FDA
Ocular implant approved for macular telangiectasia
Brand name: Encelto
Generic name: revakinagene taroretcel-lwey
Manufacturer: Neurotech Pharmaceuticals, Inc.
Approval date: March 6, 2025
FDA approved Encelto (revakinagene taroretcel-lwey), an allogeneic encapsulated cell-based gene therapy, for the treatment of adults with idiopathic macular telangiectasia type 2 (MacTel).
MacTel is a neurodegenerative disease of the retina that causes progressive and irreversible vision loss.
Efficacy
Approval was based on data from two phase 3 trials (NTMT-03-A [NCT03316300] and NTMT-03-B [NCT03319849]) that compared the safety and efficacy of revakinagene taroretcel with sham implant in MacTel patients. The primary endpoint in both trials was the rate of change in ellipsoid zone area loss from baseline to month 24. In both trials, treatment with revakinagene taroretcel statistically significantly slowed the loss of macular photoreceptors relative to sham treatment.
Safety
The most common adverse reactions reported in the trials (incidence ≥2%) included conjunctival hemorrhage, delayed dark adaptation, foreign body sensation, eye pain, suture related complications, miosis, conjunctival hyperemia, eye pruritus, ocular discomfort, vitreous hemorrhage, blurred vision, headache, dry eye, eye irritation, cataract progression or formation, vitreous floaters, severe vision loss, eye discharge, anterior chamber cell, iridocyclitis.
Sources:
Neurotech Pharmaceuticals. Encelto (revakinagene taroretcel-lwey) [package insert]. U.S. Food and Drug Administration. Revised March 2025. Accessed March 11, 2025. https://www.fda.gov/media/185726/download?attachment
Neurotech’s Encelto™ (revakinagene taroretcel-lwey) approved by the FDA for the treatment of macular telangiectasia type 2 (MacTel). [News release]. Neurotech Pharmaceuticals. 2025. https://www.neurotechpharmaceuticals.com/wp-content/uploads/Neurotech_Press-Release_BLA_Approval_FINAL.pdf