Ocugen announced that the Committee for Medicinal Products for Human Use, CHMP, of the European Medicines Agency, EMA, reviewed the study design, endpoints and planned statistical analysis of the pivotal OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa 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. In each arm, participants will be randomized 2:1 to the treatment group 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.
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- Ocugen, Inc. Announces Positive Scientific Advice from the European Medicines Agency Related to the Approval Pathway for OCU400—Modifier Gene Therapy for Broad Retinitis Pigmentosa Indication
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