Amicus’ rare disease drug, once indefinitely delayed, wins FDA approval
After 12 years, Amicus Therapeutics has won Food and Drug Administration approval for its first therapy, a treatment for a rare and sometimes deadly disease.
After 12 years, millions of dollars, and a major reversal of fortune, Amicus Therapeutics won Food and Drug Administration approval on Friday for its first therapy, a treatment for a rare and sometimes deadly disease.
The drug, called Galafold, targets , an inherited disorder in which the lack of a key enzyme leads to buildups up fats that can cause
