On November 8, 2024, Neurotech Pharmaceuticals (Cumberland, RI, USA), a biotechnology company focused on sustained drug delivery for chronic retinal diseases, announced a three-month delay in the U.S. Food and Drug Administration’s (FDA) decision on its NT-501 treatment for Macular Telangiectasia Type 2 (MacTel).
This delay extends the Prescription Drug User Fee Act (PDUFA) goal date to March 18, 2025. Neurotech noted that the FDA required additional data to complete its review of the NT-501 biologics license application (BLA), which the company promptly provided.
“We are committed to providing the Agency any information needed to complete the review of the NT-501 BLA,” said Richard Small, Neurotech’s CEO, in a news release. “Neurotech will continue in our effort to bring this important therapy to MacTel patients.”
NT-501, or revakinagene taroretcel, uses Neurotech’s encapsulated cell therapy (ECT) platform, a novel cell-based system made with allogeneic retinal pigment epithelium (RPE) cells designed to continuously deliver therapeutic proteins to the retina.
The ECT platform involves a small capsule, surgically implanted into the patient’s vitreous and sutured to the sclera during an outpatient procedure. This capsule releases a neuroprotective protein known as CNTF (ciliary neurotrophic factor) directly to retinal cells while allowing essential nutrients to enter. The innovative design aims to slow degeneration and improve visual outcomes for patients with chronic retinal conditions.
MacTel is a rare and progressive neurodegenerative condition that affects central vision in both eyes due to localized retinal and vascular changes. Neurotech’s NT-501 therapy specifically targets the retinal degeneration characteristic of MacTel, aiming to provide sustained treatment to help preserve vision over time.
The BLA delay means additional waiting for patients and healthcare providers hoping for a new therapeutic option for MacTel, which has limited treatments. Neurotech’s ECT platform offers potential for long-term solutions in retinal care, and with NT-501’s promising clinical results, stakeholders are watching the FDA’s review process closely.
