Retina recap: EMA and US FDA approvals, March 2025

Image credit: ©Jenn Miranda, via Canva. FDA and EMA logos courtesy respective agencies.

New clinical trial developments have been in the headlines throughout Q1 2025. In the month of March, breakthrough trials progressed and regulatory bodies such as the European Medicines Agency (EMA) and United States Food and Drug Administration (FDA) issued approvals. Here, the top approvals in March for new candidates and expanded uses of existing retina treatments.

EMA issues positive opinion for GA and Stargardt disease candidates

The EMA and its Committee for Advanced Therapies (CAT) has provided a positive opinion for 2 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. The classification will accelerate the regulatory review timeline of the gene therapies and 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 2025, when the EMA provided ATMP designation to OCU400, Ocugen's gene therapy indicated for treating retinitis pigmentosa.

FDA approves first treatment for MacTel

Among these notable approvals, the US FDA approved revakinagene taroretcel-lwey (ENCELTO) from Neurotech Pharmaceuticals for the treatment of Macular Telangiectasia type 2 (MacTel). The approval marked ENCELTO as is the first and only FDA-approved Treatment for MacTel.

ENCELTO utilises an encapsulated cell therapy (ECT) technology designed to continually deliver therapeutic doses of ciliary neurotrophic factor (CNTF) to the retina to assist in slowing the progression of the disease. Neurotech’s ECT platform is a cell-based gene therapy delivery system designed to provide long-term, sustained delivery of therapeutic proteins for the treatment of chronic eye diseases. The platform consists of a small, semi-permeable capsule that is surgically implanted, containing proprietary allogeneic retinal pigment epithelium (RPE) cells genetically engineered to produce specific therapeutic proteins for targeted disease treatment.

Experts told the Eye Care Network that the approval represents a major breakthrough in the MacTel space. “Approval for this treatment for this disease that is incurable right now...will be a wonderful situation for retina specialists and for the patients. First of all for the patients, and then for the retina specialists who will be able to provide treatment for this incurable disease,” said Anat Loewenstein, MD, professor and head of retina, Division of Ophthalmology, Tel Aviv Medical Center, VP Ambulatory Services; president, EURETINA; Sydney Fox Chair of Ophthalmology, Tel Aviv University; and president, Israeli Ophthalmological Society, Tel Aviv, Israel.

FDA approves expanded label for uveitis treatment

The FDA also approved an expanded label for fluocinolone acetonide intravitreal implant (ILUVIEN) that includes an indication for the treatment of chronic non-infectious uveitis affecting the posterior segment of the eye (NIU-PS). In addition to the expansion of this label, the approval includes updates to the label including to the Warnings and Precautions section. This treatment is already approved for both diabetic macular oedema and NIU-PS outside the US, including in 17 European countries.