Targeting factor D of the alternative complement pathway reduces geographic atrophy progression secondary to age-related macular degeneration

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Science Translational Medicine  21 Jun 2017:
Vol. 9, Issue 395, eaaf1443
DOI: 10.1126/scitranslmed.aaf1443

Illuminating a new treatment for macular degeneration

Geographic atrophy secondary to age-related macular degeneration is a major cause of vision loss for which there is no treatment. Yaspan et al. now report the results of the MAHALO phase 2 randomized, controlled trial that evaluated lampalizumab in patients with geographic atrophy secondary to age-related macular degeneration. Lampalizumab is a specific inhibitor of complement factor D, a pivotal regulator of the alternative complement pathway. The MAHALO study met its primary efficacy endpoint with a 20% reduction in lesion area progression compared to sham control with monthly lampalizumab treatment. Moreover, lampalizumab showed an acceptable safety profile. A more substantial monthly treatment benefit of 44% reduction in geographic atrophy progression versus sham control was observed in a subgroup of patients who were complement factor I risk-allele carriers.

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