Acucela has announced that the Food and Drug Administration (FDA) has granted orphan drug designation to their candidate, emixustat (emixustat hydrochloride), for the treatment of Stargardt disease.

Stargardt disease, also referred to as Stargardt macular atrophy, is a rare, genetically-inherited disease that directly affects the retina and often results in the slow progression of vision loss in children. 

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Emixustat modulates the visual cycle by inhibiting a critical enzyme of this pathway, retinal pigment epithelium protein 65 (RPE65). In animal models of Stargardt disease and retinal degeneration, emixustat was found to stop and reverse the accumulation of A2E and to preserve the integrity of the retina. Clinical studies involving human patients with Stargardt disease are currently being planned.

“We are very pleased to receive FDA’s orphan drug designation for emixustat to treat Stargardt disease. We are actively advancing the development of emixustat to address this unmet medical need,” said Dr. Ryo Kubota, MD, PhD, and CEO of Acucela.

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