FDA orphan drug status to Acucela for Stargardt Disease

Release Date: 13-Jan-2017

Acucela Inc. specializes in identifying and developing novel therapeutics to treat and slow the progression of sight-threatening ophthalmic diseases, declared that US Food and Drug Administration has granted orphan drug designation to its to Acucela’s leading drug candidate emixustat hydrochloride (“emixustat”) for the treatment of Stargardt disease.

Stargardt disease, or fundus flavimaculatus, is a rare, genetically inherited disease that directly affects the retina of the eye, often resulting in the slow progression of vision loss in children. It may also be referred to as Stargardt macular dystrophy or juvenile macular degeneration and affects approximately 1 in 10,000 individuals worldwide.

The FDA's designation of Orphan Status is granted to promote the development of new therapies for rare diseases and disorders. Orphan Status can be applied to products proven safe and effective in treating conditions that affect a relatively small number of patients, and the designation may entitle up to seven years of US marketing exclusivity upon regulatory approval. Companies whose drugs have an orphan designation also receive certain tax credits and are exempted from paying prescription drug user fees normally required of companies submitting products for approval.

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