Ocugen Announces Positive Opinion of European Medicines Agency's Committee for Advanced Therapies for Advanced Therapy Medicinal Product Classification for Modifier Gene Therapy Candidate OCU400 for Retinitis Pigmentosa

Core Viewpoint - Ocugen, Inc. has received a positive opinion from the European Medicines Agency (EMA) for the Advanced Therapy Medicinal Product (ATMP) classification of its gene therapy OCU400, which is aimed at treating retinitis pigmentosa (RP) [1][2] Company Developments - The ATMP classification is a significant milestone for Ocugen, allowing the company to advance its clinical and commercial strategy for OCU400, with the goal of providing this therapy to RP patients in the U.S. and Europe by 2027 [2] - The ongoing Phase 3 trial, named liMeliGhT, is currently enrolling 150 participants, divided into two arms based on gene mutations, with a treatment group receiving OCU400 and a control group [4][5] - Ocugen plans to file for Marketing Authorization Application (MAA) in Europe and a Biologics License Application (BLA) in the U.S. simultaneously upon completion of the Phase 3 trial [3] Industry Context - Retinitis pigmentosa affects approximately 310,000 patients across the U.S., EU, and Canada, with no approved treatments currently available to slow or stop the progression of the disease [5] - OCU400 is designed as a gene-agnostic modifier gene therapy based on the NR2E3 gene, which plays a crucial role in retinal health and function [6]