The US Food and Drug Administration (FDA) has approved Encelto (revakinagene taroretcel-lwey; Neurotech Pharmaceuticals), an ocular insert that represents the first treatment for Macular Telangiectasia type 2 (MacTel).

Encelto (formerly known as NT-501) is an encapsulated cell therapy that is implanted in the vitreous; it is designed to deliver sustained therapeutic doses of ciliary neurotrophic factor (CNTF) to the retina. This technology is designed to slow the progression of MacTel, which has no other FDA-approved treatments.

The Encelto insert was shown to be effective and safe in the phase 2 study NTMT-02 and a pair of parallel phase 3 studies, NTMT-03A and NTMT-03B. Pooled data from the trials was reported at last fall’s American Academy of Ophthalmology meeting. The FDA based its approval on the phase 3 trial data, which showed that Encelto significantly slowed macular photoreceptor loss over 24 months.

MacTel is a rare, progressive neurodegenerative disease of the retina that leads to central vision loss. MacTel typically develops in middle-aged adults and has no known cure. In the United States, it is estimated that approximately 0.1% of the population, or around 250,000 people, have MacTel. Worldwide, the prevalence is believed to be similar, affecting roughly 2 million people.

“This is a historic moment for the MacTel community, as Encelto becomes the first-ever FDA-approved treatment for this vision-threatening disease,” said Thomas M. Aaberg Jr, MD, Neurotech’s chief medical officer. “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.”

Encelto is expected to be available in the United States in June 2025. RP