Acucela treatment receives FDA orphan drug designation

The U.S. Food and Drug Administration granted orphan drug designation to Acucela's leading drug candidate emixustat hydrochloride, which was created to treat Stargardt disease.
The Orphan Drug Act allows the FDA to give special status to certain drugs intended to fight rare diseases and disorders – those that affect fewer than 200,000 people in the U.S.
“We are very pleased to receive FDA’s orphan drug designation for emixustat to treat Stargardt disease,” Acucela Chairman, President and CEO Dr. Ryo Kubota said in a statement. “We are actively advancing the development of emixustat to address this unmet medical need.”
Stargardt disease, a genetically inherited disease that causes vision loss in children, affects roughly 1 in 10,000 individuals around the globe. Symptons usually appear in adolescence, but sometimes can occur later in life. Estimates show that the disease affects less than 40,000 patients in the U.S.
Acucela, a subsidiary of Kubota Pharmaceuticals Holdings Co. Ltd., focuses on developing innovative drugs to preserve and restore vision for millions of people worldwide.