Acucela Inc. (“Acucela”), a clinical-stage ophthalmology company and wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Tokyo 4596), announced today that the European Medicines Agency (EMA) granted orphan designation to Acucela’s leading drug candidate emixustat hydrochloride (“emixustat”) for the treatment of Stargardt disease. The orphan designation does not apply to other indications for which emixustat is being developed.
To receive an orphan designation from the EMA, the medicine has to be developed for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition that affects no more than five in 10,000 within the European Union. Upon receiving the orphan designation, the sponsor will qualify for multiple development incentives which include ten years of market exclusivity after product approval, fee reductions of regulatory activities, access to the centralized marketing authorization procedure, and protocol assistance that provides scientific advice for the orphan designated medicines.
In January 2017, Acucela also received orphan drug designation for emixustat for the treatment of Stargardt disease by the FDA (U.S. Food and Drug Administration). According to the FDA’s Center for Drug Evaluation and Research’s annual report, the number of approved orphan drugs has been increasing year by year, and 58% of the drugs approved by the FDA in 2018 were orphan drugs. Many of them are expected to have peak sales of over US$1 billion.
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