EU reg­u­la­to­ry com­mit­tee rec­om­mends 8 new drugs for ap­proval, in­clud­ing Bris­tol My­er­s' BC­MA CAR-T

At its June meet­ing, the Eu­ro­pean Med­i­cine Agency’s Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use rec­om­mend­ed eight new drugs for ap­proval, bring­ing this year’s count to 50 pos­i­tive opin­ions.

While the Eu­ro­pean Com­mis­sion doesn’t need to fol­low the CHMP’s rec­om­men­da­tions, it typ­i­cal­ly does. Here are the drugs that got the com­mit­tee’s green light this month:

Abec­ma 

A few months af­ter be­com­ing the first mar­ket­ed BC­MA CAR-T ther­a­py, blue­bird and Bris­tol My­ers Squibb’s Abec­ma (al­so known as ide-cel) has snagged a CHMP rec­om­men­da­tion for con­di­tion­al mar­ket­ing ap­proval in re­lapsed or re­frac­to­ry mul­ti­ple myelo­ma. The com­mit­tee rec­om­mends the drug as a fourth-line ther­a­py, af­ter pa­tients have tried an im­munomod­u­la­to­ry agent, a pro­tea­some in­hibitor and an an­ti-CD38 an­ti­body.

Abec­ma’s US ap­proval came about a year af­ter BMS and blue­bird got slammed with a sur­prise refuse-to-file let­ter. Reg­u­la­tors based their de­ci­sion on da­ta from the piv­otal Phase II Kar­M­Ma tri­al, which showed that among 100 pa­tients tak­ing ide-cel, 72% saw their tu­mors shrink and 28% achieved a strin­gent com­plete re­sponse. An es­ti­mat­ed 65% of those had re­mis­sion last­ing at least 12 months, the com­pa­nies said.

“As the first CAR T cell ther­a­py for re­lapsed and re­frac­to­ry mul­ti­ple myelo­ma to re­ceive a pos­i­tive CHMP opin­ion, Abec­ma rep­re­sents a po­ten­tial new treat­ment ap­proach for pa­tients in Eu­rope bat­tling this in­cur­able blood can­cer,” said Noah Berkowitz, BMS’ se­nior VP of cell ther­a­py de­vel­op­ment, in a state­ment.

The part­ners said back in March that the drug would have a US list price of $419,500.

How­ev­er, ri­vals like J&J and Leg­end are catch­ing up. Cil­ta-cel was grant­ed pri­or­i­ty re­view in May, the same month the part­ners re­leased Phase II da­ta show­ing a 95% over­all re­sponse rate, with a 75% strin­gent com­plete re­sponse rate. Those da­ta are part of the pack­age J&J is sub­mit­ting in rolling ap­pli­ca­tions for ap­provals in Eu­rope and the US.

BMS al­so came away with an­oth­er CHMP win, as the com­mit­tee rec­om­mend­ed ex­pand­ing Op­di­vo’s la­bel to in­clude the ad­ju­vant treat­ment of adults with esophageal or gas­troe­sophageal junc­tion can­cer fol­low­ing pri­or ther­a­py.

Vosori­tide

Bio­Marin’s vosori­tide is still await­ing a de­ci­sion from the FDA, but the CHMP has rec­om­mend­ed an OK for the treat­ment of achon­dropla­sia, a con­di­tion that caus­es dwarfism.

The CHMP’s rec­om­men­da­tion cov­ers pa­tients two years and old­er un­til their growth plates are closed, which oc­curs af­ter pu­ber­ty when chil­dren reach their fi­nal adult height.

Up­on get­ting vosori­tide across the FDA’s doorstep back in No­vem­ber, Bio­Marin said the drug spurred an­nu­al growth of 1.57 cm over place­bo and pa­tients, on av­er­age, gained 2.2 cm in height com­pared to what nat­ur­al his­to­ry sug­gest­ed they would in a Phase III study. Phase II da­ta sug­gest that the drug helped add up to 9.0 cm in cu­mu­la­tive ad­di­tion­al height over 4 and a half years. That was in pa­tients 5 to 18 years old. How­ev­er, the FDA made it clear that a pan­el of out­side ex­perts had rec­om­mend­ed two-year con­trolled tri­als across dif­fer­ent age groups.

