Ocugen, Inc. has announced that the European Medicines Agency (EMA) has granted orphan medicinal product designation to OCU410ST for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3). This designation underscores the potential of OCU410ST to address a significant unmet need in patients with these rare inherited retinal diseases.
Significance of EMA Designation
The orphan medicinal product designation by the EMA provides several benefits to Ocugen, including protocol assistance, reduced regulatory fees, research grants, and 10 years of market exclusivity upon regulatory approval. This support is crucial for advancing the development of OCU410ST and bringing it to patients who currently have limited or no treatment options.
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.”
Prior FDA Orphan Drug Designation
In April 2023, the U.S. Food and Drug Administration (FDA) also granted orphan drug designation to OCU410ST. Stargardt disease, one of the conditions included in the EMA designation, affects approximately 100,000 people in the U.S. and Europe combined.
Clinical Trial Progress
Ocugen is currently conducting the Phase 1/2 OCU410ST GARDian trial for Stargardt disease. The first phase of dosing is complete, and the Data and Safety Monitoring Board (DSMB) has recommended moving forward with Phase 2, based on the favorable safety and tolerability profile observed to date.
Preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the trial were recently presented at Ocugen’s Clinical Showcase. Data from evaluable subjects at six months demonstrated an 84% reduction in atrophic lesion growth in treated eyes versus untreated fellow eyes.
Mechanism of Action
OCU410ST utilizes an adeno-associated virus (AAV) delivery platform to introduce the RORA (RAR Related Orphan Receptor A) gene into the retina. This modifier gene therapy approach is based on Nuclear Hormone Receptors (NHRs) that regulate various physiological functions, including photoreceptor development and maintenance, metabolism, phototransduction, inflammation, and cell survival networks.
Future Plans
Ocugen intends to pursue an accelerated marketing authorization application (MAA) for OCU410ST, aiming to expedite the availability of this potential treatment for patients with ABCA4-associated retinopathies.
Dr. Huma Qamar, Chief Medical Officer at Ocugen, commented, “We are encouraged by the preliminary efficacy data showing stabilization or improvement in visual function and retinal structure outcomes in OCU410ST treated eyes. These positive clinical and regulatory milestones continue to support the potential for OCU410ST to address inherited retinal diseases with a one-time therapy for life.”
