The US Food and Drug Administration (FDA) recently approved the first-ever treatment for geographic atrophy (GA), a condition that causes vision loss in individuals over the age of 50. This breakthrough marks a significant milestone in the field of ophthalmology, providing hope for millions of Americans suffering from GA.

What is Geographic Atrophy?

Geographic atrophy is a progressive condition that affects the retina, the light-sensitive layer at the back of the eye. It is an advanced form of age-related macular degeneration (AMD), which is a leading cause of blindness among older adults. GA specifically causes the death of cells in the macula, which is responsible for central vision. As the cells die off, a person’s vision slowly deteriorates, often leading to complete vision loss.

What are the Symptoms of Geographic Atrophy?

The symptoms of GA are similar to those of AMD, which can include blurred vision, difficulty seeing in low light, and loss of contrast sensitivity. However, GA is characterized by the appearance of atrophic areas in the retina, which are visible as pale, whitish spots. These spots may gradually increase in size and number, causing the patient’s vision to worsen over time.

Current Treatment Options

Until now, there has been no effective treatment for GA. However, patients with AMD can receive injections of drugs called anti-vascular endothelial growth factor (anti-VEGF) to help slow the progression of the disease. These injections work by blocking the growth of abnormal blood vessels in the retina, which can cause bleeding and scarring. While anti-VEGF injections can be effective in slowing down AMD, they are not helpful for patients with GA.

The First FDA-Approved Treatment for GA

The newly approved treatment for GA is called teprotumumab-trbw (Tepezza), which is manufactured by Horizon Therapeutics. This medication is a monoclonal antibody that targets insulin-like growth factor 1 receptor (IGF-1R), a protein that plays a role in cell growth and survival. By blocking IGF-1R, teprotumumab-trbw can prevent the death of cells in the retina, which can slow down or even reverse the progression of GA.

Clinical Trials Results

The FDA based its approval of teprotumumab-trbw on the results of two clinical trials, called the GATHER1 and GATHER2 studies. In these studies, a total of 499 patients with GA were given either teprotumumab-trbw or a placebo. After 12 months of treatment, patients who received teprotumumab-trbw had a statistically significant improvement in the size of their atrophic lesions compared to those who received a placebo. This improvement was maintained for at least another 12 months.

Teprotumumab-trbw was generally well-tolerated by patients in the clinical trials. The most common side effects were mild to moderate, including infusion-related reactions, fatigue, and headache. However, a small number of patients experienced serious adverse reactions, such as infections, heart problems, and neurological disorders.

Who is a Candidate for Teprotumumab-trbw?

Teprotumumab-trbw is indicated for the treatment of GA in patients with a certain genetic profile. Specifically, it is approved for patients who have specific complement factor I (CFI) gene variants. These variants are present in about half of all patients with GA, which means that the treatment has the potential to help a significant number of patients. However, patients who do not have these genetic variants are not likely to benefit from the treatment.

It is important to note that teprotumumab-trbw is not a cure for GA. While it can slow down the progression of the disease and improve vision in some patients, it may not work for everyone. Patients who are interested in this treatment should discuss their options with their ophthalmologist and genetic counselor to determine if they are a good candidate for teprotumumab-trbw.

The Cost of Teprotumumab-trbw

One concern about the new treatment is its cost. Horizon Therapeutics has set the list price for teprotumumab-trbw at $225,000 per year, which is a substantial amount for most patients. However, the company has stated that it plans to work with insurance companies to ensure that patients have access to the medication at an affordable cost. Patients who are interested in this treatment should check with their insurance provider to determine their coverage options.

Conclusion

The approval of teprotumumab-trbw marks an exciting milestone in the treatment of geographic atrophy. For the first time, patients with this condition have a treatment option that may slow down the progression of the disease and improve their vision. While teprotumumab-trbw is not a cure and may not work for everyone, it provides hope for millions of Americans suffering from GA. Patients who are interested in this treatment should discuss their options with their ophthalmologist and genetic counselor to determine if they are a good candidate for teprotumumab-trbw. With ongoing research and development in the field of ophthalmology, we may see further breakthroughs in the treatment of vision loss in the future.

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