FDA Grants RMAT Designation to RPESC-RPE-4W Transplantation for Dry AMD

Credit: US Food and Drug Administration

The US Food and Drug Administration (FDA) awarded Regenerative Medicine Advanced Therapy (RMAT) designation to RPESC-RPE-4W transplantation for the treatment of patients with dry age-related macular degeneration (AMD).¹

Announced by Luxa Biotechnology, on February 18, 2025, the company will present early safety, tolerability, and efficacy data of RPESC-RPE-4W transplantation in the first 6 patients with dry AMD patients at the 77th Annual Wills Eye Conference on March 6, 2025.

“This RMAT designation was granted following the FDA’s review of our compelling preliminary Phase 1 data and underscores the transformative potential of RPESC-RPE-4W in addressing the unmet needs of dry AMD patients who have lost or are losing their vision,” said Keith Dionne, PhD, chief executive officer of Luxa Biotechnology, in a statement.

Nearly 200 million people are affected by AMD, with estimates expected to increase to 288 million in the next two decades. It is the leading reason for adult blindness in the Western world, including 20 million Americans, with 80% classified as the dry form. Vision loss is gradual in the early or intermediate dry AMD state, but can progress to the severe, advanced stage of geographic atrophy (GA).²

Under development by Luxa Biotechnology, RPESC-RPE-4W is a cell product derived from adult retinal pigment epithelial (RPE) stem cells (RPESC) generating RPE cell progeny (RPESC-RPE). Transplantation of progenitor-stage RPESC-RPE cells after 4 weeks of differentiation (RPESC-RPE-4W) can replace native RPE cells lost during AMD progression.¹

At the time of the RMAT designation, Luxa Biotechnology announced the RPESC program had initiated a Phase 1/2a clinical trial for RPESC-RPE-4W transplantation in dry AMD.

The FDA’s RMAT designation accelerates the development and review of promising regenerative medicine therapies treating serious life-threatening diseases. Established under the 21st Century Cures Act, these therapies must have demonstrated preliminary clinical evidence of the potential to address unmet medical needs.

With this RMAT designation, Luxa Biotechnology will receive significant benefits for the clinical review process, including early interaction with FDA leadership to guide clinical development, the potential for an accelerated approach, and Priority Review of the product’s Biologics License application (BLA).

“We look forward to collaborating closely with the FDA to bring this potential paradigm-changing treatment to patients as efficiently as possible, restoring vision, and improving the quality of life for millions,” Dionne added.

References

1. _{Luxa Biotechnology announces FDA Regenerative Medicine Advanced Therapy (RMAT) designation granted to RPESC-RPE-4W transplantation for the treatment of dry age-related macular degeneration. Business Wire. February 18, 2025. Accessed February 18, 2025. https://www.businesswire.com/news/home/20250218104018/en/Luxa-Biotechnology-Announces-FDA-Regenerative-Medicine-Advanced-Therapy-RMAT-Designation-Granted-to-RPESC-RPE-4W-Transplantation-for-the-Treatment-of-Dry-Age-Related-Macular-Degeneration.}
2. _{Ruia S, Kaufman EJ. Macular Degeneration. [Updated 2023 Jul 31]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK560778/}