FDA rais­es ques­tions about Mallinck­rodt's HRS-1 drug be­fore ad­vi­so­ry hear­ing

Last Au­gust, UK drug­mak­er Mallinck­rodt pre­sent­ed pos­i­tive topline find­ings for its em­bat­tled ter­li­pressin, il­lus­trat­ing the can­di­date met its pri­ma­ry end­point in treat­ing a life-threat­en­ing form of liv­er dis­ease that caus­es kid­ney fail­ure.

But the FDA con­tin­ues to have ques­tions about ter­li­pressin’s safe­ty and ef­fi­ca­cy. Af­ter de­ter­min­ing from the out­set of the tri­al that the pri­ma­ry end­point would on­ly clas­si­fy as a sur­ro­gate end­point, the agency is ques­tion­ing whether or not pa­tients “trend­ed to­ward clin­i­cal im­prove­ments” while on ter­li­pressin. Reg­u­la­tors will con­vene in a hear­ing to­day and have re­leased a doc­u­ment out­lin­ing their think­ing ahead of the meet­ing.

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