EMA provides positive ATMP opinion for gene therapy products OCU410 and OCU410ST

According to Ocugen, OCU410 and OCU410ST both demonstrated clinically meaningful visual function gains in patients. Image credit: ©rost9 – stock.adobe.com

In a press release, biotech firm Ocugen announced that the European Medicines Agency (EMA) and its Committee for Advanced Therapies (CAT) has provided a positive opinion for two of its novel gene therapy products. OCU410 is intended for the treatment of vision loss due to geographic atrophy (GA) associated with dry age-related macular degeneration (AMD); OCU410ST is being developed to treat Stargardt disease, a condition associated with ABCA4-related retinopathies.

Ocugen confirmed that both products had received Advanced Therapy Medicinal Product (ATMP) classification from the European Commission's EMA.¹ The classification will accelerate the regulatory review timeline of the gene therapies, and will enable Ocugen to receive scientific guidance and protocol assistance from the EMA.

The EMA’s positive opinion on OCU410 and OCU410ST follows a similar update from early February, when the EMA provided ATMP designation to OCU400, Ocugen's gene therapy indicated for treating retinitis pigmentosa.²

OCU410 and OCU410ST both utilise an adeno-associated virus platform for gene delivery. Preliminary 9-month data assessing OCU410 showed considerably lower lesion growth and clinically meaningful visual function gains for patients with GA. The 6-month data from Phase 1 of the OCU410ST GARDian clinical trial demonstrated slower lesion growth from baseline in treated eyes versus untreated fellow eyes, as well as clinically meaningful visual improvement in patients with Stargardt disease.¹

Huma Qamar, MD, MPH, CMI, the chief medical officer at Ocugen, spoke about the trial results in the press release.¹ “The novel modifier gene in OCU410 and OCU410ST targets all four pathways linked with dAMD and Stargardt and is delivered through a single subretinal injection as a one-and-done treatment. We are very pleased with the structural and functional outcomes demonstrated by both of these candidates, along with a stellar safety profile.”

In the press release, Shankar Musunuri, PhD, MBA, chairman, CEO and co-founder of Ocugen, explained that the ATMP designation could help OCU410 and OCU410ST address patients’ unmet needs in the very near future. “Dosing of Phase 2 in the ongoing OCU410 ArMaDa clinical trial is complete, and we are on track to initiate the Phase 3 clinical trial next year to pursue potential Marketing Authorisation Application and Biologics License Application filings in 2028,” he said. “Last week, the US Food and Drug Administration endorsed Ocugen’s plan to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST, which can be the basis of BLA and potential MAA submissions in 2027.”

References

1. Ocugen Announces Positive Opinion of EMA’s Committee for Advanced Therapies for ATMP Classification for Novel Modifier Gene Therapy Candidate OCU410 for Geographic Atrophy and OCU410ST for Stargardt Disease. Ocugen. Press release. Published March 3, 2025. Accessed March 4, 2025. https://www.globenewswire.com/fr/news-release/2025/03/03/3035695/0/en/Ocugen-Announces-Positive-Opinion-of-EMA-s-Committee-for-Advanced-Therapies-for-ATMP-Classification-for-Novel-Modifier-Gene-Therapy-Candidate-OCU410-for-Geographic-Atrophy-and-OCU4.html

2. Koennecke G. Ocugen, Inc. receives European Medicines Agency ATMP designation for new gene therapy. Ophthalmology Times Europe. Published February 3, 2025. Accessed March 4, 2025. https://europe.ophthalmologytimes.com/view/ocugen-inc-receives-european-medicines-agency-atmp-designation-for-new-gene-therapy