FDA Approves Revakinagene Taroretcel (ENCELTO) for MacTel

Credit: US Food and Drug Administration

The US Food and Drug Administration (FDA) has approved revakinagene taroretcel-lwey (ENCELTO), formerly known as NT-501, for the treatment of Macular Telangiectasia type 2 (MacTel).¹

Announced by Neurotech Pharmaceuticals, on March 6, 2025, this approval marks the first and only FDA–approved treatment for MacTel, a neurodegenerative disease of the retina that can cause progressive and irreversible vision loss.

“I have seen the impact that MacTel can have on patients and their quality of life,” said Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas.¹ “Now with an FDA-approved treatment, I am confident that ENCELTO will be able to meaningfully slow disease progression for many patients affected by MacTel, allowing them the opportunity to preserve more functional vision over time.”

A bilateral neurodegenerative disease, MacTel is characterized by localized retinal degeneration, leading to vision loss and secondary alterations to the retinal vasculature. Its gradually progressive nature can notably impact a patient’s quality of life by impairing both distant and near vision.²

Revakinagene taroretcel was designed by Neurotech to deliver continuous therapeutic doses of ciliary neurotrophic factor (CNTF) to the retina using the company’s encapsulated cell therapy (ECT) technology. The platform is a cell-based gene therapy system designed for sustained delivery of therapeutic proteins, comprised of a small, semi-permeable capsule containing allogeneic retinal pigment epithelium (RPE) cells genetically engineered for targeted treatment.³

After surgical implantation, Neurotech indicated the semi-permeable exterior membrane allows essential nutrients to enter and therapeutic proteins to travel to the retina. Its exterior membrane can protect encapsulated RPE cells from the host's immune system, allowing for sustained survival and functionality.

Targeted to slow the progression of the disease, the submission and approval were based on positive results after implantation in two Phase 3 trials. These pivotal data showed revakinagene taroretcel significantly halted the loss of macular photoreceptors in patients with MacTel across 24 months.¹

"This is a historic moment for the MacTel community, as ENCELTO becomes the first-ever FDA-approved treatment for this vision-threatening disease,” said Thomas M. Aaberg Jr, MD, chief medical officer of Neurotech.¹ “For those who have been affected by MacTel and for all who have supported this journey, today we look forward to a future where vision loss from MacTel may be slowed."

In their announcement, Neurotech indicated that revakinagene taroretcel is expected to become available in the United States for patients with MacTel beginning in June 2025.¹

“Today marks an extraordinary milestone for patients, the retina community, and Neurotech,” said Richard Small, chief executive officer of Neurotech.¹ “I would like to express my gratitude to clinical study participants, clinical investigators and their teams, and the entire Neurotech organization who have helped make this a reality.”

References

1. Neurotech’s encelto(tm) (Revakinagene Taroretcel-lwey) approved by the FDA for the treatment of Macular Telangiectasia Type 2 (MacTel). neurotech. March 6, 2025. Accessed March 6, 2025. https://www.neurotechpharmaceuticals.com/neurotechs-enceltotm-revakinagene-taroretcel-lwey-approved-by-the-fda-for-the-treatment-of-macular-telangiectasia-type-2-mactel/.

2. Kedarisetti KC, Narayanan R, Stewart MW, Reddy Gurram N, Khanani AM. Macular Telangiectasia Type 2: A Comprehensive Review. Clin Ophthalmol. 2022;16:3297-3309. Published 2022 Oct 10. doi:10.2147/OPTH.S373538

3. Encapsulated cell therapy: Science & Technology. neurotech. January 14, 2025. Accessed March 6, 2025. https://www.neurotechpharmaceuticals.com/science-technology/.