The European Medicines Agency has issued orphan designation to emixustat for the treatment of Stargardt disease, Acucela announced in a press release.
Emixustat hydrochloride modulates the visual cycle by inhibiting RPE65 protein, a critical enzyme of the pathway, the release said.
The FDA granted orphan drug designation to emixustat in January 2017 for the treatment of Stargardt disease. The current designation from the EMA does not cover other possible indications for emixustat.
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