Macular telangiectasia (MacTel) type 2, a rare neurodegenerative eye condition, has long left many without access to effective treatment. This disease silently affects central vision, making everyday tasks like reading and recognizing faces increasingly challenging for those impacted. However, patients and biopharma professionals alike have reason to look ahead with renewed optimism. On March 6, 2025, Neurotech Pharmaceuticals marked a milestone in vision care with the FDA’s approval of Encelto, the first-ever treatment for MacTel type 2.
This approval is not just a victory for patients—it signifies a groundbreaking moment for cell therapy and gene therapy technologies tailored to address chronic eye diseases. For market access teams and biopharma professionals, Encelto represents a fascinating case study in therapeutic innovation.
What Is Macular Telangiectasia (MacTel) Type 2?
MacTel type 2 is a progressive and rare neurodegenerative condition affecting the macula, the central part of the retina responsible for sharp, detailed vision. The disease is characterized by the dilation of small blood vessels in the retina, often leading to neovascularization, bleeding, and fluid leakage. Over time, the structural integrity of the retina is compromised, resulting in loss of central vision.
Typically diagnosed in middle-aged adults, MacTel type 2 affects both eyes, although the severity may differ between them. According to current prevalence estimates, MacTel type 2 impacts around 0.1% of the population, translating to approximately 165,000 patients in the United States.
Despite its significant impact on patients’ quality of life, MacTel has historically lacked FDA-approved treatment options—until now.
Enter Encelto: A Cell Therapy Built for MacTel
Approved by the FDA in March 2025, Encelto marks a significant step forward for treating this rare condition. It leverages Neurotech’s proprietary encapsulated cell therapy (ECT) platform to deliver therapeutic proteins to the retina.
How Does Encelto Work?
Encelto’s approach to treating MacTel is both innovative and practical. The therapy is delivered via a small, implanted membrane loaded with genetically engineered retinal pigment epithelial (RPE) cells. These cells are programmed to produce ciliary neurotrophic factor (CNTF), a protein known to support the survival of photoreceptors in the retina.
The membrane’s semi-permeable exterior allows essential nutrients to nourish the implanted RPE cells while releasing CNTF to the retina. The key advantage? Continuous, localized protein delivery right where it’s needed most.
Unlike traditional treatments for retinal diseases, which often involve repeated injections, Encelto is designed for long-term use following a straightforward surgical procedure.
Clinical Data Supporting FDA Approval
Encelto’s approval was based on robust results from two Phase 3 trials. Over a 24-month period, the implant achieved remarkable outcomes, reducing the rate of disease progression by 56.4% in one trial and 29.2% in the other.
Commercial Debut & Market Implications
Encelto is set to roll out in the U.S. starting June 2025. This launch positions Neurotech Pharmaceuticals to make its commercial debut, with Encelto leading as the cornerstone of the company’s portfolio.
Neurotech’s CEO, Richard Small, emphasized the long-term aspirations of the therapy, describing it as a solution designed to alleviate the burden placed on patients by the previously unmet need for effective MacTel treatment. While the company has not disclosed pricing details, discussions are ongoing to ensure market access and affordability.
From a market access perspective, Encelto presents a compelling case for payers and providers:
- First-to-market advantage as the only FDA-approved treatment for MacTel.
- Evidence demonstrating its value through reduced disease progression.
- Improved patient adherence compared to repeat injection regimens.
These factors set the stage for strategic opportunities in educating both healthcare professionals and patients about Encelto’s benefits.
Broader Implications for Biopharma & Cell Therapy
Encelto also offers critical insights that extend beyond ophthalmology. For biopharma professionals, the therapy stands out as a breakthrough in targeted cell-based gene therapy. Neurotech’s ECT platform, which enables sustained protein delivery, exemplifies how cell-based solutions can be scaled for other degenerative diseases.
Encelto’s approval reflects increasing acceptance of advanced therapeutic modalities by regulatory authorities like the FDA. This sets new benchmarks for how next-generation therapies can leverage clinical data and innovation to secure market entry.
What This Means for Patients
For the MacTel community, Encelto represents more than just a treatment—it offers hope. Patients who once faced a clear trajectory of declining central vision can now benefit from a therapy that slows the disease’s progression and preserves their sight for longer.
Dr. Thomas Aaberg Jr., Neurotech’s Chief Medical Officer, captured this sentiment best, stating, “This is a historic moment for the MacTel community. We look forward to a future where vision loss from MacTel may be slowed.”
Looking Ahead
Neurotech’s FDA approval of Encelto serves as a reminder that innovation can thrive in even the smallest pockets of need. For biopharma professionals, this success highlights the importance of investing in rare disease research, leveraging advanced technologies, and navigating regulatory pathways effectively.
As Encelto enters the U.S. market, the next few years will tell a broader story about the potential for cell therapy to reshape how we approach chronic eye diseases—and healthcare as a whole.
For those involved in market access, life sciences innovation, or patient advocacy, Neurotech’s achievement exemplifies the transformative power of persistence and technology to enhance lives. Staying informed on breakthrough cases like Encelto is vital for organizations looking to integrate cutting-edge cell or gene therapy solutions into their strategies.
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