Reg­u­la­tors had ini­tial­ly set an Au­gust 2021 PDU­FA date, then ex­tend­ed the de­ci­sion un­til No­vem­ber af­ter Bio­Marin sub­mit­ted two-year re­sults from a Phase III ex­ten­sion study.

Mon­ju­vi

Al­most a year af­ter the FDA grant­ed Mor­phoSys and In­cyte a quick okay on Mon­ju­vi, the CHMP has giv­en the CD19-tar­get­ing mon­o­clon­al an­ti­body a rec­om­men­da­tion in re­lapsed or re­frac­to­ry dif­fuse large B-cell lym­phoma (DL­B­CL).

The con­di­tion­al mar­ket­ing au­tho­riza­tion would like­ly come for taf­a­sita­m­ab in com­bo with lenalido­mide, fol­lowed by taf­a­sita­m­ab monother­a­py in pa­tients who aren’t el­i­gi­ble for au­tol­o­gous stem cell trans­plant, ac­cord­ing to the CHMP’s opin­ion.

The drug, which would be mar­ket­ed as Min­ju­vi in Eu­rope, raked in $22 mil­lion from its launch last Au­gust to the end of De­cem­ber. Re­sults re­leased last May showed a 59% re­sponse rate, a 39% com­plete re­sponse rate, and a me­di­an du­ra­tion of re­sponse of  34.6 months — near­ly 3 years.

“Pa­tients with re­lapsed or re­frac­to­ry DL­B­CL have lim­it­ed treat­ment op­tions and of­ten face a poor prog­no­sis. There is an ur­gent need for ef­fec­tive ther­a­pies and if ap­proved, this com­bi­na­tion could pro­vide pa­tients in Eu­rope with an im­por­tant new ther­a­peu­tic op­tion,” said Malte Pe­ters, Mor­phoSys’ chief R&D of­fi­cer.

In­cyte and Mor­phoSys share glob­al de­vel­op­ment rights to taf­a­sita­m­ab, and In­cyte has ex­clu­sive com­mer­cial­iza­tion rights to taf­a­sita­m­ab out­side the Unit­ed States.

Bimek­izum­ab

Bel­gium’s UCB isn’t ex­pect­ing an an­swer from the FDA on its bimek­izum­ab un­til Oc­to­ber. But the CHMP has giv­en it the thumbs-up for the treat­ment of mod­er­ate to se­vere plaque pso­ri­a­sis.

Over the last cou­ple of years, UCB has claimed Phase III wins against J&J’s block­buster Ste­lara, Ab­b­Vie’s Hu­mi­ra, and No­var­tis’ Cosen­tyx.

Cosen­tyx blocks in­ter­leukin-17F, one of the IL-17 cy­tokines in­volved in pso­ri­a­sis. The idea be­hind bimek­izum­ab was that by block­ing both in­ter­leukin 17s in­volved in pso­ri­a­sis — IL-17F and IL-17A — you could have a stronger ef­fect.

UCB says 58.6% of pa­tients who took bimek­izum­ab were com­plete­ly cleared of skin le­sions af­ter 16 weeks, com­pared to 20.9% of pa­tients on Ste­lara. It al­so out­per­formed Ste­lara at how many pa­tients were clear af­ter one year and at less­er bench­marks for plaque clear­ance, with more than 8 out of 10 pa­tients show­ing 90% im­prove­ment, com­pared to rough­ly half on Ste­lara, the com­pa­ny said.

When pit­ted against a place­bo, bimek­izum­ab helped 68% of pa­tients achieve com­plete­ly clear skin, and more than 90% see a 90% im­prove­ment, ac­cord­ing to Phase III da­ta re­leased in 2019. For place­bo that num­ber was 1.2%.

