FDA grants RMAT designation to Ocugen’s OCU400 for treatment of retinitis pigmentosa associated with RHO mutations


According to the company, the Regenerative Medicine Advanced Therapy designation will help expedite the development of new regenerative medicines.

©Daniele Depascale – stock.adobe.com

Image credit: ©Daniele Depascale – stock.adobe.com

Ocugen Inc today announced the FDA has granted RMAT designation to its investigational product OCU400 for the treatment of retinitis pigmentosa (RP) associated 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,” Arun Upadhyay, PhD, chief scientific officer and head of R&D at Ocugen said in a news release. “FDA’s decision also reinforces the sense of urgency to bring a therapeutic option to these patients.”

According to the company, RMAT designation for OCU400 was based on preliminary clinical data supporting the maintenance and improvement of visual acuity and function in RP patients in the OCU400 -101 Phase 1/2 clinical trial as measured by best corrected visual acuity (BCVA), low luminance visual acuity (LLVA), and multi-luminance mobility test (MLMT).1

The company noted in its news release RMAT designation is part of the 21st Century Cures Act. The program was created to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse, or cure a serious condition. Receiving RMAT designation offers sponsor companies all the benefits of the fast track and breakthrough therapy designation programs, including early interactions with the FDA. The company noted it is working with the FDA to finalize the Phase 3 protocol necessary to advance the clinical development for OCU400 to support an application for marketing authorization.1

Current data support the gene-agnostic mechanism of action for OCU400, which suggests that it may be able to provide treatment benefit to a broader group of RP and Leber congenital amaurosis (LCA) patients. Ocugen noted in its news release it will submit additional efficacy and safety data for OCU400 in RP and LCA patients to the FDA in the future to potentially expand this RMAT designation to broader RP and LCA patient populations.

RHO mutations affect more than 10,000 of the 110,000 people in the United States diagnosed with RP. In the latest clinical study update from the Phase 1/2 trial of OCU400, 86% (6/7) of RHO mutation subjects experienced either stabilization or improvement in MLMT scores from baseline, among which 29% (2/7) demonstrated 3 Lux luminance level improvement. There are currently no treatment options available for RP patients with RHO gene mutations.1

OCU400 represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptors (NHRs) that regulate diverse physiological functions, such as homeostasis, reproduction, development, and metabolism to potentially improve retinal health and function.

  1. Ocugen Announces OCU400 Receives Regenerative Medicine Advanced Therapy (RMAT) Designation for Treatment of Retinitis Pigmentosa Associated with RHO Mutations | Ocugen, Inc. Ocugen, Inc. Published 2023. Accessed December 19, 2023. https://ir.ocugen.com/news-releases/news-release-details/ocugen-announces-ocu400-receives-regenerative-medicine-advanced
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