FDA and EMA approvals in retina this month
In the month of March, not only did trials progress, but approvals for new and expanded uses of treatments were issued.
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With companies around the globe working to development new candidates for treating patients with retinal diseases, there are always new clinical trial developments in the headlines. In the month of March, not only did trials progress, but approvals for new and expanded uses of treatments were issued.
The first FDA approved treatment for MacTel
Among these notable approvals, the US Food and Drug Administration (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 utilizes 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.
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 edema (DME) and NIU-PS outside the US, including in 17 European countries.
EMA issues positive opinion for GA and Stargardt disease candidates
The European Medicines Agency (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.
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