SEATTLE--(BUSINESS WIRE)--Acucela Inc. (“Acucela”), a clinical-stage ophthalmology company and wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Tokyo 4596) that specializes in identifying and developing novel therapeutics to treat and slow the progression of sight-threatening ophthalmic diseases, announced today that the FDA (U.S. Food and Drug Administration) granted orphan drug designation to Acucela’s leading drug candidate emixustat hydrochloride (“emixustat”) for the treatment of Stargardt disease. The orphan drug designation does not apply to other indications emixustat is being developed.
The Orphan Drug Act (ODA) provides for granting special status to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the U.S.
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 worldwide1. The most common form of the disease is caused by a genetic mutation of the ABCA4 gene leading to the accumulation of toxic vitamin A byproducts (primarily A2E) in the retina, which results in the gradual deterioration of photoreceptors and vision. Symptoms of Stargardt disease typically appear during childhood or adolescence, but in some cases difficulty with eyesight and vision loss may not be identified until later in life.
Stargardt disease affects less than 40,000 patients in the U.S. where it is recognized as an orphan disease, subject to the Orphan Drug Act. Currently, there are no known therapies that exist to slow the advance of the disease, and it is recognized as a serious unmet medical need by the United States Foundation of Fighting Blindness and the National Eye Institute.
The visual cycle is the process by which vitamin A is recycled in the eye; vitamin A is crucial to the visual process. Emixustat modulates the visual cycle by inhibiting a critical enzyme of this pathway, retinal pigment epithelium protein 65 (RPE65). Slowing the visual cycle reduces the availability of vitamin A derivatives (11-cis- and all-trans-retinal) to form precursors of A2E and related compounds. 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. Emixustat when delivered orally was found to be generally well tolerated in human clinical studies with delayed dark adaptation being the most common ocular adverse event. Acucela is planning to explore emixustat’s potential to stop or slow the progression of vision loss in patients diagnosed with Stargardt disease in future clinical studies.
Dr. Ryo Kubota, MD, PhD, and Chairman, President and CEO of Acucela, states, “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.”
1 Kaplan J, Gerber S, Larget-Piet D et al. A gene for Stargardt’s disease (fundus flavimaculatus) maps to the short arm of chromosome 1. Nature Genetics 1993;5(3):308-311.
About Acucela Inc.
Acucela Inc. is a wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Tokyo 4596) committed to translating innovation into a diverse portfolio of drugs and devices to preserve and restore vision for millions of people worldwide. Acucela’s pipeline includes drug candidates and therapeutics for the treatment of retinitis pigmentosa, proliferative diabetic retinopathy, intermediate AMD, Diabetic macular edema, Stargardt disease and cataracts. http://www.acucela.com; http://www.kubotaholdings.co.jp/en/
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