FDA grants Priority Review to novel treatment for macular telangiectasia type 2
Neurotech's ocular implant NT-501 utilizes encapsulated cell therapy to slow the progression of macular telangiectasia type 2 (MacTel). The implant was given a PDUFA goal date of December 17, 2024.
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Neurotech announced that the investigative implant revakinagene taroretcel (NT-501) has been approved for Priority Review of Biologics License Application (BLA) by the FDA and has been granted a prescription drug user fee act (PDUFA) goal date of December 17, 2024. The investigational implant leverages encapsulated cell therapy (ECT) to slow the progression of macular telangiectasia type 2 (MacTel), which is a neurogenerative retinal disease that causes central vision deterioration.1
The implant leverages ciliary neurotrophic factor (CNTF), which is a neuroprotective protein that promotes not only the survival of photoreceptors, but also the maintenance of them. Targeted therapy slows retinal degeneration via sustained delivery of CNTF, resulting in improved long-term visual outcomes for patients.
“This is a significant achievement for Neurotech,” said Richard Small, Chief Executive Officer. “I would like to express my gratitude to our employees for reaching this important milestone.
NT-501 delivers sustained therapeutic doses of CNTF to the retina to slow the progression of MacTel and the resulting vision loss. The delivery system is designed to deliver proteins directly to the retina, meaning that the applications may extend beyond MacTel and into the treatment of other retinal diseases in the future.
Neurotech released topline results in November 2022 for 2 replicative phase 3 clinical trials, which investigated ECT and NT-501 for the treatment of MacTel. Both demonstrated statistical significance for the pre-specified end point, which was the rate of change in ellipsoid zone (EZ) area loss from baseline through 24 months and which indicates a change in rate of photoreceptor loss.2 The positive results laid the foundation for the company's BLA.
While rare, MacTel is a challenging disease for patients. The progressive, neurodegenerative disease of the retina is characterized by localized retinal degeneration with secondary changes to the retinal vasculature. Because MacTel tends to affect both retinas, the resulting central vision loss can greatly affect patients' quality of life.
The implant, which is a small, semi-permeable capsule filled with proprietary allogeneic RPE cells, is surgically inserted into the patient's vitreous and sutured to the sclera in an outpatient procedure. The semi-permeable membrane is a significant part of the implant's success, as it allows essential nutrients to enter as the therapeutic proteins exit into the vitreous. The membrane also protects the encapsulated RPE cells from the immune system, which extends their long-term functionality.
