FDA Grants Ocugen’s OCU400 RMAT Designation for RHO-Linked Retinis Pigmentosa

Ocugen announced that the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to OCU400 for treating retinitis pigmentosa (RP) linked with RHO mutations.

“RMAT designation is a significant accomplishment for the OCU400 clinical development program, as it validates the potential for our game-changing gene therapy approach to fulfill an unmet medical need for people who are facing blindness due to RP,” said Arun Upadhyay, PhD, Chief Scientific Officer and Head of R&D at Ocugen. “FDA’s decision also reinforces the sense of urgency to bring a therapeutic option to these patients.”

The RMAT designation was granted based on preliminary clinical data from the OCU400-101 Phase 1/2 trial, indicating the maintenance and enhancement of visual acuity and function in RP patients. This was assessed through Best Corrected Visual Acuity (BCVA), Low Luminance Visual Acuity (LLVA), and Multi-Luminance Mobility Test (MLMT).

RMAT, established under the 21st Century Cures Act, expedites the review of regenerative medicine therapies for serious conditions. Ocugen is collaborating with the FDA to finalize the Phase 3 protocol for OCU400's clinical development.

The gene-agnostic mechanism of action for OCU400 suggests potential treatment benefits for a broader RP and Leber congenital amaurosis (LCA) patient group. Ocugen plans to submit additional efficacy and safety data to the FDA in the future to broaden RMAT designation. RHO mutations impact over 10,000 of the 110,000 diagnosed RP individuals in the U.S.

In the Phase 1/2 trial update, 86% of RHO mutation subjects experienced stabilization or improvement in MLMT scores. Currently, there are no treatment options for RP patients with RHO gene mutations. OCU400, Ocugen's modifier gene therapy, utilizes Nuclear Hormone Receptors (NHRs) to potentially enhance retinal health and function.