Ocugen, Inc. 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. OCU400 is the first gene therapy to enter Phase 3 with a broad retinitis pigmentosa (RP) indication. This classification is a significant milestone towards bringing OCU400 to the European market.
The ATMP classification accelerates the regulatory review timeline for this potential one-time gene therapy. It also allows Ocugen to interact more frequently with the EMA for scientific advice and protocol assistance as the company pursues a Marketing Authorization Application (MAA) filing in 2026. Both the U.S. FDA and EMA have acknowledged that the ongoing single, pivotal Phase 3 trial of OCU400 can suffice for BLA/MAA submissions.
RP affects nearly 310,000 patients in the U.S., EU, and Canada, and is associated with mutations in over 100 genes. OCU400's gene-agnostic modifier gene therapy approach aims to reset dysfunctional gene networks to reestablish cellular homeostasis, offering a potential treatment for nearly 98% of the RP patient population. This designation supports Ocugen's clinical and commercial strategy to provide this novel therapy to all eligible RP patients.
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