ENCELTO becomes the first and only FDA-approved therapy for macular telangiectasia type 2.
Neurotech Pharmaceuticals (Rhode Island, United States) announced that the U.S. Food and Drug Administration (FDA) has approved ENCELTO (revakinagene taroretcel-lwey) for the treatment of idiopathic macular telangiectasia type 2 (MacTel).
With this approval, ENCELTO becomes the first and only FDA-approved treatment for MacTel, a rare neurodegenerative retinal disease that leads to irreversible vision loss. The drug is expected to become available to U.S. patients starting in June 2025.
The FDA approval is based on two Phase III clinical trials, which demonstrated that ENCELTO significantly slowed the loss of macular photoreceptors over 24 months in patients with MacTel.1
“This is a historic moment for the MacTel community, as ENCELTO becomes the first-ever FDA approved treatment for this vision-threatening disease,” said Dr. Thomas M. Aaberg Jr., chief medical officer at Neurotech Pharmaceuticals. “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.”
Neurotech Pharmaceuticals announces the FDA approval for ENCELTO on LinkedIn
How ENCELTO works
ENCELTO is an allogeneic encapsulated cell therapy (ECT) that delivers ciliary neurotrophic factor (CNTF) directly to the retina. It features an intravitreal implant containing genetically engineered human epithelial cells, which continuously produce and release CNTF to the affected area.
By maintaining a consistent therapeutic presence, this approach aims to support macular photoreceptor preservation and slow the progression of vision loss related to MacTel.
Insights from experts and Neurotech CEO
Dr. Charles C. Wykoff, MD, PhD, of Retinal Consultants of Texas and a clinical investigator, emphasized the impact of the new therapy.
“I have seen the impact that MacTel can have on patients and their quality of life. Now with an FDA-approved treatment, I am confident that ENCELTO 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.”
“Today marks an extraordinary milestone for patients, the retina community and Neurotech,” said Richard Small, CEO of Neurotech Pharmaceuticals. I would like to express my gratitude to clinical study participants, clinical investigators and their teams, and the entire Neurotech organization who have helped make this a reality.”
More information regarding patient access, distribution and prescribing guidelines will be released in the coming months.
For more on this story, see Neurotech’s full press release announcing the FDA approval.
Reference
- Neurotech Pharmaceuticals, Inc. announces positive phase 3 topline results for NT-501 implant in macular telangiectasia type 2. Press release. November 2, 2022. https://www.neurotechpharmaceuticals.com/wp-content/uploads/Neurotech-Topline-PR__FINAL_11022022.pdf Accessed on March 6, 2025.
