Ocugen, Inc. has received FDA approval to initiate an expanded access program (EAP) for OCU400, a modifier gene therapy, in adult patients with retinitis pigmentosa (RP). This decision allows patients with significant unmet medical needs to access the investigational treatment outside of the ongoing Phase 3 liMeliGhT clinical trial.
The EAP is designed for RP patients aged 18 and older with early, intermediate, or advanced disease who have at least minimal retinal preservation and may benefit from OCU400's mechanism of action. This includes patients who did not meet inclusion criteria for the Phase 1/2 or Phase 3 trials, as well as those who participated in the Phase 1/2 study and are eligible for contralateral eye dosing.
Addressing Unmet Needs in RP
Retinitis pigmentosa affects approximately 300,000 individuals in the U.S. and Europe, and 1.6 million globally. Many RP patients with mutations in multiple genes currently lack therapeutic options. OCU400 represents a novel approach by targeting the NR2E3 gene, a nuclear hormone receptor that regulates key physiological functions within the retina, including photoreceptor development, metabolism, and cell survival.
"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. "The EAP strengthens our commitment to serving RP patients."
OCU400's Mechanism of Action
OCU400 aims to reset dysfunctional gene networks in retinal cells, re-establishing healthy cellular homeostasis and potentially improving vision in RP patients. Unlike single-gene replacement therapies, OCU400's modifier gene therapy platform targets multiple retinal diseases caused by various gene mutations.
Clinical Development and Regulatory Pathway
Ocugen is actively enrolling patients in the Phase 3 liMeliGhT clinical trial, a one-year study with 150 participants. The trial includes two arms: one with patients having RHO gene mutations and another with mutations in other genes. Within each arm, participants are randomized 2:1 to receive OCU400 (2.5 x1010 vector genomes/eye) or remain in the untreated control group. The company anticipates potential BLA and MAA approval for OCU400 in 2026.
"The OCU400 EAP gives RP patients access to this novel modifier gene therapy outside of the ongoing Phase 3 study," said Dr. Lejla Vajzovic, Director, Duke Surgical Vitreoretinal Fellowship Program, Associate Professor of Ophthalmology with Tenure, Adult and Pediatric Vitreoretinal Surgery and Disease, Duke University Eye Center, and Retina Scientific Advisory Board Chair of Ocugen.
Expanding Access and Enrollment
Ocugen's Chief Medical Officer, Dr. Huma Qamar, stated, "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 received orphan drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. The European Medicines Agency (EMA) has accepted the U.S.-based trial for submission of a Marketing Authorization Application (MAA).
