Ocugen, Inc. (OCGN) has announced that the European Medicines Agency has awarded orphan medicinal product status to OCU410ST for the treatment of ABCA4-associated retinopathies. These include Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3. Previously, in April 2023, the FDA had granted orphan drug designation to OCU410ST.
Dr. Huma Qamar, Chief Medical Officer at Ocugen, commented: "These significant clinical and regulatory advancements bolster the potential of OCU410ST as a groundbreaking single-dose therapy for inherited retinal diseases."
Ocugen intends to seek an accelerated marketing authorization for OCU410ST.
