MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) announced that the European Commission has granted Advanced Therapy Medicinal Product (ATMP) classifications for its gene therapies OCU410 and OCU410ST. This regulatory milestone marks a critical step forward in addressing devastating vision loss diseases, including Geographic Atrophy (GA) and Stargardt disease, which collectively impact millions globally.
OCU410 is a first-of-its-kind modifier gene therapy designed to treat GA associated with advanced dry age-related macular degeneration (dAMD). Meanwhile, OCU410ST targets Stargardt disease, a rare genetic condition stemming from ABCA4-related retinopathies. Current treatment options for GA are limited to two approved products in the U.S., both requiring frequent dosing, and there are no approved therapies for Stargardt disease worldwide.
“Receiving ATMP status for these therapeutic candidates is a noteworthy achievement,” stated Dr. Shankar Musunuri, Chairman and CEO of Ocugen. “This classification paves the way for accelerated regulatory pathways and brings us closer to fulfilling critical unmet medical needs.”
Promising Clinical Progress
Both OCU410 and OCU410ST have demonstrated encouraging early trial results. Data from the Phase 1 portion of the OCU410 ArMaDa trial showed a 44% reduction in lesion growth and a 10-letter improvement in visual function in treated GA patients over nine months. Treated eyes also preserved more retinal tissue compared to published outcomes for existing therapies.
Similarly, OCU410ST achieved a 52% reduction in lesion growth and statistically significant improvements in visual acuity in six-month Phase 1 data from the GARDian trial. Ocugen is on track to initiate a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST by mid-2025, with the potential for regulatory filings by 2027. A Phase 3 trial for OCU410 is expected to begin in 2025.
A Transformative Approach to Retinal Treatment
Both therapies utilize Ocugen’s adeno-associated virus (AAV) platform to deliver the RORA (ROR Related Orphan Receptor A) gene directly to the retina via a single subretinal injection. This novel mechanism targets the four pathways implicated in GA and Stargardt diseases, focusing on reducing oxidative stress, lipid deposition, inflammation, and complement system overactivation. The goal is a one-time treatment designed to preserve vision and improve quality of life for patients with progressive vision loss.
By achieving ATMP classification, Ocugen gains enhanced collaboration opportunities with the European Medicines Agency (EMA), including scientific guidance, while positioning its novel therapies for potential breakthroughs in vision care.
With substantial progress in the clinic and a robust development pipeline, Ocugen continues to push the boundaries of genetic medicine to tackle blindness-inducing diseases that lack viable solutions.
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