Last week, the company announced its potential Fabry disease treatment drug migalastat met its primary endpoints in a late-stage study. The results showed Fabry patients who take drugs via injection for treatment could switch to the pill every other day and maintain kidney function necessary to keep Fabry disease in check. Fabry is a rare genetic disease that causes fat to build up in the body and causes pain, loss of hearing and cloudy eyes. It eventually leads to kidney failure, stroke and death.
Amicus now plans to submit migalastat to the European Medicines Agency early next year for regulatory approval based on the results. The company plans to schedule a meeting with the Food and Drug Administration to determine if the results, coupled with previous Phase III study data, are enough to move forward with a submission for U.S. regulatory approval.
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