Ocugen, Inc. announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency reviewed the study design, endpoints and planned statistical analysis of the pivotal OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP) and provided acceptability of the U.S.-based trial for submission of a Marketing Authorization Application. EMA provided this opinion based on safety and tolerability of OCU400 demonstrated in the Phase 1/2 study. The Phase 3 liMeliGhT study will have a sample size of 150 participants primarily in the U.S.?one arm of 75 participants with RHO gene mutations and the other arm with 75 participants that are gene agnostic (representing multiple gene mutations associated with RP).

In each arm, participants will be randomized 2:1 to the treatment group (2.5 x 1010 vg/eye of OCU400) and untreated control group, respectively. The positive scientific advice from EMA is in alignment with U.S. FDA clearance of the IND amendment to initiate the Phase 3 liMeliGhT clinical trial of OCU400. OCU400 is the first gene therapy to enter Phase 3 with a broad RP indication.

Previously, OCU400 received broad Orphan Drug Designation for RP and Leber congenital amaurosis in the EU. The EMA opinion is an extremely favorable outcome, as it will potentially reduce the time and cost to gain marketing authorization in the EU. With this milestone, OCU400 remains on track for 2026 BLA and MAA approval targets.