Ocugen Announces European Medicines Agency Grants Orphan Medicinal Product Designation for Modifier Gene Therapy Candidate OCU410ST for Treatment of ABCA4-Associated Retinopathies including Stargardt Disease

Core Viewpoint - Ocugen, Inc. has received orphan medicinal product designation from the European Medicines Agency (EMA) for its treatment OCU410ST aimed at addressing ABCA4-associated retinopathies, including Stargardt disease, which currently lacks available therapies [1][2]. Group 1: Regulatory Designation and Benefits - The EMA's orphan medicinal product designation provides various advantages for drug developers, including protocol assistance, reduced regulatory fees, research grants, and 10 years of market exclusivity post-approval [3]. - The U.S. Food and Drug Administration (FDA) had previously granted orphan drug designation to OCU410ST in April 2023, highlighting the drug's significance in treating rare diseases [2]. Group 2: Clinical Trial Progress - Dosing for the first phase of the Phase 1/2 OCU410ST GARDian trial for Stargardt disease has been completed, with the Data and Safety Monitoring Board (DSMB) recommending progression to Phase 2 due to a favorable safety and tolerability profile [4]. - Preliminary data from the Phase 1 dose-escalation portion of the trial indicated an 84% reduction in atrophic lesion growth in treated eyes compared to untreated eyes after six months [5]. Group 3: Future Plans and Technology - Ocugen plans to pursue an accelerated marketing authorization application (MAA) for OCU410ST, indicating a commitment to expedite the drug's availability [7]. - OCU410ST employs an AAV delivery platform for retinal delivery of the RORA gene, representing Ocugen's innovative modifier gene therapy approach that targets multiple physiological functions [6].