Originally published by 2 Minute Medicine® (view original article). Reused on AccessMedicine with permission.

1. Avacincaptad pegol significantly slowed geographic atrophy over 12 months.

2. Exudative macular neovascularization was comparable among treatment groups.

Evidence Rating Level: 1 (Excellent)

Study Rundown:

Geographic atrophy is a severe form of age-related macular degeneration that can lead to irreversible vision loss. Currently, limited treatments exist. Avacincaptad pegol is a complement C5 inhibitor that may play a role in slowing the progression of geographic atrophy, although little is currently known. This randomized controlled trial aimed to assess the safety and efficacy of avacincaptad pegol in patients with age-related macular degeneration. The primary outcome of this study was geographic atrophy lesion size, measured by fundus autofluorescence at baseline, 6 months, and 12 months. Key secondary outcomes included adverse events within this study population. According to study results, avacincaptad pegol 2 mg significantly reduced geographic atrophy growth over 12 months compared to placebo. A major strength of this study was the large sample size and longitudinal follow-up design.

In-depth [randomized-controlled trial]:

Between Jun 22, 2020, and Jul 23, 2021, 1422 patients were screened for eligibility across 205 retina clinics worldwide. Included were patients ≥ 50 years with non-central geographic atrophy and corrected visual acuity between 20/25 and 20/320. Altogether, 448 patients (225 in avacincaptad pegol 2 mg and 223 in placebo) were included in the final analysis. Avacincaptad pegol 2 mg significantly slowed geographic atrophy compared to placebo over 12 months (0.336 mm/year, standard error [SE] 0.032 vs. 0.392 mm/year, SE 0.033; difference 0.056 mm/year [14%], p=0.0064). Furthermore, the secondary outcome showed that avacincaptad pegol 2 mg was well-tolerated and did not lead to significant ocular safety concerns (49% in avacincaptad pegol group and 37% in placebo group had treatment-emergent adverse events). Overall, findings, from this study suggest that monthly avacincaptad pegol 2 mg treatment may slow disease progression and potentially alter the course of geographic atrophy in patients.

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