Groundbreaking Drug Launches
Revakinagene taroretcel (Encelto)
Mechanism of Action: Ciliary neurotrophic factor replacements; Neurogenesis stimulants Drug class: Gene therapies Originator and Owner: Neurotech USA Indication: Retinal telangiectasis Approval date: 5 March 2025 Trials: NTMT-03-A and NTMT-03-B
Retinal telangiectasis
Retinal telangiectasia is a condition that affects the retina, the light-sensitive layer at the back of the eye. The retina captures images entering the eye and sends them to the brain for processing. This condition occurs when small blood vessels in the macula (a part of the retina) grow abnormally. These vessels may widen (dilate) and leak. The macula plays a crucial role in sharp vision, allowing you to perform tasks like reading fine print and threading a needle. When the blood vessels around the macula leak, it can lead to complications such as macular edema, where the retina becomes swollen or thickened. This can result in blurry vision and loss of central vision.
Symptoms • Blurry vision • Trouble reading • Loss of peripheral vision • Metamorphopsia, a condition where straight lines appear distorted or wavy
Prevalence
The prevalence of macular telangiectasia type 2 (MacTel 2) in the United States is 0.1% of the population.
Treatment
Treatment for retinal telangiectasia depends on the stage of the disease. There is no specific treatment for the early stages, so regular eye exams are essential to monitor for any progression. For more advanced cases, treatment options may include laser therapy, certain eye drops, and anti-VEGF (anti-vascular endothelial growth factor) injections. These therapies aim to reduce swelling in the macula.
Why is Revakinagene taroretcel poised to make an impact in 2025?
Currently, there are no treatments for the early stages of the condition, leaving eye doctors to manage complications and preserve as much vision as possible. The primary recommended care approach is interdisciplinary, focusing on lifestyle modifications such as managing diabetes or hypertension, avoiding smoking, using sunglasses, and following a healthy diet.
Revakinagene taroretcel offers a potential breakthrough as the first treatment specifically targeting MacTel Type 2, with the potential to slow its progression. The gene therapy utilizes Neurotech’s Encapsulated Cell Therapy platform to deliver CNTF, a neuroprotective protein, to slow retinal degeneration and improve visual outcomes in MacTel patients. There are no approved treatments for MacTel, despite its significant impact on quality of life.
In March 2025, the US FDA approved ENCELTO™ (revakinagene taroretcel-lwey) for the treatment of Macular Telangiectasia type 2 (MacTel 2). This drug is expected to launch in June 2025.
In February 2019, the US FDA had granted fast track designation for revakinagene taroretcel, for the treatment of patients with macular telangiectasia type 2.
Regulatory milestones
Registrational Trials
The regulatory applications to the US FDA were based on the efficacy of the NTMT-03-A and NTMT-03-B phase 3 trials.
NTMT-03-A:
A Phase 3 Multicenter, Randomized, Sham-Controlled Study to Determine the Safety and Efficacy of Revakinagene taroretcel (NT-501) in Macular Telangiectasia Type 2 (NCT03316300).
NTMT-03-B:
A Phase 3 Multicenter, Randomized, Sham-Controlled Study to Determine the Safety and Efficacy of Revakinagene taroretcel (NT-501) in Macular Telangiectasia Type 2 (NCT03319849).
“Data from these trials demonstrated significant reduction in ellipsoid zone (EZ) area loss in MacTel patients, with NTMT-03-A reducing loss by 56.4% and NTMT-03-B by 29.2% at 24 months, indicating slowed disease progression compared to sham treatment”