Ocugen Inc., (NASDAQ:OCGN), an ophthalmic biopharmaceutical company based in Malvern, Pennsylvania, has announced that the European Commission has provided a positive opinion from the European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT) for OCU400 Advanced Therapy Medicinal Product (ATMP) classification. According to the company, OCU400 is the first gene therapy to enter Phase 3 with a broad retinitis pigmentosa (RP) indication. OCU400, a modifier gene therapy candidate for the treatment of RP and LCA, aimed to treat RP associated NR2E3 and rhodopsin (RHO) mutations, and LCA with mutation(s) in the CEP290 gene.
Ocugen has confirmed that their Phase 3 “liMeliGhT” clinical trial is enrolling patients with a study sample size of 150 participants—one arm of 75 participants with RHO gene mutations and the other arm with 75 participants “that are gene agnostic” (clinicaltrials.gov identifier: NCT06388200). In each arm, participants will be randomized 2:1 to the treatment group (2.5 x 1010vg/eye of OCU400) and untreated control group, respectively. The primary endpoint of the proposed trial is performance on the company’s “Luminance Dependent Navigation Assessment (LDNA)” navigational course, an enhanced version of the MLMT used for the approval of voretigene neparvovec (Luxturna). Patients eight years of age and older, with early through late-stage advancement of RP, are being recruited. OCU400 is aimed to provide gene-agnostic modifier gene treatment based on NHR gene, NR2E3 which regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. According to the company, “OCU400 resets altered/affected cellular gene networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with inherited retinal diseases”.
In their announcement, Ocugen reported that ATMP classification is granted to medicines that may offer “ground-breaking opportunities for the treatment of disease and accelerates the regulatory review timeline of this potential one-time gene therapy for life”. Additionally, the ATMP classification allows Ocugen to interact with EMA more frequently for scientific advice and protocol assistance as the company pursues Marketing Authorization Application (MAA) filing in FY2026. Following their presentation, Dr. Huma Qamar, Chief Medical Officer at Ocugen commented that, “we are encouraged by the EMA’s recognition of OCU400 as the Phase 3 liMeliGhT clinical trial advances. “ look forward to working collaboratively with the EMA to address the unmet medical need that remains for nearly 98% of the RP patient population.”
Figure 1. Dr. Qamar, Chief Medical Officer, Ocugen is experienced in developing Phase I-IV clinical protocols and execution of clinical studies across multiple therapeutic areas, including gene and cell therapy, vaccines, oncology, rheumatology, dermatology, neurology, cardiology, hepatology, and infectious diseases. Dr. Qamar was instrumental overseeing Phase 1/2 of Retinitis Pigmentosa trials as VP, Head of Clinical Development & Clinical Operations for Ocugen.
Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen stated that “receiving ATMP classification is another significant milestone toward bringing OCU400 to the market in Europe. This designation makes it possible to stay on track with our clinical and commercial strategy and potentially provide this novel modifier gene therapy candidate to all RP patients in the United States (U.S.) and Europe by 2027.”