Neurotech Pharmaceuticals is poised to make its commercial debut following the U.S. Food and Drug Administration’s (FDA) approval of its novel cell therapy technology. The approval, granted last week, marks a significant milestone for the privately held biotech company as it introduces its treatment for macular telangiectasia (MacTel) type 2, a rare and vision-threatening neurodegenerative condition.
The FDA has authorized Neurotech’s cell therapy implant, revakinagene taroretcel (branded as Encelto), to treat the debilitating disease. According to the company, this marks the first-ever FDA-approved treatment for MacTel, a condition that primarily affects the central vision and can lead to severe visual impairment.
Understanding MacTel Type 2
MacTel type 2 is a progressive disease in which small blood vessels at the center of the retina dilate. In some cases, new blood vessels form beneath the retina, a process known as neovascularization. These abnormal vessels can bleed or leak fluid, leading to swelling or thickening of the macula—the light-sensitive part of the retina responsible for central vision. This condition, which primarily affects middle-aged adults, can result in the loss of fine detail vision and typically impacts both eyes, though the severity may vary.
Encelto’s Innovative Approach
Encelto leverages Neurotech’s proprietary encapsulated cell therapy (ECT) platform, a cutting-edge technology designed to deliver therapeutic doses of ciliary neurotrophic factor directly to the retina. The encapsulated implant is made of a small, semipermeable capsule containing genetically engineered retinal pigment epithelium (RPE) cells that produce the therapeutic protein. Once implanted via surgery, the capsule’s exterior allows essential nutrients to enter while allowing therapeutic proteins to exit, effectively reaching the retina.
This unique mechanism offers a distinct advantage over traditional treatments for eye disorders, which often require frequent injections or refillable implants. Neurotech’s ECT platform aims to provide a continuous, localized treatment that could significantly reduce the burden on patients.
Successful Clinical Trials Lead to Approval
The FDA’s approval of Encelto was based on the results of two late-stage clinical trials, which demonstrated that the drug could significantly slow the progression of MacTel. Over a 24-month period, Encelto was shown to reduce the loss of macular photoreceptors by 56.4% and 29.2% compared to simulated treatment in the Phase 3 trials.
Dr. Thomas Aaberg Jr., Neurotech’s Chief Medical Officer, hailed the approval as a “historic moment” for the MacTel community. “For those who have been affected by MacTel and for all who have supported this journey, today we look forward to a future where vision loss from MacTel may be slowed,” he said.
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