Pfiz­er’s block­buster safe­ty is­sue jumps the At­lantic as EMA launch­es its own re­view of Xel­janz

Pfiz­er’s Xel­janz woes have spread to Eu­rope.

Af­ter the FDA an­nounced ear­li­er this week that it is in­ves­ti­gat­ing the in­creased risk of blood clots and death linked to their block­buster JAK in­hibitor seen in a post-mar­ket­ing study, the EMA says they’ll now make their own as­sess­ment on the safe­ty of the drug.

“EMA is as­sess­ing the re­sults of the study and will con­sid­er what reg­u­la­to­ry ac­tion is need­ed,” the agency said in a state­ment sent to End­points News. The EMA added that pa­tients should con­sult a doc­tor if they have any ques­tions.

Eu­ro­pean reg­u­la­tors had proved much tougher than the FDA on Xel­janz, wait­ing un­til 2017 to ap­prove the drug for rheuma­toid arthri­tis af­ter de­lay­ing the roll­out for more safe­ty da­ta — 6 years af­ter the FDA OK.

The drug en­tered the mar­ket with a black box warn­ing of a high­er risk of in­fec­tions, but the phar­ma gi­ant set off alarm bells when re­searchers said they were tak­ing peo­ple off the 10 mg dose used in their as­sess­ment of the drug’s safe­ty among pa­tients with an added car­dio risk af­ter the tri­al mon­i­tors con­clud­ed that the drug was as­so­ci­at­ed with high­er rates of blood clots and death.

Both the FDA and EMA note that the rec­om­mend­ed dose for Xel­janz in RA is 5 mg twice a day, but the drug is ap­proved at 10 mg for ul­cer­a­tive col­i­tis. Eli Lil­ly al­so faced its own chal­lenges af­ter the FDA ini­tial­ly re­ject­ed its JAK in­hibitor baric­i­tinib, fi­nal­ly ap­prov­ing it as Olu­mi­ant last year at a low­er dose than it had sought.

The new safe­ty alert comes at a bad time for Pfiz­er, which is de­pend­ing on ris­ing sales of its block­buster drug at a time when US law­mak­ers have been ham­mer­ing hard against an­nu­al price hikes. Oth­er JAKs are in the late-stage pipeline with their own claims on safe­ty and ef­fi­ca­cy that will now face fur­ther scruti­ny.

On the oth­er hand, any new RA ther­a­py en­ter­ing the field with a bet­ter safe­ty pro­file will be look­ing at a se­ri­ous­ly dis­rupt­ed mar­ket.

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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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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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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.

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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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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House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

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