MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has received a key regulatory boost in its bid to bring a gene therapy for Stargardt disease to market, with the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) agreeing that a single, U.S.-based Phase 2/3 trial could support a Marketing Authorization Application in the EU.
The EMA opinion follows encouraging Phase 1 results for OCU410ST, which demonstrated a 48% slower growth in retinal lesions and nearly a two-line (nine-letter) improvement in best corrected visual acuity (BCVA) at 12 months in treated eyes compared to untreated controls. The therapy, delivered as a one-time subretinal injection, targets approximately 100,000 patients in the U.S. and Europe who currently have no approved treatment options.
“This positive opinion endorses a single trial as the basis for both BLA and MAA submissions and brings us closer to providing a one-time, modifier gene therapy,” said Dr. Shankar Musunuri, Ocugen’s chairman, CEO, and co-founder.
The ongoing GARDian3 pivotal study will enroll 51 participants with Stargardt disease, with 34 receiving OCU410ST and 17 serving as untreated controls. The trial’s adaptive design includes a masked interim analysis after eight months for 24 participants. The primary endpoint is the reduction of atrophic lesion size, with secondary measures assessing improvements in BCVA and low luminance visual acuity (LLVA). Data from one-year follow-up will be used to support both U.S. and EU regulatory filings.
The EMA alignment could shorten Ocugen’s path to market and reduce development costs. It follows other program milestones this year, including Rare Pediatric Disease Designation in May, FDA clearance of an investigational new drug application in June, and first patient dosing in July. Enrollment is expected to finish in early 2026, with a Biologics License Application planned for the first half of 2027 — part of the company’s goal to submit three BLAs within three years.
OCU410ST uses an adeno-associated virus (AAV) platform to deliver the RORA gene, aiming to regulate disease pathways such as lipofuscin buildup, oxidative stress, inflammation, and photoreceptor cell survival. Stargardt disease, the most common inherited form of macular degeneration, typically causes progressive central vision loss beginning in childhood or adolescence, though onset and severity vary.
For the latest news on everything happening in Chester County and the surrounding area, be sure to follow MyChesCo on Google News and MSN.