MedPath

FDA Extends Review Timeline for Neurotech's NT-501 MacTel Treatment

Neurotech Pharmaceuticals announced an update on its biologics license application for NT-501, a treatment for macular telangiectasia type 2 (MacTel), with the FDA extending the review period by three months to March 18, 2025, to allow for additional data evaluation.

Neurotech Pharmaceuticals has provided an update on the status of its biologics license application (BLA) for NT-501 (revakinagene taroretcel), a novel treatment aimed at addressing macular telangiectasia type 2 (MacTel). The U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) goal date by three months, shifting the anticipated decision date from December 17, 2024, to March 18, 2025. This extension is intended to afford the FDA additional time to thoroughly assess supplemental data submitted by Neurotech in response to specific requests from the agency.
Understanding Macular Telangiectasia Type 2 (MacTel)
MacTel is a rare, slowly progressive retinal disease characterized by localized degeneration of the retina and distinctive vascular changes in the retinal blood vessels. The condition typically affects both eyes, leading to a gradual loss of central vision. Currently, there are limited treatment options available for MacTel, highlighting the potential significance of NT-501 as a transformative therapeutic option for patients.
The ECT Platform: A Novel Approach to Chronic Retinal Diseases
NT-501 utilizes Neurotech’s Encapsulated Cell Therapy (ECT) platform, an innovative cell-based delivery system designed for the continuous, sustained release of therapeutic proteins to target chronic retinal diseases. The ECT platform comprises a small, semi-permeable capsule containing genetically engineered retinal pigment epithelium (RPE) cells. These cells are modified to produce specific therapeutic proteins, offering a targeted, long-term therapeutic effect directly to the retina.
How NT-501 Works for MacTel Treatment
NT-501 is engineered to deliver Ciliary Neurotrophic Factor (CNTF) to the retina through the ECT platform. CNTF has demonstrated potential in addressing the underlying pathology of chronic retinal diseases like MacTel by supporting retinal cell health and function. The controlled release mechanism of the ECT system ensures consistent and localized treatment, aiming to slow disease progression and preserve vision in individuals affected by MacTel.
Neurotech’s update marks a significant advancement in the potential availability of NT-501 for MacTel patients, a condition with currently limited treatment options. The extended PDUFA goal date to March 2025 represents the latest timeline for a decision on this innovative therapy.
Subscribe Icon

Stay Updated with Our Daily Newsletter

Get the latest pharmaceutical insights, research highlights, and industry updates delivered to your inbox every day.

Related Topics

Nuvation Bio's Taletrectinib Gains FDA Priority Review and Expanded Access Program for ROS1+ NSCLC

The FDA granted priority review to Nuvation Bio's NDA for taletrectinib, a next-generation ROS1 inhibitor, for advanced ROS1+ non-small cell lung cancer, setting a PDUFA date of June 23, 2025.
Taletrectinib demonstrated promising efficacy and tolerability in the TRUST-I and TRUST-II trials, showing durable responses and prolonged progression-free survival in ROS1+ NSCLC patients.

FDA Lifts Partial Hold on BioNTech and OncoC4's BNT316 Trial for Squamous NSCLC

The FDA has lifted the partial clinical hold on the Phase 3 PresERVE-003 trial evaluating BNT316 (gotistobart) in metastatic non-small cell lung cancer (NSCLC).
Enrollment will continue solely for patients with squamous NSCLC, following alignment with the FDA based on available trial data.

FDA Approves Companion Diagnostic for Tepotinib in mNSCLC with MET Exon 14 Skipping Alterations

The FDA approved FoundationOne Liquid CDx as a companion diagnostic for tepotinib, aiding in identifying mNSCLC patients with MET exon 14 skipping alterations.
Tepotinib received regular approval in February 2024 for mNSCLC patients with MET exon 14 skipping alterations, following an earlier accelerated approval in 2021.

Tonix Pharmaceuticals' TNX-102 SL Awaits FDA Decision for Fibromyalgia Treatment

Tonix Pharmaceuticals' TNX-102 SL, a non-opioid analgesic, has been assigned an FDA PDUFA goal date of August 15, 2025, for fibromyalgia treatment, potentially offering a new option for millions.
TNX-102 SL demonstrated significant pain reduction and improved sleep quality in Phase 3 trials, addressing key fibromyalgia symptoms with a well-tolerated safety profile.

FDA Extends Review of Neurotech's NT-501 for Macular Telangiectasia Type 2

The FDA has extended the PDUFA goal date for Neurotech's NT-501 BLA by three months, now set for March 18, 2025, to allow for review of additional data.
NT-501 is an encapsulated cell therapy delivering ciliary neurotrophic factor (CNTF) for treating Macular Telangiectasia Type 2 (MacTel).

FDA Grants Priority Review to TLX101-CDx for Glioma Imaging

The FDA has granted priority review to Telix's TLX101-CDx (18F-FET), an imaging agent for progressive or recurrent glioma, with a decision expected by April 26, 2025.
TLX101-CDx targets LAT1 and LAT2 membrane transport proteins and aims to improve brain cancer imaging, addressing the limitations of conventional MRI.

NT-501 Encapsulated Cell Therapy Shows Promise in Glaucoma Neuroprotection

Phase II study shows NT-501 implant is well-tolerated in primary open-angle glaucoma patients, with no severe adverse events reported.
The therapy maintains intraocular pressure and supports retinal health, crucial for glaucoma management, over a 24-month period.

Find Therapeutics Doses First Subject in Phase I Trial of FTX-101 for Chronic Optic Neuropathy

Find Therapeutics initiated a Phase I clinical trial of FTX-101, a potential treatment for chronic optic neuropathy, after receiving FDA approval.
The Phase I trial is a two-part study evaluating the safety, tolerability, and pharmacokinetics of FTX-101 versus placebo in healthy volunteers.

Neuraptive's NTX-001 Receives FDA Breakthrough Therapy Designation for Peripheral Nerve Injury

NTX-001, a drug-device combination by Neuraptive Therapeutics, has received Breakthrough Therapy Designation from the FDA for treating peripheral nerve injuries requiring repair.
The designation was granted based on promising Phase 2 NEUROFUSE study results, potentially accelerating NTX-001's development and review process.

FDA Extends Review of Neurotech's NT-501 for Macular Telangiectasia Type 2

The FDA has extended the PDUFA goal date for Neurotech's NT-501 BLA by three months, now set for March 18, 2025, to allow for review of additional data.
NT-501 (revakinagene taroretcel) is under review for treating macular telangiectasia type 2 (MacTel), a rare neurodegenerative disease causing gradual vision loss.

Reference News

[1]
Neurotech FDA Update on NT-501 MacTel Treatment| OBN
ophthalmologybreakingnews.com · Jan 7, 2025

Neurotech Pharmaceuticals' NT-501, a treatment for MacTel, faces a delayed FDA decision to March 18, 2025, due to extend...

© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy