FDA Approves Revakinagene for MacTel

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

Encelto was evaluated in 2 phase III clinical trials (protocol A, NCT03316300; protocol B, NCT03319849), data from which were reported in November 2022. The randomized, sham-controlled, multicenter studies were primarily assessing the rate of change in ellipsoid zone area loss, which correlates to photoreceptor loss, from baseline through 24 months as well as the safety of the implant and procedure. It was found that the protocol A trial had a 56.4% rate of reduction (P <.0001) in treated patients and protocol B had a 29.2% rate of reduction (P = .021).