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.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER