Geographic atrophy (GA) is a severe, progressive eye condition and an advanced form of dry age-related macular degeneration (AMD) that causes central vision loss. Historically, there were no approved treatments for GA. However, the recent availability of pegcetacoplan (Syfovre) marks a significant development, as it is the first targeted therapy approved to address the progression of geographic atrophy.
Understanding Geographic Atrophy
Geographic atrophy is a late stage of dry age-related macular degeneration, characterized by the gradual, irreversible loss of retinal tissue. It specifically affects the macula, the central part of the retina responsible for sharp, detailed vision. This damage involves the degeneration of photoreceptor cells, retinal pigment epithelial (RPE) cells, and the underlying choriocapillaris.
GA progression often begins with blurry or distorted vision, leading to a central blind spot as the disease advances. This makes activities like reading or recognizing faces challenging. The damaged areas, or lesions, expand over time, resembling a map, which gives the condition its “geographic” name. While peripheral vision remains unaffected, central vision loss can severely impair independence.
Research indicates an overactive complement system, part of the body’s immune response, contributes to GA progression. This dysregulated system can cause chronic inflammation and cellular damage in the retina, often triggered by fatty deposits called drusen and other cellular debris.
The Role of Pegcetacoplan
Pegcetacoplan (Syfovre) is a medication designed to target and regulate the complement system in geographic atrophy. It functions as a C3 inhibitor, binding to and preventing the cleavage of the C3 protein, a central component of the complement cascade. By blocking this protein, pegcetacoplan disrupts the excessive immune activity contributing to retinal cell damage in GA.
This targeted inhibition helps slow the growth of atrophic lesions in the retina. Clinical studies have demonstrated that pegcetacoplan reduces the rate of GA lesion enlargement over a two-year period, with effectiveness increasing over time. This mechanism helps preserve vision by slowing disease progression.
Pegcetacoplan is the first treatment approved by the U.S. Food and Drug Administration (FDA) specifically for geographic atrophy secondary to age-related macular degeneration. This approval, granted in February 2023, was a significant advancement for individuals with this previously untreatable condition, offering a new therapeutic option to slow GA progression.
Administering Pegcetacoplan and Patient Experience
Pegcetacoplan is administered directly into the eye through an intravitreal injection. These injections are performed by a retina specialist in a clinical setting. The frequency of treatment can vary, with injections given either monthly or every other month, depending on the individual patient’s needs and the doctor’s assessment.
Patients may experience some temporary discomfort during and after the injection, such as mild eye pain or irritation. Other common effects can include small specks or floaters in vision, or a temporary increase in eye pressure. Bleeding in the white part of the eye, appearing as red spots, is also a common observation.
There are potential side effects that warrant close monitoring. These include the development of new or worsening wet AMD, eye inflammation, or, in rare instances, more severe complications like retinal detachment or infection. Patients are advised to report any sudden changes in vision, pain, or redness to their retina specialist promptly.
Who is a Candidate for Pegcetacoplan?
Pegcetacoplan is indicated for individuals diagnosed with geographic atrophy. The decision to initiate treatment is made by an ophthalmologist or retina specialist following a comprehensive eye examination and evaluation of the patient’s specific condition. This assessment typically includes analyzing the size and location of the GA lesions.
While the treatment aims to slow lesion growth, it does not reverse existing vision loss or improve visual acuity. Therefore, candidacy often involves a discussion of the potential benefits in slowing further progression versus the risks associated with the injections. Patients with lesions that are not yet affecting the fovea, the very center of the macula, may experience greater benefit from the treatment as it aims to preserve existing central vision.
A personalized discussion with a healthcare provider is important to determine if pegcetacoplan is a suitable option. The specialist will consider the patient’s overall eye health, any other co-existing eye conditions, and individual risk factors. The goal is to make an informed decision based on the specific characteristics of the geographic atrophy and the patient’s general health profile.