Ciliary neurotrophic factor

Neurotech Pharmaceuticals announced the FDA extended the PDUFA goal date for NT-501's BLA to March 18, 2025, for MacTel treatment. NT-501 uses ECT to deliver CNTF, aiming to slow retinal degeneration. MacTel is a rare disease causing central vision deterioration. ECT provides sustained therapeutic protein delivery for chronic retinal diseases.

Neurotech Pharmaceuticals announced the FDA extended the PDUFA goal date for NT-501's BLA to March 18, 2025, to review additional data. NT-501, using the ECT platform, delivers CNTF for treating MacTel, a rare retinal disease causing vision loss. The ECT platform enables sustained therapeutic protein delivery, aiming to slow retinal degeneration.

Neurotech updates on Biologics License Application (BLA) for NT-501, targeting Macular Telangiectasia Type 2 (MacTel) treatment.

NT-501 ocular implant showed functional and structural benefits in MacTel patients, with a 36% reduction in EZ area and 68% reduction in reading speed loss over 2 years, according to data presented at AAO 2024.

Neurotech Pharmaceuticals announced an FDA extension of the PDUFA goal date for NT-501's BLA review to March 18, 2025, to assess additional data. NT-501, using the ECT platform, aims to treat MacTel, a rare disease causing central vision deterioration, by delivering CNTF for chronic retinal diseases.

The Usher syndrome market, valued at US$ 1.2 Billion in 2023, is projected to grow to US$ 1.7 Billion by 2034, with a CAGR of 3.61%. This rare genetic disorder, causing hearing and progressive vision loss, is driven by gene mutations and advancements in therapeutic solutions like cochlear and retinal implants. The market benefits from rehabilitation techniques, R&D for novel treatments, and gene therapy. The US leads in patient pool and market size. Recent developments include ProQR Therapeutics' asset divestiture. Key treatments under development include NT-501 and Ultevursen, targeting vision loss in Usher syndrome.

The EU regulates ATMPs under Directive 2001/83/EC and Regulation EC No. 1394/2007, requiring marketing authorization for commercialization. ATMPs include gene, cell, and tissue-based therapies, with specific classifications like sCTMPs and TEPs. The EMA oversees evaluations, with CAT providing scientific recommendations. Orphan medicinal products receive special designations, offering incentives. Clinical trials and GMP compliance are mandatory for ATMP authorization.