FDA Approves Innovative Eye Implant to Combat Vision Loss in Rare Retina Disease

On March 6, 2025, the FDA granted approval for ENCELTO, a pioneering eye implant designed to slow the progression of macular telangiectasia type 2 (MacTel), a rare and degenerative retinal disease. This development marks a significant breakthrough, as prior to this, there were no approved treatments specifically targeting the advancement of this condition.

Developed by Neurotech Pharmaceuticals, ENCELTO stems from a collaborative effort involving the Scripps Research Institute and the Lowy Medical Research Institute. The device is set to be available to U.S. patients by June 2025. It functions by continuously releasing ciliary neurotrophic factor (CNTF), a protein naturally supporting nerve cell health in the retina, acting as a neuroprotectant to delay cell degeneration.

MacTel primarily affects the central part of the retina, impairing sharp, detailed vision essential for tasks like reading and facial recognition. The condition involves neurodegeneration—progressive death of photoreceptors—rather than vascular abnormalities as previously believed. The FDA's approval was based on two phase 3 clinical studies demonstrating that ENCELTO slowed the loss of light-sensitive retinal cells over 24 months. Remarkably, implants removed after as long as 14.5 years still produced effective CNTF levels, suggesting the device’s long-term potential.

This represents the first FDA-approved cell-based neuroprotective therapy for a neurodegenerative disease, highlighting the importance of basic science and collaboration in medical innovation. Friedlander, scientific advisor and president of the Lowy Medical Research Institute, emphasized the significance of this advancement, noting its potential to extend beyond MacTel to other neurodegenerative eye diseases like glaucoma and age-related macular degeneration.

The development of ENCELTO was motivated by early research indicating that gene therapy could deliver neuroprotectants directly to the retina. Friedlander’s team enhanced this approach with a collagen-based capsule containing genetically modified retinal pigment epithelial cells that safely and effectively release CNTF over time. This innovation offers hope for patients facing vision loss due to neurodegeneration, with ongoing research to explore additional therapeutic applications.

Ultimately, ENCELTO’s approval reflects years of dedicated research and clinical testing, embodying a significant milestone in neuroprotective treatments for retinal diseases, and demonstrating the potential for targeted, long-lasting therapies to improve quality of life for many patients.