US FDA approves revakinagene taroretcel-lwey (ENCELTO) for macular telangiectasia type 2

MacTel is a bilateral, neurodegenerative disease of the retina in adults that causes progressive and irreversible vision loss. Image credit: ©gguy – stock.adobe.com

The US Food and Drug Administration (FDA) has approved revakinagene taroretcel-lwey (Encelto) from Neurotech Pharmaceuticals for the treatment of Macular Telangiectasia type 2 (MacTel).

At the date of approval, Encelto is the first and only FDA-approved Treatment for MacTel. Encelto utilises 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.

The company stated in a press release that the approval from the FDA was based on results from two phase 3 trials. Results from the trials showed that after placement of the implant, Encelto significantly slowed the loss of macular photoreceptors in MacTel patients over 24 months.

Richard Small, CEO of Neurotech Pharmaceuticals, and Charles C Wykoff, MD, PhD, Retinal Consultants of Texas and clinical investigator, commented on the approval in a press release from the company.

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

“I have seen the impact that MacTel can have on patients and their quality of life,” said Wykoff. “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.”

MacTel is a bilateral, neurodegenerative disease of the retina in adults with characteristic localised retinal degeneration that causes progressive and irreversible vision loss and secondary alterations of the retinal vasculature.

The company stated that Encelto is expected to be available in the United States for patients starting in June 2025.

Reference

1. Neurotech’s ENCELTO (revakinagene taroretcel-lwey) Approved by the FDA for the Treatment of Macular Telangiectasia Type 2 (MacTel). Press release. Neurotech Pharmaceuticals. Published 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/