Ocugen’s ongoing Phase 2/3 GARDian3 trial for Stargardt disease has received a positive opinion from the EMA’s Committee for Medicinal Products for Human Use (CHMP). The CHMP confirmed the acceptability of a single U.S.-based trial for a future Marketing Authorization Application (MAA) submission. This decision follows promising Phase 1 GARDian trial data, which demonstrated a 48% slower lesion growth rate in treated eyes compared to untreated controls. Additionally, the Phase 1 trial showed a statistically significant (p=0.031) improvement in best corrected visual acuity (BCVA) of nearly 2 lines/9 letters at 12 months.
This EMA opinion streamlines Ocugen’s regulatory pathway, allowing it to leverage a single pivotal study for both U.S. and EU submissions. This approach reduces development costs and potentially accelerates timelines. It aligns with broader regulatory trends towards greater harmonization between agencies, particularly for rare diseases with limited patient populations. The single-trial strategy carries inherent risk: if the Phase 2/3 study fails to meet its primary endpoint, it jeopardizes both BLA and MAA filings.
The Phase 2/3 GARDian3 trial will enroll 51 Stargardt patients, with 34 receiving a single subretinal injection of OCU410ST and 17 in an untreated control arm. The adaptive design incorporates a masked interim analysis at eight months. The primary endpoint focuses on the reduction of atrophic lesion size, while key secondary endpoints assess BCVA and low luminance visual acuity (LLVA) improvements. The trial’s relatively small size and adaptive nature reflect the challenges of recruiting for rare ophthalmic conditions. This approach, while efficient, also requires robust statistical planning and careful endpoint selection.
The CHMP’s acceptance of the U.S.-based trial underscores the increasing importance of high-quality data over geographic distribution in regulatory decision-making. This shift benefits smaller biotech companies like Ocugen, which may lack the resources for extensive multinational trials. It also reinforces the FDA’s influence on global regulatory standards, particularly for innovative therapies. The streamlined process may also influence payer dynamics, providing a more predictable pathway for potential reimbursement negotiations in multiple markets.
Ocugen aims to complete enrollment in Q1 2026, targeting a BLA filing in H1 2027. The company’s reliance on this single pivotal study for both U.S. and EU approval places significant pressure on trial execution. The tight timeframe leaves little room for delays or unexpected outcomes. Investors will be watching closely for updates on enrollment progress, interim analysis results, and any potential adjustments to the trial protocol. The long-term success of OCU410ST hinges not only on its clinical efficacy but also on the company’s ability to navigate complex regulatory and reimbursement landscapes in multiple markets.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
