In a stun­ning about face, the FDA is now wel­com­ing the new drug ap­pli­ca­tion from Alk­er­mes it re­ject­ed 2 weeks ago

Some­body at the FDA just got a rude wake up call.

Just two weeks af­ter the FDA in­vit­ed Wall Street to pum­mel Alk­er­mes $ALKS by re­fus­ing to even ac­cept its ap­pli­ca­tions for the de­pres­sion drug ALKS-5461, the biotech says that the agency has sud­den­ly de­cid­ed to sim­ply change its mind.

Ac­cord­ing to Alk­er­mes, the stun­ning about face came af­ter the com­pa­ny “clar­i­fied cer­tain as­pects of the NDA sub­mis­sion. No ad­di­tion­al da­ta or analy­ses were sub­mit­ted by Alk­er­mes to FDA.”

Alk­er­mes shares im­me­di­ate­ly shot up 20%, re­gain­ing $1.3 bil­lion of its mar­ket cap. But it couldn’t hold on to that, though, drop­ping to an 8% gain mid-morn­ing. Its PDU­FA date was set for Jan­u­ary 31, 2019.

Alk­er­mes said in ear­ly April that the FDA was de­mand­ing that the com­pa­ny con­duct ad­di­tion­al tri­als of 5461, a move that ham­mered the com­pa­ny’s share price as an­a­lysts pon­dered the years of work and ex­pense in tak­ing an­oth­er risky shot at gain­ing pos­i­tive clin­i­cal da­ta.

The com­pa­ny was throw­ing the dice on its ap­pli­ca­tion when it first filed, hop­ing to per­suade the FDA that its one pos­i­tive piv­otal study along with ad­di­tion­al da­ta would out­weigh the two failed tri­als that tar­nished the drug. That would not have met the agency’s long es­tab­lished stan­dard for two pos­i­tive stud­ies, but as I’ve writ­ten ear­li­er the FDA has been ratch­et­ing down its hur­dles to get more drugs ap­proved.

The change up marks an­oth­er sign of the dra­mat­ic shift in the FDA’s stance on drug ap­pli­ca­tions and its stan­dards for re­views, rais­ing some ma­jor ques­tions for the in­dus­try as well as con­sumers. Since Scott Got­tlieb be­came com­mis­sion­er in 2017 af­ter he was nom­i­nat­ed for Pres­i­dent Don­ald Trump with a man­date to speed new drug ap­provals, the agency re­versed it­self on three key drug re­jec­tions for Eli Lil­ly, Ther­a­peu­tic­sMD and Am­i­cus, wav­ing off its ear­li­er de­mands for more work.

None of these re­ver­sals car­ry a guar­an­tee of an ap­proval, but they do re­flect changes in di­rec­tion that all drug de­vel­op­ers will pay close at­ten­tion to.

Bar­clays summed it up this way:

Giv­en the po­lit­i­cal na­ture of the cur­rent FDA ad­min­is­tra­tion, we could see a sce­nario where nor­mal-course re­view for ALKS-5461 felt more “ap­pro­pri­ate” than con­tro­ver­sy raised by an RTF, es­pe­cial­ly con­sid­er­ing the prod­uct’s pri­or Fast Track des­ig­na­tion and med­ical need for in­no­va­tion with­in the in­di­ca­tion. How­ev­er, we con­tin­ue to feel ALKS is long from out of the woods on ALKS-5461 and will need to con­tin­ue to en­gage with FDA dur­ing re­view with po­ten­tial for the on­go­ing Phase 3 to play a role in any po­ten­tial ap­proval or re­view de­ci­sion.

Who at the FDA over­ruled the de­ci­sion to re­ject Alk­er­mes’ drug ap­pli­ca­tion? And why?

The agency, bound by strict rules of si­lence when it comes to com­ment­ing on com­pa­nies and drugs out­side a care­ful­ly reg­u­lat­ed set of pub­lic dis­clo­sures, de­clined com­ment — as they al­ways do.

Craig Hop­kin­son

Craig Hop­kin­son, chief med­ical of­fi­cer and se­nior vice pres­i­dent of med­i­cines de­vel­op­ment and med­ical af­fairs at Alk­er­mes had this to say:

“We will con­tin­ue to en­gage with the FDA through­out the re­view process, as we work to bring this im­por­tant med­i­cine to pa­tients.”

Im­age: Alk­er­mes CEO Richard Pops at an End­points event, Jan­u­ary 2017. END­POINTS NEWS

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