Emixustat hydrochloride is primarily used in the treatment of certain eye disorders, particularly those related to retinal diseases. It functions as a visual cycle modulator, targeting the biochemical processes involved in vision. This compound is specifically investigated for its potential to slow the progression of geographic atrophy, an advanced form of age-related macular degeneration (AMD). By inhibiting the enzyme RPE65, it reduces the accumulation of toxic byproducts in the retina, thereby helping to preserve vision in affected individuals. Its application is significant in ophthalmology, offering a therapeutic approach to manage conditions that currently have limited treatment options.