FDA Approves OCU400 Expanded Access Program for Retinitis Pigmentosa

Lejla Vajzovic, MD

Credit: Duke University Eye Center

The US Food and Drug Administration (FDA) has approved an expanded access program (EAP) for the treatment of adult patients with retinitis pigmentosa (RP) with OCU400, a modifier gene therapy product candidate.¹

Announced by Ocugen on August 5, 2024, the EAP is open for patients with early, intermediate to advanced RP, with at least minimal retinal preservation, who may benefit from the drug’s mechanism of action before Biologics License Application (BLA) approval.

Ocugen is also actively dosing patients with early to late-stage retinitis pigmentosa with OCU400 in the one-year Phase 3 liMeliGhT clinical trial.

“Retinitis pigmentosa patients with mutations in multiple genes currently have no therapeutic options,” said Lejla Vajzovic, MD, associate professor of ophthalmology, Duke University Eye Center, and Retina Scientific Advisory Board Chair, Ocugen. “As a retinal surgeon, I am encouraged by the therapeutic potential of OCU400 to provide long-term benefit.”

OCU400 is a modifier gene therapy product based on the nuclear hormone receptor (NHR) gene NR2E3 – the gene regulates physiologic functions in the retina, including photoreceptor development and maintenance, metabolism, phototransduction, inflammation, and cell survival.

OCU400’s mechanism of action can potentially reset a dysfunctional gene network to healthy cellular homeostasis for retinal cells in retinitis pigmentosa.

In one-year Phase 1/2 trial results at the American Society of Retina Specialists (ASRS) 42nd Annual Meeting, 89% of participants treated with OCU400 experienced stabilization or improvement in efficacy endpoints, including best-corrected visual acuity (BCVA) and low-luminance visual acuity (LLVA).²

The Phase 3 liMeliGhT trial will enroll a sample size of 150 participants, including 75 with RHO gene mutations and 75 with mutations in other genes.¹ Across each arm, patients will be randomized 2:1 to OCU400 treatment (2.5 x 1010 Ve/eye) and untreated control cohorts, respectively.

This newly approved EAP is a US-only protocol for eligible adults ≥18 years with early, intermediate to advanced retinitis pigmentosa with at least minimal retinal preservation and patients who participated in the OCU400 Phase 1/2 study who qualify for dosing in the contralateral eye.

The EAP can also include those who failed to meet inclusion criteria in the Phase ½ and Phase 3 trials and patients with retinitis pigmentosa who could benefit from the mechanism of action of OCU400 before BLA approval.

“We are excited to expand our enrollment to include patients representing a diverse array of RP gene mutations,” said Huma Qamar, chief medical officer, Ocugen. “This program reflects our ongoing commitment to develop a safe and effective therapy for retinitis pigmentosa patients who may not have other treatment options.”

Previously, OCU400 received Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. The European Medicines Agency (EMA) has additionally accepted the US-based trial for submission of a Marketing Authorization Application (MAA).

As patient dosing in the Phase 3 clinical trial has started, Ocugen has confirmed that OCU400 remains on track for targeted BLA and MAA approval in 2026.

“With positive Phase ½ study data and an ongoing Phase 3 liMeliGhT clinical trial, we now plan to work with clinicians, patients, and the retinitis pigmentosa community to provide access to OCU400 for eligible patients through our EAP,” said Shankar Musunuri, PhD, MBA, chairman, chief executive officer, and co-founder, Ocugen.

Reference

1. _{Ocugen, Inc.. announces FDA approval of Expanded Access program for patients with retinitis pigmentosa. Ocugen, Inc. August 5, 2024. Accessed August 5, 2024. https://ir.ocugen.com/news-releases/news-release-details/ocugen-inc-announces-fda-approval-expanded-access-program.}
2. _{Iapoce C. OCU400 shows benefit for retinitis pigmentosa in phase 1/2 data. HCP Live. July 20, 2024. Accessed August 5, 2024. https://www.hcplive.com/view/ocu400-shows-benefit-for-retinitis-pigmentosa-in-phase-1-2-data.}