, 2025-05-07 19:28:00
Key takeaways:
- Regenerative medicine advanced therapy designation expedites the development and review of therapies.
- The designation was supported by early clinical data from a phase 1/2 trial.
Editor’s note: This is a developing news story. Please check back soon for updates.
The FDA granted regenerative medicine advanced therapy designation to OPGx-LCA5 for the treatment of Leber congenital amaurosis due to genetic variations in the LCA5 gene, according to a press release from Opus Genetics.
The gene therapy is designed to deliver a functional LCA5 gene to the outer retina using an adeno-associated virus 8 vector. Early clinical evidence from an ongoing phase 1/2 open-label dose-escalation trial investigating the safety and potential efficacy of OPGx-LCA5 served as the basis for the regenerative medicine advanced therapy (RMAT) designation.
RMAT designation, which expedites the development and review of therapies, provides sponsors “guidance on efficient development and manufacturing and the opportunity to discuss surrogate endpoints to support accelerated approval,” the release said.
“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,” George Magrath, MD, CEO of Opus Genetics, said in the release. “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.”
As Healio previously reported, the FDA granted rare pediatric disease designation to OPGx-LCA5 in August 2024.
