Neurotech Pharmaceuticals’ eye cell therapy implant has faced a setback as the US Food and Drug Administration (FDA) has extended its Prescription Drug User Fee Act (PDUFA) date.
The US regulatory agency has changed the PDUFA date for the therapy’s biologics license application (BLA) for NT-501 (revakinagene taroretcel) to 18 March 2025, three months after the original date of 17 December 2024 the FDA set back in June. The agency stated that it needed the extra time to “review additional data provided by the Neurotech in response to recent requests from the FDA”.
The company is seeking approval for the NT-501 implant as a treatment for macular telangiectasia type 2 (MacTel), a rare ocular neurodegenerative disease. The implant received fast track designation for this indication in December 2018.
The NT-501 cell therapy implant contains allogeneic retinal pigment epithelium (RPE) cells. The implant has a semipermeable membrane that allows the therapeutic proteins like the ciliary neurotrophic factor (CNTF), produced by the RPE cells, to exit whilst protecting these cells from the host’s immune system, providing a targeted and continuous treatment.
The company has previously shared that two parallel Phase III trials (protocol A, NCT03316300; protocol B, NCT03319849) of NT-501 met its primary endpoint of the rate of change in ellipsoid zone area loss, which correlates to loss of photoreceptors and sight, from baseline through 24 months.
The rate of reduction of progression of disease in the treatment group in protocol A and B studies was 56.4% and 29.2%, respectively. The company also noted that the implant was safe, durable, and well tolerated in both trials.
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By GlobalData
Multiple companies are developing cell and gene therapies for eye disorders. SparingVision has an adeno-associated virus-based gene therapy, SPVN20, for improving visual acuity in patients with advanced and late-stage retinitis pigmentosa. The therapy is being evaluated in patients with stage two and three retinitis pigmentosa patients in the Phase I/II PRODYGY study (NCT05748873).
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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