Neurotech Pharmaceuticals announced that the FDA has extended the Prescription Drug User Fee Act (PDUFA) goal date for NT-501 (revakinagene taroretcel) by three months, setting the new date to March 18, 2025. This extension allows the FDA to review additional data provided by Neurotech in response to recent requests. NT-501 is a Biologics License Application (BLA) under review for the treatment of macular telangiectasia type 2 (MacTel).
Richard Small, CEO of Neurotech Pharmaceuticals, stated, "We are committed to providing the agency any information needed to complete the review of the NT-501 BLA. Neurotech will continue in our effort to bring this important therapy to MacTel patients."
MacTel and NT-501
MacTel is a rare neurodegenerative disease characterized by localized retinal degeneration and secondary alterations of the retinal vasculature. It typically affects both eyes, leading to a gradual deterioration in central vision. Currently, there are limited treatment options available, highlighting the unmet medical need for effective therapies.
NT-501 leverages Neurotech's Encapsulated Cell Technology (ECT) platform. This platform is designed for long-term, sustained delivery of therapeutic proteins for chronic retinal diseases. The ECT platform consists of a small, semi-permeable capsule containing proprietary allogeneic retinal pigment epithelium (RPE) cells genetically engineered to produce specific therapeutic proteins targeted at the disease.
NT-501 utilizes the ECT platform to deliver ciliary neurotrophic factor (CNTF) for the treatment of chronic retinal diseases, including MacTel. The priority review granted by the FDA in June underscores the potential significance of NT-501 in addressing this rare and debilitating condition.
