Ocugen's OCU410ST, a modifier gene therapy, has received orphan medicinal product designation from the European Medicines Agency (EMA) for the treatment of ABCA4-associated retinopathies, which include Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3). This designation follows the orphan drug designation granted by the U.S. Food and Drug Administration (FDA) in April 2023, underscoring the potential of OCU410ST in addressing these rare and debilitating conditions.
The orphan medicinal product designation by the EMA provides several benefits to drug developers, including protocol assistance, reduced regulatory fees, access to research grants, and a 10-year period of market exclusivity upon regulatory approval. These incentives are designed to encourage the development of treatments for rare diseases that affect a small patient population.
Clinical Trial Progress and Efficacy Data
The Phase 1/2 OCU410ST GARDian trial for Stargardt disease has completed its first phase of dosing, with the Data and Safety Monitoring Board (DSMB) recommending the initiation of Phase 2 enrollment in October 2024. Preliminary data from the Phase 1 dose-escalation portion of the trial, presented at Ocugen’s Clinical Showcase, demonstrated promising efficacy and safety results.
Evaluable subjects at 6 months showed an 84% reduction in atrophic lesion growth in treated eyes compared to their untreated fellow eyes. This significant reduction suggests a potential therapeutic benefit of OCU410ST in slowing the progression of Stargardt disease, a leading cause of inherited vision loss.
Mechanism of Action and Future Plans
OCU410ST utilizes an adeno-associated virus (AAV) delivery platform to introduce the RORA (RAR Related Orphan Receptor A) gene into the retina. This approach leverages Ocugen's modifier gene therapy platform, which is based on Nuclear Hormone Receptors (NHRs) that regulate various physiological functions, including photoreceptor development, maintenance, metabolism, phototransduction, inflammation, and cell survival networks.
Ocugen intends to pursue an accelerated marketing authorization application (MAA) for OCU410ST, aiming to expedite the availability of this potential treatment to patients with ABCA4-associated retinopathies. The company's commitment to advancing OCU410ST reflects the urgent need for effective therapies for these conditions, which currently lack approved treatments.
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. 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.”
