Thumbs Up. Thumbs Down: On rook­ie mis­takes, a class ac­tion suit and get­ting past fail­ure


End­points as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

Por­to­la makes a rook­ie mis­take, and gets ham­mered by a CRL

If any com­pa­ny looked to be sit­ting in the right place at the right time with a new drug ap­pli­ca­tion, it was Por­to­la. The FDA had al­ready giv­en the com­pa­ny a break­through des­ig­na­tion, of­fer­ing ready ac­cess that com­pa­ny ex­ecs say they took ad­van­tage of reg­u­lar­ly. But when it came down to the PDU­FA date, the biotech was forced to an­nounce that the agency had re­ject­ed the ap­pli­ca­tion. Why? In a call with an­a­lysts, Por­to­la’s crew sound­ed like they had fun­da­men­tal­ly mis­judged the FDA’s at­ti­tude on man­u­fac­tur­ing and da­ta is­sues. Points they thought could have been clar­i­fied af­ter an ap­proval couldn’t wait. And now it will spend some time an­swer­ing the FDA’s CRL rather than mar­ket­ing its an­ti-an­ti­co­ag­u­lant. This was a sim­ple rook­ie mis­take, but we’ve seen re­peat­ed re­jec­tions this year based on CMC is­sues. Oth­er de­vel­op­ers should take very care­ful note of it.

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