Back in April, the FDA set a PDU­FA date for Oct. 15.

By­ooviz 

Just as Genen­tech’s long-run­ning block­buster Lu­cen­tis is set to lose patent pro­tec­tion in Eu­rope, the CHMP has rec­om­mend­ed an OK for Sam­sung Bioepis and Bio­gen’s biosim­i­lar, dubbed By­ooviz.

The com­mit­tee is rec­om­mend­ing the drug for use in a slate of vi­su­al im­pair­ments in­clud­ing: wet age-re­lat­ed mac­u­lar de­gen­er­a­tion, vi­su­al im­pair­ment due to di­a­bet­ic mac­u­lar ede­ma, pro­lif­er­a­tive di­a­bet­ic retinopa­thy, vi­su­al im­pair­ment due to mac­u­lar ede­ma sec­ondary to reti­nal vein oc­clu­sion (branch RVO or cen­tral RVO), and vi­su­al im­pair­ment due to choroidal neo­vas­cu­lar­i­sa­tion.

Lu­cen­tis was first ap­proved for wet AMD in the US back in 2006. In 2017, it be­came the first FDA-ap­proved treat­ment for all forms of di­a­bet­ic retinopa­thy, the lead­ing cause of blind­ness in work­ing-age adults, back in 2017. How­ev­er, the mar­ket’s now crowd­ed with long-act­ing AMD drugs, in­clud­ing Re­gen­eron’s Eylea and No­var­tis’s Beovu.

“Biosim­i­lars could help broad­en ac­cess and of­fer sig­nif­i­cant health­care sav­ings through the treat­ment of these com­plex and of­ten de­bil­i­tat­ing oph­thalmic dis­eases,” said Ian Hen­shaw, Glob­al Head of Biosim­i­lars at Bio­gen.

Rox­adu­s­tat

Eu­rope could soon have its first oral in­hibitor of hy­pox­ia-in­ducible fac­tor (HIF) pro­lyl hy­drox­y­lase (PH), ac­cord­ing to Astel­las and Fi­bro­Gen.

The CHMP gave rox­adu­s­tat — which would be mar­ket­ed as Evren­zo — a pos­i­tive opin­ion for the treat­ment of ane­mia in pa­tients with chron­ic kid­ney dis­ease. The drug is al­ready ap­proved in this in­di­ca­tion in Japan, Chi­na and Chile, and is un­der re­view in the US.

The drug ran in­to trou­ble back in April, when Fi­bro­Gen back­tracked on some of its key safe­ty da­ta. Ac­cord­ing to CEO En­rique Con­ter­no, Fi­bro­Gen ex­ecs be­came “aware” that their analy­sis in­clud­ed post hoc changes to strat­i­fi­ca­tion fac­tors used to as­sess the haz­ard ra­tio of their drug — es­sen­tial­ly ma­nip­u­lat­ing the da­ta to en­hance how much the ther­a­py re­duced key risks for pa­tients and shar­ing that false pro­file with in­vestors and reg­u­la­tors. Us­ing pre-spec­i­fied strat­i­fi­ca­tion fac­tors raised the risk on key safe­ty end­points, which re­volved pri­mar­i­ly around MACE, a com­pos­ite end­point of all-cause mor­tal­i­ty, stroke, and my­ocar­dial in­farc­tion.

Shares plunged on the news, evis­cer­at­ing about a bil­lion dol­lars of Fi­bro­Gen’s mar­ket cap.

Two more gener­ics

The CHMP has al­so rec­om­mend­ed mar­ket­ing au­tho­riza­tion for two gener­ics: Abi­raterone My­lan (al­so known as abi­raterone ac­etate, a gener­ic to Janssen’s Zyti­ga) for metasta­t­ic prostate can­cer and Fin­golimod My­lan (fin­golimod, a gener­ic to No­var­tis’ Gilenya) for re­laps­ing-re­mit­ting mul­ti­ple scle­ro­sis with high dis­ease ac­tiv­i­ty.

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Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

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