Ocugen, Inc. announced that the European Medicines Agency (EMA) has granted orphan medicinal product designation for OCU410ST. This designation is for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3). This recognition is a significant step towards providing a therapeutic option for Stargardt patients, who currently have no available treatments.
The orphan designation in Europe offers several benefits to drug developers, including protocol assistance, reduced regulatory fees, research grants, and 10 years of market exclusivity following regulatory approval. Stargardt disease affects approximately 100,000 people in the U.S. and Europe combined. The U.S. Food and Drug Administration (FDA) previously granted orphan drug designation to OCU410ST in April 2023.
Preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the Phase 1/2 OCU410ST GARDian clinical trial, recently presented at Ocugen’s Clinical Showcase, demonstrated an 84% reduction in atrophic lesion growth in treated eyes versus untreated fellow eyes at six months. This positive clinical data, combined with the EMA designation, supports the potential for OCU410ST as a one-time therapy for inherited retinal diseases.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.