MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has received orphan medicinal product designation from the European Medicines Agency (EMA) for OCU410ST, a promising treatment for ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3). This designation marks a significant step forward in Ocugen’s efforts to address these rare eye conditions.
Dr. Arun Upadhyay, Chief Scientific Officer and Head of R&D at Ocugen, expressed the company’s commitment, stating, “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.” The EMA’s designation is a crucial milestone, offering benefits such as protocol assistance, reduced fees, research grants, and 10 years of market exclusivity if the therapy is approved.
Stargardt disease, affecting approximately 100,000 individuals across the U.S. and Europe, currently has no available treatments. The U.S. Food and Drug Administration (FDA) previously granted orphan drug designation to OCU410ST in April 2023, further supporting its potential in treating this condition.
Ocugen’s ongoing Phase 1/2 OCU410ST GARDian trial has already completed dosing for its initial phase. Encouraging safety and tolerability data have prompted the Data and Safety Monitoring Board to recommend advancing to Phase 2. Preliminary results showcased at Ocugen’s Clinical Showcase reveal an impressive 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 promising results, saying, “We are encouraged by the preliminary efficacy data showing stabilization or improvement in visual function and retinal structure outcomes in OCU410ST treated eyes.” These findings, coupled with regulatory progress, underscore the potential of OCU410ST to transform treatment for inherited retinal diseases.
OCU410ST leverages an AAV delivery platform for the RORA gene, illustrating 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.
With plans for an accelerated marketing authorization application (MAA), Ocugen is poised to make significant strides in offering a one-time, life-changing treatment for patients with Stargardt disease and similar conditions.
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