FDA Notifies Ocugen to Begin Expanded Access Program for Retinitis Pigmentosa Treatment with OCU400

The United States Food and Drug Administration (FDA) has given Ocugen, Inc. the green light to commence its expanded access program (EAP) for treating patients aged 18 and older with retinitis pigmentosa (RP) using OCU400. This modifier gene therapy product candidate is currently showing promising results in clinical trials.

Shankar Musunuri, PhD, MBA, Chairman, CEO, and Co-founder of Ocugen, provided an update on OCU400's progress in a recent press release. He stated, “Each clinical milestone achieved by OCU400 brings us closer to providing a potential one-time treatment for life to patients living with RP. With positive Phase 1/2 study data and an ongoing Phase 3 liMeliGhT clinical trial, we now plan to work with clinicians, patients, and the RP community to provide access to OCU400 for eligible patients through our EAP. The EAP strengthens our commitment to serving RP patients—300,000 in the US and Europe and 1.6 million globally.”

The EAP issuance typically allows patients with serious or life-threatening conditions and unmet medical needs to access treatments outside of a clinical trial that are not yet approved by the FDA.

For the OCU400 EAP, patients with early, intermediate, to advanced RP who have at least minimal retinal preservation and may benefit from OCU400's mechanism of action can access the treatment prior to the approval of the Biologics License Application (BLA). Ocugen is actively dosing patients in the Phase 3 liMeliGhT clinical trial.

Lejla Vajzovic, MD, FASRS, Director of the Duke Surgical Vitreoretinal Fellowship Program, Associate Professor of Ophthalmology with Tenure, Adult and Pediatric Vitreoretinal Surgery and Disease at Duke University Eye Center, and Retina Scientific Advisory Board Chair of Ocugen, emphasized the long-term potential of this candidate. She noted, “RP patients with mutations in multiple genes currently have no therapeutic options. As a retinal surgeon, I am encouraged by the therapeutic potential of OCU400 to provide long-term benefit. The OCU400 EAP gives RP patients access to this novel modifier gene therapy outside of the ongoing Phase 3 study.”

Huma Qamar, Ocugen’s Chief Medical Officer, expressed enthusiasm about the news and the current enrollment in clinical trials. She said, “We are pleased to make OCU400 available to patients beyond our Phase 3 liMeliGhT clinical trial through this EAP. We are excited to expand our enrollment to include patients representing a diverse array of RP gene mutations. This program reflects our ongoing commitment to develop a safe and effective therapy for RP patients who may not have other treatment options.”

Previously, Ocugen noted that OCU400 has received orphan drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA and that the European Medicines Agency (EMA) accepted the US-based trial for submission of a Marketing Authorization Application (MAA). With the dosing of patients in the Phase 3 clinical trial program underway, OCU400 remains on track for targeted BLA and MAA approval in 2026.

Retinitis pigmentosa (RP) is a group of genetic disorders that result in the breakdown and loss of cells in the retina. This leads to symptoms like difficulty seeing at night and a loss of peripheral vision. RP affects approximately 1.6 million people globally, and current treatment options are limited, making advancements like OCU400 crucial for improving the quality of life for those affected by this condition.