March 6 marked an important milestone for our patients: the FDA approval of the first treatment for a new disease indication, Macular Telangiectasia type 2 (MacTel). This treatment utilizes an encapsulated cell therapy technology designed to continually deliver ciliary neurotrophic factor to the retina to slow the progression of the disease. Results from the two phase 3 registration trials showed that this surgical implant significantly decreased the loss of macular photoreceptors in MacTel patients over 24 months. The treatment is expected to be available in the United States for patients starting in June 2025.
The Duke Reading Center, led by Director Glenn Jaffe, MD and involving a team including Sina Farsiu, PhD, Eleonora Lad, MD, PhD, and Sandra Stinnett, PhD, performed the data analyses for the registration trials and was central to the FDA approval. These trials also pioneered the use of a novel clinical trial endpoint, ellipsoid zone (photoreceptor) loss area, which is already being employed in clinical trials of other retinal diseases.