MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) announced a significant milestone with the U.S. Food and Drug Administration (FDA), gaining alignment to advance its Phase 2/3 pivotal confirmatory trial of OCU410ST, a promising gene therapy for Stargardt disease. If successful, the trial results could form the basis for a biologics license application (BLA) submission as early as 2027.
A Potential Game-Changer for Stargardt Disease
Stargardt disease, a progressive genetic eye disorder that leads to vision loss, currently has no treatment options. Affecting approximately 100,000 individuals in the U.S. and Europe combined, the disease presents a critical unmet need that OCU410ST aims to address as a one-time gene therapy solution.
The FDA’s endorsement of the trial design builds on data from the GARDian Phase 1 trial, which revealed compelling results, including a 52% reduction in lesion growth and a statistically significant two-line improvement in best corrected visual acuity (BCVA) at the 6-month follow-up. Importantly, the therapy demonstrated a strong safety profile, with no reported cases of ischemic optic neuropathy, intraocular inflammation, or other serious adverse effects.
“This new development allows us to initiate a pivotal confirmatory trial for this game-changing, one-time treatment,” stated Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “Now patients suffering from Stargardt disease have a new hope where previously none existed.”
Expedited Regulatory Pathway
OCU410ST has already secured orphan drug designations from both the FDA and the European Medicines Agency (EMA), reflecting its potential as an innovative treatment for rare diseases. The collaboration with the FDA has enabled Ocugen to accelerate the clinical timeline of the program, potentially shortening development by two to three years.
“This approach has never been explored in clinical trials for Stargardt disease, and it underscores the potential of OCU410ST to meet a critical unmet medical need,” said Dr. Lejla Vajzovic, Retina Scientific Advisory Board Chair of Ocugen and Professor at the Duke University Eye Center.
The Phase 2/3 trial will enroll 51 participants, with 34 receiving a single subretinal injection of OCU410ST and 17 serving as untreated controls. The primary endpoint is a change in atrophic lesion size, with secondary endpoints focusing on visual acuity improvements. One-year data will serve as the foundation for the BLA filing.
A Step Toward Curing Blindness
With promising preliminary data and a streamlined regulatory process, Ocugen is poised to transform the landscape of Stargardt disease treatment. The company’s ambition to bring groundbreaking therapies to market reflects its broader mission to tackle blindness diseases and reduce the burden on patients and healthcare systems worldwide.
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