EMA de­vel­op­ing physi­cian guid­ance for po­ten­tial liv­er dam­age af­ter tak­ing Zol­gens­ma

The Eu­ro­pean Med­i­cines Agency is work­ing on new ad­vice for health­care providers treat­ing pa­tients with Zol­gens­ma, five months af­ter two child pa­tient deaths in Rus­sia and Kaza­khstan.

That in­cludes mon­i­tor­ing the liv­er func­tion of pa­tients treat­ed with the gene ther­a­py and quick­ly as­sess­ing cas­es of sus­pect­ed liv­er dam­age, Reuters first re­port­ed.

The EMA re­leased its own state­ment af­ter the Phar­ma­covig­i­lance Risk As­sess­ment Com­mit­tee meet­ing on Jan. 12:

Health­care pro­fes­sion­als should prompt­ly as­sess pa­tients with wors­en­ing liv­er func­tion tests and/or signs or symp­toms of acute liv­er ill­ness. If pa­tients do not re­spond ad­e­quate­ly to treat­ment with cor­ti­cos­teroids, treat­ing physi­cians should con­sult a pae­di­atric gas­troen­terol­o­gist or he­pa­tol­o­gist and con­sid­er ad­just­ment of the cor­ti­cos­teroid reg­i­men.

Back in Au­gust, No­var­tis con­firmed that two pa­tients had died from acute liv­er fail­ure af­ter treat­ment with Zol­gens­ma, No­var­tis’ gene ther­a­py de­signed as a one-time treat­ment for spinal mus­cu­lar at­ro­phy, a rare and fa­tal dis­ease. They died ap­prox­i­mate­ly five to six weeks post-Zol­gens­ma in­fu­sion, and about one to 10 days af­ter doc­tors be­gan to ta­per cor­ti­cos­teroids.

A No­var­tis spokesper­son told End­points News that dis­cus­sions with the EMA have been on­go­ing since No­var­tis first no­ti­fied the agency of these cas­es and that there is no longer an in­ves­ti­ga­tion in­to the two pa­tient deaths.

When asked about the in­ves­ti­ga­tion’s find­ings, the com­pa­ny re­spond­ed, “The two deaths were the re­sult of acute liv­er fail­ure fol­low­ing treat­ment with Zol­gens­ma. Liv­er tox­i­c­i­ty, in­clud­ing acute liv­er fail­ure, is a known risk as­so­ci­at­ed with Zol­gens­ma and the sub­ject of la­bel­ing in­clud­ing our boxed warn­ing in US.”

Fur­ther dis­cus­sion on guid­ance for physi­cians will go to sev­er­al more com­mit­tees with­in the EMA, in­clud­ing the Com­mit­tee for Ad­vanced Ther­a­pies and to the Hu­man Med­i­cines Com­mit­tee.

If the guid­ance is ap­proved, it will be sent to health­care providers by the mar­ket­ing au­tho­riza­tion hold­ers.

“We con­tin­u­al­ly as­sess changes to treat­ment or mon­i­tor­ing rec­om­men­da­tions for Zol­gens­ma. Pend­ing health au­thor­i­ty re­view and ap­proval, the Zol­gens­ma la­bel will be up­dat­ed to in­form pre­scribers that fa­tal acute liv­er fail­ure has been re­port­ed,” a No­var­tis spokesper­son told End­points via email. “Ad­di­tion­al­ly, com­mu­ni­ca­tions dis­trib­uted to pre­scribers stress the im­por­tance of en­hanced mon­i­tor­ing of liv­er func­tion dur­ing cor­ti­cos­teroid ta­per­ing pe­ri­od, and for at least three months af­ter Zol­gens­ma in­fu­sion, and/or at oth­er times as clin­i­cal­ly in­di­cat­ed.”

The drug won con­di­tion­al EU ap­proval in 2020 and is con­sid­ered one of the most ex­pen­sive drugs in the world, with a price tag of $2.1 mil­lion.

Illustration: Assistant Editor Kathy Wong for Endpoints News

How Pur­due's $272M ad­dic­tion pay­out fund­ed a new home for its dis­card­ed non-opi­oid re­search

Don Kyle spent more than 20 years working for Purdue Pharma, right through the US opioid epidemic that led to the company’s rise and eventual infamy. But contrary to Purdue’s focus on OxyContin, Kyle was researching non-opioid painkillers — that is, until the company shelved his research.

As the company’s legal troubles mounted, Kyle found an unlikely way to reboot the project. In 2019, he took his work to an Oklahoma State University center that’s slated to receive more than two-thirds of the state’s $272 million settlement with Purdue over claims that the drugmaker’s behavior ignited the epidemic of opioid use and abuse.

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Utpal Koppikar, new Verily CFO

Ex­clu­sive: Ver­i­ly wel­comes Atara Bio­ther­a­peu­tics vet­er­an as new CFO

Verily, Alphabet’s life sciences outfit, has plucked a new CFO from the ranks of Atara Biotherapeutics, the company announced on Wednesday.

