the FDA had accepted its DF-003 into the RDEP program. DF-003 is an oral alpha kinase 1 (ALPK1) inhibitor targeting the rare genetic disease ROSAH syndrome, which leads to blindness. The candidate also has US orphan drug and fast track status.

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