John Oyler, BeiGene CEO (Paul Yeung/Bloomberg via Getty Images)

FDA ap­proves BeiGene's Brukin­sa in rare blood can­cer amid com­pa­ny's US ex­pan­sion plans

BeiGene has been any­thing but shy about its US ex­pan­sion plans cen­tered around blood can­cer drug Brukin­sa. To see those plans through, BeiGene will have to go right at Ab­b­Vie and J&J’s Im­bru­vi­ca, and Brukin­sa now has one more ar­row in its quiver as part of that bat­tle.

The FDA on Wednes­day ap­proved BTK in­hibitor Brukin­sa to treat Walden­ström’s macroglob­u­line­mia, a rare form of lym­phoma, af­ter the drug aced a head-to-head show­down with Im­bru­vi­ca, the on­ly oth­er drug ap­proved for that dis­ease, BeiGene said in a re­lease.

The agency based its re­view on da­ta from the Phase III AS­PEN study, which pit­ted Brukin­sa against Im­bru­vi­ca on a pri­ma­ry end­point of a very good par­tial re­sponse (VG­PR) as judged by an in­de­pen­dent re­view com­mit­tee. BeiGene’s drug post­ed a VG­PR rate of 28% com­pared with 19% for Im­bru­vi­ca by one cri­te­ri­on, and an­oth­er mea­sure saw rates of 16% for Brukin­sa and 7% for Im­bru­vi­ca.

The drug’s la­bel de­ter­mined Brukin­sa’s par­tial re­sponse rate as 78%, com­pared with 78% for Im­bru­vi­ca, and a 12-month event-free du­ra­tion of re­sponse rate of 94%, as op­posed to 88% for Im­bru­vi­ca. The most com­mon side ef­fects for Brukin­sa’s drug were de­creased neu­trophil count, up­per res­pi­ra­to­ry tract in­fec­tion, de­creased platelet count, rash, he­m­or­rhage, mus­cu­loskele­tal pain, de­creased he­mo­glo­bin, bruis­ing, di­ar­rhea, pneu­mo­nia and cough.

This is now the sec­ond ap­proval for Brukin­sa in the US and comes as BeiGene looks to great­ly ex­pand its foot­print state­side. To do so, the com­pa­ny has tak­en the fight right at Im­bru­vi­ca, look­ing to steal mar­ket share away from the block­buster hema­tol­ogy drug from Ab­b­Vie and J&J to help fu­el its own ex­pan­sion.

Jane Huang

“With 11 reg­u­la­to­ry ap­provals in un­der two years, in­clud­ing two in the U.S., Brukin­sa is demon­strat­ing its grow­ing util­i­ty as a treat­ment op­tion for B-cell ma­lig­nan­cies and ex­pand­ing its foot­print to po­ten­tial­ly ben­e­fit more pa­tients world­wide,” BeiGene’s hema­tol­ogy CMO Jane Huang said in a state­ment. “We will con­tin­ue to eval­u­ate Brukin­sa in its broad glob­al clin­i­cal pro­gram and look for­ward to ad­di­tion­al clin­i­cal ev­i­dence to es­tab­lish its po­si­tion as a po­ten­tial­ly best-in-class med­i­cine.”

In ear­ly Au­gust, BeiGene an­nounced plans to es­tab­lish a sprawl­ing, 42-acre R&D and man­u­fac­tur­ing fa­cil­i­ty in Hopewell, NJ. The site ex­pects to hire hun­dreds in the com­ing years, BeiGene said at the time, and will serve as the US/Chi­nese drug­mak­er’s wav­ing flag for its grow­ing mar­ket am­bi­tions with Brukin­sa at the fore­front.

The drug in Ju­ly beat a com­bi­na­tion of chemother­a­py ben­damus­tine and Roche’s Rit­ux­an to ex­tend the lives and halt the pro­gres­sion of dis­ease in pa­tients with chron­ic lym­pho­cyt­ic leukemia or small lym­pho­cyt­ic leukemia in a Phase III study. That’s an­oth­er ma­jor Im­bru­vi­ca in­di­ca­tion, and if the com­pa­ny wins FDA ap­proval, it will add to its June 2020 ap­proval for the treat­ment of CLL and SLL in Chi­na. Brukin­sa al­so sports an FDA ap­proval in man­tle cell lym­phoma.

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; and more

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.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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Elcin Barker Ergun, Menarini Group CEO

Amid Roche and Sanofi's oral SERD set­backs, Menar­i­ni gets speedy re­view at FDA

Menarini and Radius Health are getting a speedy review at the FDA for their oral SERD breast cancer drug months after the field opened up with competitors failing and fleeing.

It was a one-two-three punch in March, April and May as Sanofi flunked its first big test for its oral selective estrogen receptor degrader (SERD), Roche also flamed out in a Phase II and G1 Therapeutics ended its program after scoping out the data and potential partners.

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