News
Article
Author(s):
The approval is based on results from 2 phase 3 clinical trials that showed revakinagene taroretcel’s efficacy in macular telangiectasia type 2 (MacTel).
Updated Thursday, March 6, 2025, at 11:58 AM.
Image credit: ThawKyar | stock.adobe.com
Protocol A
Protocol B
The FDA approved revakinagene taroretcel-lwey (Encelto; Neurotech Pharmaceuticals, Inc.) for the treatment of patients with macular telangiectasia type 2 (MacTel). With this approval, revakinagene taroretcel has become the first and only FDA-approved treatment for MacTel. The approval was based on results from 2 phase 3 trials, which demonstrated that revakinagene taroretcel significantly slowed the loss of macular photoreceptors in patients with MacTel over 24 months.1
Revakinagene taroretcel is an allogeneic encapsulated cell-based gene therapy indicated for the treatment of adults with idiopathic MacTel type 2. It is intended for surgical intravitreal implantation under aseptic conditions by a qualified ophthalmologist. The recommended dose is 1 implany per affected eye, which contains 200,000 to 440,000 allogeneic retinal pigment epithelial cells expressing recombinant human ciliary neurotropic factor.1
Encapsulated cell therapy is a unique and versatile platform with the ability to continually produce and deliver targeted therapeutics, both alone and in combination, to the back of the eye long-term, protecting the cells from host immunological components. Recombinant cell lines derived from transfected NTC-200 cells are able to secrete major classes of therapeutics, including antibodies, fusion proteins, and growth factors, potentially enabling the ECT platform to treat a broad array of chronic eye diseases.2
MacTel type 2 is a gradually progressive disease that affects quality of life by impairing both near and distant vision, according to an abstract published in Clinical Ophthalmology. Previously, the disease was considered a vascular condition; however, recent evidence suggests it is better defined as neurodegenerative etiology, with primary involvement of Muller cells. In advanced cases of MacTel, both retinal pigment epithelium hyperplasia and subretinal neovascularization are responsible for most of the vision loss.3
“I have seen the impact that MacTel can have on patients and their quality of life,” clinical investigator Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, Houston, TX, said in a news release. “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.”1
Results from 2 randomized, multicenter phase 3 clinical trials, referred to as Protocol A (NCT03316300)4 and Protocol B (NCT03319849),5 demonstrated the safety and efficacy of revakinagene taroretcel in the treatment of MacTel. The trials enrolled and randomly assigned patients to receive either the revakinagene taroretcel implant (Protocol A: n = 58; Protocol B: n = 59) or a sham surgery (Protocol A: n = 57; Protocol B: n = 54) that mimicked the implant procedure.1,4,5
Compared with the sham procedure, revakinagene taroretcel demonstrated its ability to slow the loss of macular photoreceptors in patients with MacTel over a 24-month duration, according to a news release. It is expected to be available to US patients beginning June 2025.1
The most common adverse reactions (incidence ≥ 2%) reported by patients receiving revakinagene taroretcel included the following: 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; and iridocyclitis.1
"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, said in the news release. “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."1
