FDA Approves First Cell Therapy for Rare Eye Disease
March 12, 2025
The Food and Drug Administration recently
approved revakinagene taroretcel-lwey (Encelto), an allogeneic encapsulated cell-based gene therapy, for the treatment of adults with idiopathic macular telangiectasia type 2 (MacTel). This marks the first and only FDA-approved therapy for the rare neurodegenerative eye disease, which causes progressive and irreversible vision loss.
Revakinagene taroretcel uses an encapsulated cell therapy technology designed to continuously deliver therapeutic doses of ciliary neurotrophic factor (CNTF) to the retina to slow disease progression. The therapy is administered through a single surgical procedure performed by a qualified ophthalmologist.
FDA's approval was supported by results from two phase 3 clinical trials, manufacturer Neurotech Pharmaceuticals
said in a media release. Results from the studies demonstrated that revakinagene taroretcel significantly slowed the loss of macular photoreceptors in patients with MacTel during a 24-month period following implant placement.
Neurotech expects the treatment to be available in the United States beginning in June.
