FDA Approves Encelto for treatment of Macular Telangiectasia Type 2

The US Food and Drug Administration (FDA) has approved Encelto (revakinagene taroretcel-lwey) for the treatment of macular telangiectasia type 2 (MacTel).

MacTel is a rare, progressive eye disease that affects the macula, leading to a gradual decline in central vision. Encelto™ is an allogeneic encapsulated cell-based gene therapy designed to slow retinal degeneration in MacTel patients. It works by delivering continuous ciliary neurotrophic factor (CNTF)-a protein that supports photoreceptor survival and maintenance-through an ocular implant.

MacTel is a neurodegenerative disease of the retina in adults that causes progressive and irreversible vision loss, significantly impacting patients' quality of life. ENCELTO utilizes 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. 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 is expected to be available in the United States for patients starting in June of 2025.

“Today marks an extraordinary milestone for patients, the retina community, and Neurotech,” said Richard Small, Chief Executive Officer. “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.”

“I have seen the impact that MacTel can have on patients and their quality of life,” said Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, Houston, TX, a clinical investigator. “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.”

"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, 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."

About Macular Telangiectasia Type 2 (MacTel)

Macular Telangiectasia Type 2 (MacTel), or idiopathic macular telangiectasia type 2, is a bilateral, neurodegenerative disease in adults with characteristic localized retinal degeneration, a group of diseases that cause the gradual loss of cells in the retina, resulting in vision loss and secondary alterations of the retinal vasculature, the network of blood vessels that supplies oxygen and nutrients to the retina.