Am­i­cus scraps a top rare dis­ease drug af­ter it flunks a piv­otal test — and shares spike

Just two months af­ter Am­i­cus Ther­a­peu­tics $FOLD got a rare re­prieve from the FDA, clear­ing the path for a new mar­ket­ing ap­pli­ca­tion for its lead drug mi­gala­s­tat, the biotech has been ham­mered by a se­vere set­back on its num­ber two drug de­vel­op­ment ef­fort. The com­pa­ny re­port­ed this morn­ing that SD-101 failed a Phase III study, miss­ing the co-pri­ma­ry and key sec­ondary end­points in treat­ing epi­der­mol­y­sis bul­losa.

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