Neurotech Provides Update on BLA for NT-501 as a Treatment for Macular Telangiectasia Type 2

Neurotech Pharmaceuticals announced that the FDA has extended the Prescription Drug User Fee Act (PDUFA) goal date by 3 months to allow time required for the FDA to review additional data provided by the company in response to recent requests from the FDA. Originally set for December 17, 2024, the new PDUFA goal date for the biologics license application (BLA) of NT-501 (revakinagene taroretcel) as a treatment for macular telangiectasia type 2 (MacTel) is March 18, 2025. In June, the FDA granted priority review for NT-501.

“We are committed to providing the agency any information needed to complete the review of the NT-501 BLA,” Richard Small, CEO of Neurotech Pharmaceuticals, said in a company news release. “Neurotech will continue in our effort to bring this important therapy to MacTel patients.

MacTel is a rare neurodegenerative disease with characteristic localized retinal degeneration and secondary alterations of the retinal vasculature. MacTel typically affects both eyes and causes a gradual deterioration in central vision. The ECT platform is a cell-based delivery system designed to provide long-term, sustained delivery of therapeutic proteins for the treatment of chronic retinal diseases. This platform consists of a small, semi-permeable capsule containing proprietary allogeneic retinal pigment epithelium (RPE) cells genetically engineered to produce specific therapeutic proteins for targeted disease treatment.

NT-501 leverages the company's ECT platform to deliver CNTF for the treatment of chronic retinal diseases, such as MacTel.