FDA Approves Ocugen’s Expanded Access Program for Retinitis Pigmentosa

Ocugen, Inc., a biotechnology firm dedicated to innovative gene and cell therapies and vaccines, has been given the green light by the FDA to commence an expanded access program (EAP) for its OCU400 product. The program aims to treat adults aged 18 and older suffering from retinitis pigmentosa (RP), a degenerative eye disease.

Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen, emphasized the significant progress made with OCU400, highlighting its Phase 1/2 study data and the ongoing Phase 3 liMeliGhT clinical trial. Dr. Musunuri noted the company's plans to collaborate with clinicians, patients, and the RP community to provide access to OCU400 through the EAP, thus reinforcing their commitment to the 1.6 million RP patients globally, including 300,000 in the U.S. and Europe.

The EAP is designed for patients with serious or life-threatening conditions who cannot wait for FDA approval. It allows these patients to access treatments outside of clinical trials. Specifically, the OCU400 EAP targets RP patients in early to advanced stages with minimal retinal preservation who could benefit from OCU400's mechanism of action before the Biologics License Application (BLA) is approved. Ocugen is currently administering the phase 3 liMeliGhT clinical trial.

Lejla Vajzovic, MD, an expert in retinal surgery at Duke University Eye Center and a member of Ocugen's Retina Scientific Advisory Board, expressed optimism about the potential long-term benefits of OCU400 for RP patients with various genetic mutations. The EAP offers these patients access to the innovative gene therapy outside the ongoing clinical study.

Dr. Huma Qamar, Ocugen’s Chief Medical Officer, expressed enthusiasm about extending the availability of OCU400 through the EAP, which allows the inclusion of diverse RP gene mutations. This initiative underscores Ocugen's dedication to developing a safe and effective therapy for RP patients who currently lack treatment options.

OCU400 has previously earned orphan drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. Moreover, the European Medicines Agency (EMA) has accepted the U.S.-based trial for a Marketing Authorization Application (MAA). With dosing underway in the Phase 3 clinical trial, Ocugen aims for BLA and MAA approvals by 2026.

The OCU400 EAP is specifically for U.S. patients who meet certain criteria: adult RP patients aged 18 and older with early to advanced disease and minimal retinal preservation, participants from the Phase 1/2 study who qualify for contralateral eye dosing, patients excluded from previous trials who could benefit from OCU400, and those who might benefit from the therapy's mechanism before BLA approval.

The Phase 3 liMeliGhT clinical trial includes 150 participants, divided into two groups based on their genetic mutations. Each group is further split between a treatment group receiving OCU400 and an untreated control group. The trial targets patients aged eight and older with varying stages of RP.

OCU400, Ocugen's gene therapy product, is centered on the NR2E3 gene, which regulates multiple functions in the retina. This therapy aims to restore healthy cellular balance, potentially improving vision in RP patients. Unlike single-gene replacement therapies, Ocugen's modifier gene therapy platform targets multiple retinal diseases and gene mutations, offering a novel approach.

Ocugen is also conducting other clinical trials for different retinal diseases, including the OCU410 Phase 1/2 ArMaDa trial for geographic atrophy secondary to dry age-related macular degeneration (dAMD) and the OCU410ST Phase 1/2 GARDian trial for Stargardt disease. These diseases affect millions of people in the U.S. and Europe.

Ocugen, Inc. focuses on groundbreaking gene and cell therapies and vaccines to improve global health. The company is advancing research in infectious and orthopedic diseases to address unmet medical needs, continuing to make significant strides in medical innovation.