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:


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.


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.


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.


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.


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.


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.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,400+ biopharma pros reading Endpoints daily — and it's free.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,400+ biopharma pros reading Endpoints daily — and it's free.

Vlad Coric, Biohaven CEO

Vlad Coric charts course for new Bio­haven with neu­ro­science push and Big Phar­ma vets on board

What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the new Biohaven post-Pfizer takeover.

Pfizer officially scooped up Biohaven’s CGRP assets on Monday, including blockbuster migraine drug Nurtec and the investigational zavegepant, for $11.6 billion. As a result, Coric spun the broader pipeline into an independent company on Tuesday — with the same R&D team behind Nurtec but about 1,000 fewer staffers and a renewed focus on neuroscience and rare disease.

In AstraZeneca's latest campaign, wild eosinophils called Phils personify the acting up often seen in uncontrolled asthma

As­traZeneca de­buts an­noy­ing pur­ple ‘Phil’ crea­tures, per­son­i­fied asth­ma eosinophils ‘be­hav­ing bad­ly’

There are some odd-looking purple creatures lurking around the halls of AstraZenca lately. The “Phil” character cutouts are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of AZ’s newest awareness effort to help people understand eosinophilic asthma.

The “Asthma Behaving Badly” characters aren’t only on the walls at AZ to show the new campaign to employees, however. The “Phils” are also showing up online on the campaign website, and in digital and social ads and posts on Facebook and Instagram.

Big Phar­ma heavy­weights seek tweaks to FDA's clin­i­cal out­come as­sess­ment guid­ance

Pfizer, GSK, Janssen, Regeneron, Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience.

The draft is the third in a series of four patient-focused drug development guidance documents that the FDA had to create as part of the 21st Century Cures Act, and they describe how stakeholders (patients, caregivers, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information for medical product development and regulatory decision-making.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,400+ biopharma pros reading Endpoints daily — and it's free.

Robert Califf, FDA commissioner (via AP Images)

User fees in ac­tion: FDA un­veils new short­ened sup­ple­ment re­view, rare dis­ease pi­lots

Thanks to PDUFA VII, signed into law last Friday by President Joe Biden, the FDA this week unveiled two new industry-friendly pilot programs to advance new rare disease endpoints via additional meetings, and to shorten FDA review times for supplemental apps aimed at unmet medical needs.

The agency this week released eagerly-awaited details behind the shortened pilot, known as the Split Real Time Application Review or STAR pilot program, which will speed up certain FDA reviews of efficacy supplements across all therapeutic areas (thanks to earlier submissions of data), but only for those that propose addressing an unmet medical need.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,400+ biopharma pros reading Endpoints daily — and it's free.

Mar­ket­ingRx roundup: No­var­tis re­cruits NFL coach for Leqvio cam­paign; Pfiz­er pro­motes ‘Sci­ence’ merch on so­cial me­dia

Novartis is turning to a winning coach to talk about Leqvio and the struggles of high cholesterol — including his own. Bruce Arians, the retired NFL head coach of the Arizona Cardinals and Super Bowl-winning Tampa Bay Buccaneers, is partnering with the pharma for its “Coaching Cholesterol” digital, social and public relations effort.

In the campaign, Arians talks about the potential for “great comebacks” in football and heart health. Once nicknamed a “quarterback whisperer,” he is now retired from fulltime coaching (although still a front-office consultant for Tampa Bay), and did a round of media interviews for Novartis, including one with People and Forbes.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,400+ biopharma pros reading Endpoints daily — and it's free.

Amy West, Novo Nordisk head of US digital innovation and transformation (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: No­vo Nordisk dig­i­tal in­no­va­tion chief Amy West dis­cuss­es phar­ma pain points and a health­care 'easy but­ton’

Amy West joined Novo Nordisk more than a decade ago to oversee marketing strategies and campaigns for its US diabetes portfolio. However, her career path shifted into digital, and she hasn’t looked back. West went from leading Novo’s first digital health strategy in the US to now heading up digital innovation and transformation.

She’s currently leading the charge at Novo Nordisk to not only go beyond the pill with digital marketing and health tech, but also test, pilot and develop groundbreaking new strategies needed in today’s consumerized healthcare world.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.