Am­i­cus CEO lob­bied FDA to re­verse FDA’s re­jec­tion of mi­gala­s­tat, claim­ing a de­lay of up to 7 years

Hand­writ­ten Note from John Crow­ley to Scott Got­tlieb.

Five days af­ter Scott Got­tlieb was sworn in as head of the FDA last May, Am­i­cus $FOLD CEO John Crow­ley was per­son­al­ly lob­by­ing him to re­verse a sting­ing re­jec­tion of mi­gala­s­tat, his new drug for rare cas­es of Fab­ry dis­ease.

He overnight­ed a let­ter — ob­tained by the Project on Gov­ern­ment Over­sight — to Got­tlieb ex­plain­ing that the com­pa­ny had de­ter­mined that it would take an ex­tra­or­di­nary 5 to 7 years to com­plete a Phase III study of gas­troin­testi­nal symp­toms that the agency had de­ter­mined would be re­quired be­fore it could con­sid­er of­fer­ing a green light for mar­ket­ing. That would not be fea­si­ble in a rea­son­able amount of time, he said. And he re­viewed new da­ta that had been shared with CDER di­rec­tor Janet Wood­cock, the FDA’s Julie Beitz and oth­ers at her re­quest.

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