FDA Approves First Treatment for Macular Telangiectasia Type 2 (MacTel)
The FDA has approved ENCELTO (revakinagene taroretcel-lwey) as the first and only treatment for macular telangiectasia type 2 (MacTel)123.
ENCELTO is an allogeneic encapsulated cell-based gene therapy developed by Neurotech Pharmaceuticals12.
The therapy utilizes encapsulated cell technology (ECT) to deliver sustained doses of ciliary neurotrophic factor (CNTF) to the retina14.
MacTel is a rare neurodegenerative disease of the retina that causes progressive and irreversible vision loss in adults13.
Approval was based on two phase 3 trials showing ENCELTO significantly slowed the loss of macular photoreceptors over 24 months12.
ENCELTO is administered as a single-dose implant containing 200,000 to 440,000 allogeneic retinal pigment epithelial cells expressing recombinant human CNTF16.
The treatment is expected to be available in the United States starting in June 202512.
Common side effects include conjunctival hemorrhage, eye pain, blurred vision, and delayed dark adaptation36.
ENCELTO is contraindicated in patients with ocular or periocular infections or known hypersensitivity to endothelial serum-free media6.
The approval marks a significant milestone for the MacTel community, offering the first FDA-approved treatment option for this vision-threatening disease124.
Sources:
1. https://www.biospace.com/press-releases/neurotechs-encelto-revakinagene-taroretcel-lwey-approved-by-the-fda-for-the-treatment-of-macular-telangiectasia-type-2-mactel
2. https://www.cgtlive.com/view/neurotech-encapsulated-cell-therapy-encelto-approved-fda-macular-telangiectasia-type-2
3. https://www.webmd.com/eye-health/news/20250307/fda-oks-first-gene-therapy-implant-for-a-rare-eye-disease
4. https://www.fightingblindness.org/news/neurotech-s-encapsulated-cell-technology-receives-fda-approval-for-treatment-of-mactel-1960
6. https://glance.eyesoneyecare.com/stories/2025-03-06/fda-approves-neurotech-s-encelto-cell-therapy-for-mactel/