Ocugen’s OCU400 Gains Key EU Approval, Advancing Phase 3 Trial for Retinitis Pigmentosa

Ocugen, Inc. has reached a significant milestone in the development of its groundbreaking gene therapy, OCU400, as the European Medicines Agency (EMA) has granted it Advanced Therapy Medicinal Product (ATMP) classification. This designation accelerates the regulatory process, bringing OCU400 a step closer to market approval for patients with retinitis pigmentosa (RP), a degenerative eye disease with no current cure. The decision highlights the therapy’s potential to offer a one-time treatment option for individuals affected by various genetic mutations associated with RP. With the Phase 3 liMeliGhT trial now underway, Ocugen aims to secure marketing approval in both the U.S. and Europe by 2027.

Dr. Shankar Musunuri, Chairman and CEO of Ocugen, emphasized the impact of this recognition, noting that it strengthens their clinical and commercial strategy. With support from both the EMA and the U.S. FDA, Ocugen is optimistic that its pivotal Phase 3 trial will be sufficient for regulatory submissions. The company’s gene-agnostic approach, based on NR2E3, aims to restore retinal function and improve the lives of RP patients. As the study enrolls participants, Ocugen remains committed to working closely with regulators to address the unmet medical needs of thousands worldwide.