FDA Approves Revakinagene Taroretcel for Macular Telangiectasia Type 2

The FDA approved revakinagene taroretcel-lwey (Encelto; Neurotech Pharmaceuticals) for the treatment of macular telangiectasia type 2 (MacTel), a neurodegenerative disease of the retina causing progressive and irreversible vision loss, Neurotech announced in a press release.1

Revakinagene taroretcel-lwey is the first and only FDA-approved treatment available for MacTel. | Image credit: wladimir1804 - stock.adobe.com

The approval of revakinagene taroretcel, an allogeneic encapsulated cell-based gene therapy, marks the first for a treatment for MacTel. The therapy is administered via surgical intravitreal implantation and uses an encapsulated cell therapy (ECT) technology that continually delivers therapeutic doses of ciliary neurotrophic factor (CNTF) to the retina to slow MacTel progression.

"This is a historic moment for the MacTel community, as [revakinagene taroretcel] becomes the first-ever FDA-approved treatment for this vision-threatening disease,” Thomas M. Aaberg Jr, MD, chief medical officer at Neurotech, said in a statement.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."

Results from 2 replicative phase 3 trials supported the approval, with patients who underwent treatment experiencing significantly slowed loss of macular photoreceptors over 24 months.1,2 The trials were multicenter, randomized, sham-controlled studies assessing the safety and efficacy of the implant for patients with MacTel.2 The main end point was the rate of change in ellipsoid zone area loss, which correlates to photoreceptor loss, with the trials also evaluating safety.

In the protocol A trial, revakinagene taroretcel demonstrated a 56.4% rate of reduction (P <.0001) in treated patients, while the protocol B trial showed a 29.2% rate of reduction (P = .021).

MacTel is a neurodegenerative disease characterized by localized retinal degeneration, which causes gradual loss of cells in the retina, leading to vision loss and alterations of the retinal vasculature. The first-in-class ECT delivery system was designed to facilitate long-term therapeutic protein administration for chronic eye disease following surgical implantation.

"I have seen the impact that MacTel can have on patients and their quality of life,” Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, Houston, TX, a clinical investigator, said in a statement.1 “Now with an FDA-approved treatment, I am confident that [revakinagene taroretcel] 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.”

References

1. Neurotech’s Encelto (revakinagene taroretcel-lwey) approved by the FDA for the treatment of macular telangiectasia type 2 (MacTel). News release Neurotech. March 6, 2025. https://www.neurotechpharmaceuticals.com/wp-content/uploads/Neurotech_Press-Release_BLA_Approval_FINAL.pdf

2. Neurotech Pharmaceuticals, Inc. announces positive phase 3 topline results for NT-501 implant in macular telangiectasia type 2. News release. November 2, 2022. https://www.neurotechpharmaceuticals.com/wp-content/uploads/Neurotech-Topline-PR__FINAL_11022022.pdf