Ocugen, Inc., a biotechnology company specializing in gene and cell therapies and vaccines, has received FDA approval for its expanded access program (EAP) for adult patients aged 18 and older with retinitis pigmentosa (RP) to access OCU400, a gene therapy candidate.

"Every milestone we achieve with OCU400 brings us closer to offering a potential one-time treatment for RP patients. With encouraging results from our Phase 1/2 study and the ongoing Phase 3 liMeliGhT trial, we are committed to collaborating with clinicians and the RP community to make OCU400 accessible to eligible patients through our EAP. This program reinforces our dedication to supporting the 300,000 RP patients in the U.S. and Europe and the 1.6 million affected globally."

The EAP is designed for patients with serious or life-threatening conditions to access treatments not yet approved by the FDA outside of clinical trials. It targets RP patients with early to advanced disease stages with minimal retinal preservation who could benefit from OCU400 before Biologics License Application (BLA) approval. Ocugen is currently dosing patients in the Phase 3 liMeliGhT trial.

Lejla Vajzovic, MD, Director of Duke Surgical Vitreoretinal Fellowship Program and Retina Scientific Advisory Board Chair of Ocugen, noted, "RP patients with various gene mutations currently have no therapeutic options. As a retinal surgeon, I'm hopeful about the long-term benefits of OCU400. The EAP provides RP patients access to this innovative gene therapy outside of the Phase 3 study."

Dr. Huma Qamar, Ocugen’s Chief Medical Officer, added, "We're excited to offer OCU400 to patients beyond our Phase 3 liMeliGhT clinical trial through this EAP. We are broadening our enrollment to include patients with diverse RP gene mutations, reflecting our commitment to developing safe and effective therapies for RP patients who may lack other treatment options."

OCU400 has received orphan drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, and the European Medicines Agency (EMA) has accepted the U.S.-based trial for a Marketing Authorization Application (MAA). OCU400 is on track for targeted BLA and MAA approval in 2026.

The Phase 3 liMeliGhT clinical trial will last one year and include 150 participants. One arm will have 75 participants with RHO gene mutations, and the other arm will have 75 participants with other gene mutations. Participants will be randomized 2:1 to the treatment group (2.5 x10¹⁰ vector genomes/eye of OCU400) and an untreated control group. The study will recruit patients aged eight years and older with early to late-stage RP.

OCU400 is Ocugen’s gene therapy candidate based on the NR2E3 nuclear hormone receptor (NHR) gene, which regulates various physiological functions in the retina. In RP patients, OCU400 aims to reset the dysfunctional gene network to restore healthy cellular homeostasis, potentially improving vision.

Modifier gene therapy addresses unmet medical needs related to retinal diseases, including inherited retinal diseases (IRDs) such as RP, Leber congenital amaurosis (LCA), and Stargardt disease, as well as multifactorial diseases like dry age-related macular degeneration (dAMD). Ocugen’s platform uses NHRs to restore retinal homeostasis, potentially treating multiple retinal diseases caused by various gene mutations and complex diseases resulting from gene network imbalances. Ocugen has three modifier gene therapy programs in the clinic: OCU400, OCU410, and OCU410ST. In addition to the OCU400 Phase 3 liMeliGhT trial, the OCU410 Phase 1/2 ArMaDa trial for geographic atrophy (GA) secondary to dAMD and the OCU410ST Phase 1/2 GARDian trial for Stargardt disease are ongoing. GA affects about two to three million people in the U.S. and EU combined, and Stargardt disease affects nearly 100,000 people in these regions.

 

Source: ocugen.com

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