Achillion Pharmaceuticals



Age-related macular degeneration (AMD) is one of the leading causes of blindness in adults and is expected to affect approximately 196 million people worldwide by 2020. AMD is a complex, multifactorial disease which, as its name suggests, affects the central portion of the retina, called the macula. Dry AMD is the first clinical stage of the disease and typically progresses from an early, asymptomatic phase in which pigment irregularities in the retinal pigment epithelium (RPE) and small deposits of lipids and proteins can be observed on examination, to intermediate and later phases characterized by geographic atrophy (GA) and the formation of new blood vessels (neovascularization)1. At least 2-3 million older adults in the United States and major European markets have dry AMD today2. There are currently no treatments available for this stage of AMD.

AMD is known to be a complement-mediated disease and, in fact, has provided the first clinical validation of the potential of factor D as a therapeutic target in ophthalmology. Candidates from Achillion’s library of small-molecule factor D inhibitors are being evaluated for intraocular administration to treat dry AMD.

1- Advancing Therapeutic Development for Dry Age-Related Macular Degeneration (AMD). Workshop in Brief. Institute of Medicine. Washington (DC): National Academies Press (US); 2015 Jan 16.
2- ACHN Corporate Overview Presentation, July 2015. Slide 19.