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FDA approves first oral treatment for Fabry disease

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The FDA has approved the first oral treatment of Fabry disease for use in adults with a genetic mutation deemed to be “amenable” to the therapy.

The approval was granted to Amicus Therapeutics U.S. for Galafold (migalastat) after efficacy was demonstrated in a 6-month placebo-controlled clinical trial in 45 adults, according to a news release from the FDA. The approval came via the FDA’s Accelerated Approval pathway, which requires further study to verify and describe the clinical benefits of the drug.

The company had been given fast track designation for the drug last September.

“Thus far, treatment of Fabry disease has involved replacing the missing enzyme that causes the particular type of fat buildup in this disease. Galafold differs from enzyme replacement in that it increases the activity of the body’s deficient enzyme,” said Julie Beitz, MD, director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research.

Reference:

www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm616598.htm