Bris­tol My­ers Squibb earns long-await­ed FDA nod for liso-cel, join­ing short list of lym­phoma CAR-T win­ners

When Bris­tol My­ers Squibb bought out Cel­gene for $74 bil­lion back in late 2019, one of the pur­port­ed cen­ter­pieces in that deal was liso-cel, which hoped to join a small group of CAR-Ts for non-Hodgkin lym­phoma. Ex­pect­ing an ap­proval in 2020, Bris­tol has had its share of set­backs in get­ting liso-cel across the fin­ish line — but now it fi­nal­ly has the green light.

The FDA on Fri­day ap­proved Bris­tol’s Breyanzi (liso­cab­ta­gene mar­aleu­cel), a CAR-T to treat dif­fuse large B cell lym­phoma in pa­tients who have pre­vi­ous­ly re­ceived two pri­or rounds of sys­temic ther­a­py, the agency said in a re­lease.

The agency based its re­view on piv­otal Phase I da­ta show­ing 54% of pa­tients treat­ed with liso-cel achieve com­plete re­mis­sion. DL­B­CL makes up about 33% of all non-Hodgkin lym­phoma cas­es, the FDA said, of which there are around 77,000 new­ly di­ag­nosed each year.

The one-time ther­a­py will come with a $410,300 whole­sale price, Bris­tol said, say­ing it came up with that fig­ure af­ter con­sid­er­ing “many fac­tors in­clud­ing its med­ical and clin­i­cal val­ue, pa­tient val­ue and so­ci­etal val­ue.”

That’s a sig­nif­i­cant pre­mi­um over Gilead’s Yescar­ta, which launched with a $373,000. Kym­ri­ah al­so launched with a $373,000 WAC for B cell lym­phomas.

With the ap­proval will come a black-box warn­ing la­bel for both cy­tokine re­lease syn­drome, a po­ten­tial­ly fa­tal over­re­ac­tion of the im­mune sys­tem, as well as neu­ro­log­ic tox­i­c­i­ties. Those warn­ings are se­vere enough that the FDA has in­sti­tut­ed a Risk Eval­u­a­tion and Mit­i­ga­tion Strat­e­gy that will re­quire health­care fa­cil­i­ties and physi­cians ad­min­is­ter­ing the ther­a­py to be spe­cial­ly cer­ti­fied to iden­ti­fy and treat CRS and neu­ro tox­i­c­i­ties.

Those warn­ings and ad­min­is­tra­tion re­stric­tions are aren’t unique to liso-cel, how­ev­er. Yescar­ta and Kym­ri­ah, for in­stance, sport the same black box warn­ings, and the un­met clin­i­cal need will like­ly dri­ve up­take in what is an ex­treme­ly dif­fi­cult-to-treat pa­tient pop­u­la­tion.

Liso-cel is now the third CAR-T on the mar­ket for var­i­ous forms of non-Hodgkin lym­phoma, along­side Yescar­ta and Kym­ri­ah. Bris­tol aimed to have the ther­a­py ap­proved late last year, but de­lays from Covid-19 and an ug­ly in­spec­tion re­port at one of the com­pa­ny’s con­tract man­u­fac­tur­ing sites pushed the ap­proval in­to the new year.

Liso-cel was one of a group of pipeline can­di­dates tied to a $9 CVR from Bris­tol’s Cel­gene buy­out in 2019. With the ap­proval de­lay, in­vestors lost out on that pay­day when the year ex­pired — not a ma­jor is­sue for Bris­tol it­self but def­i­nite­ly a headache for the CVR traders.

In De­cem­ber, an FDA in­spec­tion at Lon­za Hous­ton’s plant found a raft of is­sues, in­clud­ing mis­la­bel­ing, prod­ucts for the US and EU drug mar­kets stored in the same bins, “poor­ly main­tained” freez­er units, and ex­pired batch­es of in­gre­di­ents that weren’t prop­er­ly dis­posed of, ac­cord­ing to a Form 483 let­ter pub­lished on­line late last month.

Lon­za, one of a group of con­trac­tors work­ing on liso-cel’s man­u­fac­tur­ing, said de­lays in the FDA’s in­spec­tion sched­ule made it dif­fi­cult to ad­just in time for an ap­proval with­in 2020. Bris­tol pre­vi­ous­ly said it re­spond­ed to the agency’s con­cerns with­in eight days of re­ceiv­ing its let­ter.

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Elizabeth Warren (Michael Brochstein/Sipa USA)(Sipa via AP Images)

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