Ocugen, Inc. Announces FDA Approval of Expanded Access Program for Patients with Retinitis Pigmentosa

MALVERN, Pa., Aug. 05, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that it has received notification from FDA to begin its expanded access program (EAP) for the treatment of adult patients, aged 18 and older, with retinitis pigmentosa (RP) with 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 (pronounced “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.”

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

The OCU400 EAP is available for patients with early, intermediate to advanced RP with at least minimal retinal preservation who may benefit from the mechanism of action of OCU400 prior to approval of the Biologics License Application (BLA). Ocugen is actively 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 Lejla Vajzovic, MD, FASRS, 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. “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.”