EMA grants orphan medicinal product designation for OCU410ST (Ocugen)

Ocugen intends to pursue an accelerated marketing authorisation application for OCU410ST. Image credit: ©Timon – stock.adobe.com

The European Medicines Agency (EMA) has granted orphan medicinal product designation for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3).

The US Food and Drug Administration (FDA) previously granted orphan drug designation to OCU410ST in April 2023.¹

According to the company’s press release,¹ orphan medicinal product designation in Europe offers certain benefits to drug developers. Benefits include protocol assistance, reduced regulatory fees, research grants, and 10 years of market exclusivity following regulatory approval.

Dr. Arun Upadhyay, Chief Scientific Officer and Head of R&D at Ocugen spoke to this milestone by saying, “We are deeply honoured 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. We are committed to advancing this treatment with urgency and dedication, with the hope of making a meaningful impact on the lives of those affected by this challenging disease."¹

GARDian trial

Dosing in the first phase of the Phase 1/2 OCU410ST GARDian trial for Stargardt disease is complete. In October of 2024, the Data and Safety Monitoring Board (DSMB) recommended moving forward with Phase 2 enrollment.²

Preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the Phase 1/2 OCU410ST GARDian clinical trial was recently presented at Ocugen’s Clinical Showcase. Data from evaluable subjects at 6 months demonstrated a remarkable 84% reduction in atrophic lesion growth in treated eyes versus untreated fellow eyes.¹

Next steps

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

Ocugen intends to pursue an accelerated marketing authorisation application for OCU410ST.

References:

1. Ocugen Announces European Medicines Agency Grants Orphan Medicinal Product Designation for Modifier Gene Therapy Candidate OCU410ST for Treatment of ABCA4-Associated Retinopathies including Stargardt Disease. Ocugen, Inc. November 20, 2024. Accessed November 22, 2024. https://www.globenewswire.com/news-release/2024/11/20/2984336/0/en/Ocugen-Announces-European-Medicines-Agency-Grants-Orphan-Medicinal-Product-Designation-for-Modifier-Gene-Therapy-Candidate-OCU410ST-for-Treatment-of-ABCA4-Associated-Retinopathies-.html

2. Data and Safety Monitoring Board Approves Initiation of Phase 2 of OCU410ST GARDian Clinical Trial for Stargardt Disease. Ocugen, Inc. October 22, 2024. Accessed November 22, 2024. https://www.globenewswire.com/news-release/2024/10/22/2966807/0/en/Data-and-Safety-Monitoring-Board-Approves-Initiation-of-Phase-2-of-OCU410ST-GARDian-Clinical-Trial-for-Stargardt-Disease.html