Ocugen, Inc. (NASDAQ: OCGN) has achieved a significant milestone with the European Medicines Agency (EMA) granting orphan medicinal product designation to OCU410ST, a gene therapy candidate for ABCA4-associated retinopathies, including Stargardt disease. This regulatory recognition underscores the potential of OCU410ST to address a critical unmet need in inherited retinal diseases affecting approximately 100,000 individuals across the U.S. and Europe.
EMA Orphan Designation Benefits
The EMA’s orphan designation provides Ocugen with several key benefits, including protocol assistance, reduced fees, access to research grants, and 10 years of market exclusivity upon approval. This support will facilitate the continued development and potential commercialization of OCU410ST in Europe.
Dr. Arun Upadhyay, Chief Scientific Officer and Head of R&D at Ocugen, stated, “We are deeply honored to receive orphan medicinal product designation from the EMA for OCU410ST. This recognition brings us one step closer to providing a much-needed option for Stargardt patients who currently have no therapies available.”
Promising Clinical Trial Results
Ocugen's ongoing Phase 1/2 OCU410ST GARDian trial has completed dosing for its initial phase, demonstrating encouraging safety and tolerability. The Data and Safety Monitoring Board recommended advancing to Phase 2 based on these positive findings. Preliminary results presented at Ocugen’s Clinical Showcase revealed an 84% reduction in atrophic lesion growth in treated eyes compared to untreated eyes over six months.
Dr. Huma Qamar, Chief Medical Officer at Ocugen, highlighted the significance of these results: “We are encouraged by the preliminary efficacy data showing stabilization or improvement in visual function and retinal structure outcomes in OCU410ST treated eyes.”
Novel Modifier Gene Therapy Approach
OCU410ST employs an AAV delivery platform to introduce the RORA gene, representing Ocugen’s innovative modifier gene therapy approach. This method targets Nuclear Hormone Receptors (NHRs) to regulate functions vital for photoreceptor health and overall eye physiology. The U.S. Food and Drug Administration (FDA) previously granted orphan drug designation to OCU410ST in April 2023.
With plans for an accelerated marketing authorization application (MAA), Ocugen aims to provide a potentially transformative, one-time treatment for patients with Stargardt disease and similar conditions.
