FDA Approves Treatment for Rare Genetic Disorder

The FDA granted approval to Amicus’ Galafold (migalastat), the first oral medication for treating adult patients with Fabry disease, a rare and serious genetic disease that causes enzyme deficiency.

The drug is indicated for adult patients with a genetic mutation that is considered “amenable”, or responsive, to the oral drug based on laboratory data.

While treatment of the disease has so far involved replacing the missing enzyme that is responsible for the disease’s fat buildup, Galafold instead increases the activity of the deficient enzyme.