FDA Approves Neurotech's ENCELTO for Macular Telangiectasia Type 2 (MacTel) Treatment

Neurotech Pharmaceuticals' ENCELTO (revakinagene taroretcel-lwey) has been approved by the FDA for the treatment of Macular Telangiectasia type 2 (MacTel), a neurodegenerative disease causing progressive and irreversible vision loss. ENCELTO utilizes encapsulated cell therapy to deliver therapeutic doses of ciliary neurotrophic factor (CNTF) to the retina, slowing disease progression. The approval is based on phase 3 trial results showing significant slowing of macular photoreceptor loss over 24 months. ENCELTO is expected to be available in the US starting June 2025.

Neurotech Pharmaceuticals' ENCELTO (revakinagene taroretcel-lwey) has recently received approval from the U.S. Food and Drug Administration (FDA) for the treatment of Macular Telangiectasia Type 2 (MacTel), a neurodegenerative condition causing progressive and irreversible vision loss [1]. ENCELTO is an innovative therapy that utilizes encapsulated cell therapy to deliver therapeutic doses of ciliary neurotrophic factor (CNTF) to the retina, thereby slowing disease progression [1].

The FDA's approval is based on the results of a phase 3 clinical trial, which demonstrated significant slowing of macular photoreceptor loss over 24 months [1]. The trial involved 7 participants with MacTel, and the primary safety outcome was a change in the parameters of the electroretinogram (ERG) [2]. The results showed that the ERG findings demonstrated a reduction in the amplitude of the scotopic b-wave in 4 participants three months after implantation, but all parameters returned to baseline values by month 12 and remained so at month 36 with no clinical impact on dark adaptation [2].

Furthermore, there was no change in visual acuity compared with baseline, and the area of the defect as measured functionally by microperimetry and structurally by the en face OCT imaging of the ellipsoid zone loss appeared unchanged from baseline [2]. These findings suggest that ENCELTO is safe and well-tolerated in eyes with MacTel.

MacTel is a bilateral degenerative condition of unknown etiology with characteristic neurosensory atrophy and perifoveal telangiectatic vessels which leak on fluorescein angiography [3]. The natural course of the disease is a gradual progressive bilateral loss of vision, occasionally accompanied by subretinal neovascularization, leading to severe vision loss [3]. Although the exact cause of MacTel is unknown, genetic studies have suggested a MacTel gene locus on chromosome 1 [3].

The approval of ENCELTO is a significant milestone in the treatment of MacTel, and it is expected to be available in the U.S. starting June 2025 [1]. Further evaluation in a randomized controlled clinical trial is warranted to test for efficacy and to explore the potential benefits of ENCELTO in a larger patient population.

References:

[1] Neurotech Pharmaceuticals. Press Release: FDA Approves ENCELTO™ (revakinagene taroretcel-lwey) for the Treatment of Macular Telangiectasia Type 2. [Link](https://www.neurotechpharma.com/news/press-releases/detail/2023/fda-approves-encelto-revakinagene-taroretcel-lwey-for-the-treatment-of-macular-telangiectasia-type-2)

[2] Sadda SR, Zangwill LM, Rosenfeld PJ, et al. Safety and tolerability of intraocular delivery of ciliary neurotrophic factor using an encapsulated cell implant for the treatment of macular telangiectasia type 2. Ophthalmology. 2013;120(10):2147-2153. [Link](https://pmc.ncbi.nlm.nih.gov/articles/PMC4361328/)

[3] National Eye Institute. Macular Telangiectasia Type 2 (MacTel). [Link](https://www.nei.nih.gov/health-topics/macular-telangiectasia/macular-telangiectasia-type-2-mactel)