MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has received approval from the Food and Drug Administration to begin its expanded access program (EAP) for the treatment of adult patients with retinitis pigmentosa (RP) using OCU400, a modifier gene therapy product candidate.
“Each clinical milestone achieved by OCU400 brings us closer to providing a potential one-time treatment for life to patients living with RP,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “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 U.S. and Europe and 1.6 million globally.”
The EAP allows patients with unmet medical needs and serious conditions to access treatments not yet approved by the FDA. The OCU400 EAP will be available for patients with early to advanced RP who have minimal retinal preservation and could benefit from OCU400 before the Biologics License Application (BLA) approval.
Ocugen is currently dosing patients in the Phase 3 liMeliGhT clinical trial. “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,” said Dr. Lejla Vajzovic, MD, FASRS, Director, Duke Surgical Vitreoretinal Fellowship Program, and Associate Professor of Ophthalmology at Duke University Eye Center. “The OCU400 EAP gives RP patients access to this novel modifier gene therapy outside of the ongoing Phase 3 study.”
“We are pleased to make OCU400 available to patients beyond our Phase 3 liMeliGhT clinical trial through this EAP,” said Dr. Huma Qamar, Ocugen’s Chief Medical Officer. “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.”
OCU400 has previously received orphan drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. The European Medicines Agency (EMA) has also accepted the U.S.-based trial for submission of a Marketing Authorization Application (MAA). With the ongoing Phase 3 clinical trial, OCU400 is on track for targeted BLA and MAA approval in 2026.
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