Opus Genetics, Inc. a clinical-stage ophthalmic biotechnology company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and other ophthalmic disorders, announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OPGx-LCA5, its investigational gene therapy for the treatment of Leber Congenital Amaurosis (LCA) due to genetic variations in the LCA5 gene.
The RMAT designation for OPGx-LCA5 is based on early clinical evidence from Opus’s ongoing Phase 1/2 open-label, dose-escalation trial, which is evaluating the safety and potential efficacy of OPGx-LCA5 in patients with severe vision loss due to confirmed mutations in the LCA5 gene. The FDA’s decision indicates recognition of the strength of this initial data and the unmet need in this patient population.
“The FDA’s decision to grant RMAT designation to OPGx-LCA5 is a major milestone for the LCA5 patient community and a strong validation of our early clinical data,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “We’re encouraged by the potential of OPGx-LCA5 to meaningfully impact patients living with this ultra-rare and debilitating form of inherited blindness, and we look forward to continued collaboration with the FDA to accelerate its development.”
In addition to the RMAT designation, Opus has been invited to participate in the FDA’s Initial Comprehensive Multidisciplinary RMAT Meeting to support Opus’ development and manufacturing strategy. Lastly, Opus has been invited to join the FDA’s Chemistry, Manufacturing and Controls (CMC) Development and Readiness Pilot (CDRP) program, which provides additional guidance for accelerating CMC development of products under an investigational new drug application.
The RMAT designation program offers the potential for expedited development and review of regenerative medicine therapies that demonstrate the potential to address serious or life-threatening diseases based on preliminary clinical evidence. The designation provides sponsors with early interactions with the FDA, guidance on efficient development and manufacturing, and the opportunity to discuss surrogate endpoints to support accelerated approval.