FDA Approves ENCELTO for Macular Telangiectasia Type 2

Summary: ENCELTO has received FDA approval as the first treatment for macular telangiectasia type 2 (MacTel), a rare eye disease that gradually affects central vision. This innovative device, which is surgically implanted, delivers a continual supply of a neuroprotective protein known as CNTF, aimed at preserving retinal cells and slowing the disease’s progression.

Developed by Neurotech Pharmaceuticals, with foundational work from Scripps Research and the Lowy Medical Research Institute, ENCELTO has demonstrated long-term effectiveness in clinical trials. Its approval is significant, as it’s the first cell-based implant designed to administer a neuroprotectant for such conditions.

Key Facts:

• First-of-Its-Kind Treatment: ENCELTO stands out as the first approved therapy for MacTel and the inaugural implant offering a neuroprotectant for neurodegenerative diseases.
• Long-Term Efficacy: Trials showed that the device continued to produce CNTF for up to 14.5 years post-implantation, displaying strong effects over two years.
• Broader Potential: Researchers are exploring ENCELTO’s application in other diseases like glaucoma and age-related macular degeneration.

Context: For individuals diagnosed with macular telangiectasia type 2, there were no FDA-approved treatments available to help slow or stop vision loss until now.

This changed on March 6, 2025, with the green light for ENCELTO, a device aimed at maintaining sight for those affected by this condition.

Neurotech Pharmaceuticals spearheaded ENCELTO’s development, with significant contributions from the Scripps Research lab led by physician-scientist Martin Friedlander and the Lowy Medical Research Institute.

This is the 17th FDA-approved therapy stemming from Scripps Research efforts, and the device is expected to be accessible to U.S. patients in June 2025.

Friedlander commented on the collaborative efforts that propelled this project, highlighting the Lowy family’s dedication and the invaluable work by Neurotech and various international scientists and clinicians.

MacTel affects the central retina, crucial for fine detail vision, leading to challenges like reading and recognizing faces, which tend to worsen gradually.

ENCELTO functions by delivering a continuous supply of ciliary neurotrophic factor (CNTF), a protein that aids the survival and health of nerve cells, including the vital photoreceptors in the retina. Essentially, CNTF acts to protect these cells from damage, thereby delaying degeneration.

The FDA’s decision was bolstered by two phase 3 clinical studies that indicated the implant slowed the loss of light-sensing retinal cells over a two-year span.

Remarkably, devices that were removed after as long as 14.5 years continued to produce CNTF at levels close to those seen at the point of implantation, suggesting long-term efficacy beyond the results noted in the trials. Further exploration of this will take place in extended phase 4 trials.

“This is the first time a cell-based therapy delivering a neuroprotectant has been approved for treating a neurodegenerative condition,” Friedlander noted.

With a focus on ENCELTO’s scientific foundation, Friedlander played a crucial role in its development, building on earlier research conducted by his Scripps team.

Initially thought to stem from blood vessel issues, breakthrough research revealed that MacTel is primarily a neurodegenerative process, involving the death of essential retinal cells.

A vital proof-of-concept study published in 2009 established that gene therapy could effectively deliver neuroprotectants directly to the retina, mitigating cell loss.

This initial success led to the further development of ENCELTO. Following a family member’s diagnosis with MacTel, the Lowy family’s quest for a solution significantly fueled the development of this targeted therapy.

Friedlander sought to find a secure, consistent method for CNTF delivery and revisited Neurotech to investigate the potential of their device, which had initially been designed for other retinal conditions.

They looked into a small, collagen-based capsule containing genetically modified retinal pigment epithelial (RPE) cells. Once implanted, this device releases CNTF in a controlled manner while protecting RPE cells from the immune system’s attacks.

There’s ongoing examination of ENCELTO for treating other neurovascular diseases beyond MacTel. Friedlander’s lab is currently investigating its potential for delivering additional therapeutic molecules for conditions such as glaucoma and age-related macular degeneration.

Friedlander observes that since the retina is an extension of the brain, neuroprotectants like CNTF may also be beneficial in preventing neurodegeneration in other diseases.

He emphasizes that the success of ENCELTO is the result of years of diligent research, determination, and collaboration among many contributors, culminating in this promising treatment.

“For a clinician-scientist, finding a treatment that could impact so many patients is immensely fulfilling,” he expressed, underscoring the journey from concept to clinical application.