The first drug to treat macular telangiectasia type 2 (MacTel) – a neurodegenerative disease of the retina – has been approved by the United States Food and Drug Administration (FDA).
Neurotech Pharmaceuticals expects the drug, Encelto (revakinagene taroretcel-lwey), will be available to consumers in the US by mid-year.
MacTel causes progressive and irreversible vision loss, significantly impacting patients’ quality of life. Encelto uses an encapsulated cell therapy technology, designed to continually deliver therapeutic doses of ciliary neurotrophic factor (CNTF) to the retina, to assist in slowing the progression of the disease.
In a media release,1 Neurotech Pharmaceuticals said Encelto is the first and only FDA-approved treatment available for MacTel. The approval was based on results from two phase 3 trials, which demonstrated that after placement of the implant, Encelto significantly slowed the loss of macular photoreceptors in MacTel patients over 24 months.
Encelto becomes the first-ever FDA-approved treatment for this vision-threatening disease
The company described the approval as an “extraordinary milestone for patients, the retina community, and Neurotech”.
“This is a historic moment for the MacTel community, as Encelto becomes the first-ever FDA-approved treatment for this vision-threatening disease,” said Chief Medical Officer, Dr Thomas M. Aaberg Jr.
Clinical investigator Dr Charles Wykoff, from Texas, said he was “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”.
Reference
- Neurotech Pharmaceuticals, Neurotech’s ENCELTOTM (revakinagene taroretcel-lwey) Approved by the FDA for the Treatment of Macular Telangiectasia Type 2 (MacTel) (media release, 6 March 2025) available at: neurotechpharmaceuticals.com/wp-content/uploads/Neurotech_Press-Release_BLA_Approval_FINAL.pdf [accessed 11 March 2025].
