Ocugen's OCU410ST Receives Orphan Drug Status from EMA for ABCA4-Related Retinopathies

MALVERN, Pa., Nov. 20, 2024 – Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company dedicated to the discovery, development, and commercialization of innovative gene and cell therapies, biologics, and vaccines, has 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).

Dr. Arun Upadhyay, Chief Scientific Officer and Head of R&D at Ocugen, expressed honor and gratitude for receiving the designation from the EMA. He emphasized the company's dedication to advancing this treatment, particularly for Stargardt disease patients who currently lack therapeutic options. Dr. Upadhyay highlighted the urgency and commitment with which Ocugen is pursuing this treatment, hoping to significantly impact the lives of those affected by these challenging diseases.

Previously, in April 2023, the U.S. Food and Drug Administration (FDA) also granted orphan drug designation to OCU410ST. Stargardt disease affects about 100,000 individuals in the U.S. and Europe combined. Orphan medicinal product designation in Europe provides certain advantages to drug developers working on treatments for rare diseases or conditions affecting fewer than 5 in 10,000 patients in the European Union. These benefits include protocol assistance, reduced regulatory fees, research grants, and ten years of market exclusivity following regulatory approval.

The first phase of the Phase 1/2 OCU410ST GARDian trial for Stargardt disease has completed dosing, and the Data and Safety Monitoring Board (DSMB) has recommended proceeding with Phase 2. The safety and tolerability profile of OCU410ST has been favorable so far. Preliminary efficacy and safety data from Phase 1 were recently presented at Ocugen’s Clinical Showcase. The data showed an impressive 84% reduction in the growth of atrophic lesions in treated eyes compared to untreated eyes.

Dr. Huma Qamar, Chief Medical Officer at Ocugen, expressed encouragement from the preliminary efficacy data, which showed stabilization or improvement in visual function and retinal structure outcomes in eyes treated with OCU410ST. These positive clinical and regulatory milestones support the potential of OCU410ST to address inherited retinal diseases with a single, one-time therapy.

OCU410ST employs an AAV delivery platform for retinal delivery of the RORA (RAR Related Orphan Receptor A) gene, showcasing the impact of Ocugen’s modifier gene therapy approach. This approach is based on Nuclear Hormone Receptors (NHRs), which regulate various physiological functions, including photoreceptor development and maintenance, metabolism, phototransduction, inflammation, and cell survival networks.

Ocugen intends to seek an accelerated marketing authorization application (MAA) for OCU410ST.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene and cell therapies, biologics, and vaccines. The company aims to improve health and offer hope to patients worldwide through innovative scientific advancements. Ocugen’s breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product. The company is also advancing research in infectious diseases to benefit public health and orthopedic diseases to address unmet medical needs.