Utpal Koppikar joins Verily after a nearly five-year stint as CFO and senior VP at Atara, though his résumé also boasts roles at Gilead and Amgen.

The news follows a major reshuffling at Verily, including several senior departures earlier this year and a round of layoffs.

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President Joe Biden at the State of the Union address with Vice President Kamala Harris and House Speaker Kevin McCarthy (Patrick Semansky/AP Images)

The drug pric­ing pres­i­dent: Biden warns of ve­to for any IRA re­peal at­tempts

President Joe Biden made clear in his “finish the job” State of the Union address last night that one of those jobs to be finished is insulin prices.

Biden’s push again to tackle insulin prices, after Republicans rebuffed the idea last summer and just after Biden won Medicare drug price negotiations/caps via the Inflation Reduction Act, shows how heavily he’s leaning into this work.

Singer Nick Jonas is back at work for Dexcom, this time for its new G7 glucose monitor.

Dex­com's spokescelebri­ty Nick Jonas re­turns to Su­per Bowl in new glu­cose mon­i­tor com­mer­cial

Dexcom is going back to the Super Bowl with its pop singer and patient spokesperson Nick Jonas. Jonas takes center stage as the lone figure in the 30-second commercial showcasing Dexcom’s next-generation G7 continuous glucose monitoring (CGM) device.

Jonas’ sleight-of-hand tricks populate the commercial — he pinches his empty fingers together and pops them open to reveal the small CGM — even as he ends the ad, saying, “It’s not magic. It just feels that way.” Jonas then disappears in a puff of smoke.

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Richard Francis, newly-appointed Teva CEO (Novartis via Facebook)

New Te­va CEO Richard Fran­cis repri­or­i­tizes to 'get back to growth'

Six weeks into his new role at the helm of Teva Pharmaceutical, Richard Francis said it’s time to “get back to growth,” starting with a good look at the company’s priorities.

The chief executive has kicked off a strategic review, he announced during Teva’s quarterly call, which will continue over the next several months and produce results sometime in the middle of 2023. That means some pipeline cuts may be in store, he told Endpoints News, while declining to offer much more detail.

Rupert Vessey, Bristol Myers Squibb head of research and early development

Up­dat­ed: R&D tur­bu­lence at Bris­tol My­ers now in­cludes the end of a $650M al­liance and the de­par­ture of a top re­search cham­pi­on

This morning biotech Dragonfly put out word that Bristol Myers Squibb has handed back all rights to its IL-12 clinical-stage drug after spending $650 million to advance it into the clinic.

The news arrives amid a turbulent R&D stage for the pharma giant, which late last week highlighted Rupert Vessey’s decision to depart this summer as head of early-stage R&D following a crucial three-year stretch after he jumped to Bristol Myers in the big Celgene buyout. During that time he struck a series of deals for Bristol Myers, and also shepherded a number of Celgene programs down the pipeline, playing a major role for a lineup of biotechs which depended on him to champion their drugs.

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Sanofi is renewing its #VaccinesForDreams campaign with more stories, such as Juan's in Argentina (Sanofi)

Sanofi re­news so­cial cam­paign to re­mind that vac­cines let peo­ple ‘Dream Big’

Sanofi is highlighting people’s dreams — both big and small — to make the point that vaccines make them possible.

The renewed “Dream Big” global social media campaign’s newest dreamer is Juan, a teacher in the Misiones rainforest in Argentina whose story is told through videos on Instagram and Sanofi’s website with the hashtag #VaccinesForDreams.

The campaign ties to Sanofi’s broader umbrella initiative “Vaccine Stories” to promote the value of vaccines and drive awareness of the need for improved vaccination coverage.

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Bill Anderson, incoming Bayer CEO (David Paul Morris/Bloomberg via Getty Images)

Bay­er taps Roche's Bill An­der­son to lead phar­ma gi­ant as CEO

We now know where Roche’s ex-pharma chief Bill Anderson is going.

German pharma giant Bayer announced Wednesday that Anderson will be taking on the role as CEO, less than six weeks after Anderson stepped down from his perch at Roche as head of the group’s pharmaceutical division.

Roche announced back in December that Anderson would depart on Dec. 31 to “pursue opportunities outside of Roche.” His replacement, Genentech vet and Roche’s current head of global product strategy, Teresa Graham, will start her role in March.

Iya Khalil, Merck VP and head of data, AI and genome sciences (Novartis)

Mer­ck-No­var­tis re­volv­ing door spins again as AI leader Iya Khalil switch­es phar­mas

As talk of AI this-and-that gobbles up headline after headline, one Big Pharma is losing its AI leader as she transitions to another drug giant: Iya Khalil will trade in her hat as Novartis’ go-to expert and leader in the space for Merck as VP and head of data, AI and genome sciences next week.

After nearly three years leading the artificial intelligence team at Novartis — as Big Pharma and biotechs alike latch onto the ripening AI-for-drug-discovery mode of operation — Khalil will switch employers to head up a similar post at Merck, where she’ll work out of Cambridge, MA beginning Feb. 13, the company tells Endpoints News.