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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