FDA Approves Revakinagene Taroretcel-Lwey (ENCELTO) for Macular Telangiectasia Type 2

The US Food and Drug Administration (FDA) has granted approval for revakinagene taroretcel-lwey (ENCELTO), developed by Neurotech Pharmaceuticals, marking the first-ever FDA-approved treatment for Macular Telangiectasia type 2 (MacTel). ENCELTO employs encapsulated cell therapy (ECT) technology to provide continuous delivery of ciliary neurotrophic factor (CNTF) to the retina, effectively slowing disease progression.

A Breakthrough in MacTel Treatment

ENCELTO represents a significant advancement in ophthalmology, as MacTel is a progressive and irreversible degenerative retinal disease. Until now, no FDA-approved treatment was available for patients suffering from this condition. The therapy utilizes Neurotech’s ECT platform, a gene therapy-based system that enables sustained, long-term delivery of therapeutic proteins to treat chronic eye diseases.

The ECT technology involves the surgical implantation of a small, semi-permeable capsule containing proprietary allogeneic retinal pigment epithelium (RPE) cells. These cells are genetically modified to continuously produce CNTF, offering targeted therapeutic effects to slow the degeneration of macular photoreceptors in MacTel patients.

Clinical Trials and FDA Approval

The FDA approval was based on compelling results from two Phase 3 clinical trials, which demonstrated the efficacy of ENCELTO in slowing vision loss over a 24-month period. The trials showed that the placement of the implant significantly reduced the rate of macular photoreceptor loss, a key factor in the progression of MacTel.

Richard Small, CEO of Neurotech Pharmaceuticals, emphasized the importance of this approval in a company press release:

“Today marks an extraordinary milestone for patients, the retina community, and Neurotech. 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.”

Charles C. Wykoff, MD, PhD, from Retinal Consultants of Texas and a clinical investigator, added:

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

Understanding Macular Telangiectasia Type 2

MacTel is a neurodegenerative retinal disease that primarily affects adults and leads to progressive vision loss. It is characterized by localized retinal degeneration and secondary alterations in retinal vasculature, significantly impacting patients' visual function.

The approval of ENCELTO provides hope for those diagnosed with MacTel, offering a novel therapeutic approach that targets the underlying disease mechanism rather than just managing symptoms.

Availability and Next Steps

Neurotech Pharmaceuticals announced that ENCELTO is expected to be available for eligible patients in the United States starting in June 2025. The company is working on a nationwide rollout to ensure accessibility to the new treatment.

With this approval, ENCELTO sets a precedent for innovative, cell-based gene therapy approaches in ophthalmology, reinforcing the role of sustained drug delivery systems in managing chronic eye diseases.