No­var­tis wins a key OK for Kym­ri­ah, but con­tin­u­ing man­u­fac­tur­ing woes hob­ble roll­out — ri­val Gilead CAR-T breaks in­to Eu­rope

No­var­tis CAR-T man­u­fac­tur­ing woes are con­tin­u­ing to put a crimp in its abil­i­ty to pro­vide ther­a­py to all po­ten­tial pa­tients.

Vas Narasimhan

The phar­ma gi­ant re­port­ed this morn­ing that the EC has stamped its ap­proval on mar­ket­ing Kym­ri­ah in Eu­rope for B-cell acute lym­phoblas­tic leukemia as well as treat­ment-re­sis­tant adults suf­fer­ing from dif­fuse large B-cell lym­phoma. But No­var­tis can on­ly make it avail­able to chil­dren with ALL now — adults will have to wait.

No­var­tis’s top ex­ecs have worked hard to smooth things over with an­a­lysts, but it’s been be­dev­iled by man­u­fac­tur­ing is­sues through­out the de­vel­op­ment stage and con­tin­ues to be af­fect­ed as it goes toe-to-toe with Gilead’s Kite, ap­proved for large B-cell lym­phoma. And Gilead is still com­ing on strong, not­ing this morn­ing that they re­ceived a Eu­ro­pean OK to mar­ket Yescar­ta for dif­fuse and pri­ma­ry me­di­asti­nal large B-cell lym­phoma.

A month ago No­var­tis CEO Vas Narasimhan not­ed dur­ing the Q2 call that:

I think it’s ear­ly days and we’ve al­ways said this is go­ing to be a five-year jour­ney with Kym­ri­ah to re­al­ly get us to be the glob­al­ly suc­cess­ful brand we want it to be.

On man­u­fac­tur­ing, we have seen some vari­abil­i­ty in our prod­uct spec­i­fi­ca­tions. This is some­thing we’re look­ing at now in DL­B­CL to make sure that we can con­tin­ue to ramp-up the de­mand. We feel con­fi­dent in the over­all longer-term out­look for Kym­ri­ah.

Liz Bar­rett

Liz Bar­rett, who runs No­var­tis on­col­o­gy, had this to say:

I think the most im­por­tant thing to let you know is that the vari­abil­i­ty that we’ve seen in the com­mer­cial spec­i­fi­ca­tions, which isn’t un­usu­al with a new ther­a­py as you launch in­to a new tar­get pa­tient pop­u­la­tion, is that we have still been able to de­liv­er fi­nal prod­uct to the ma­jor­i­ty of pa­tients.

As we’ve re­port­ed, though, the cell man­u­fac­tur­ing chief’s of­fice has been out­fit­ted with a re­volv­ing door at No­var­tis in re­cent years. Karen Walk­er left that post in ear­ly 2017, fol­lowed last spring by Spencer Fisk as he jumped ship to join the No­var­tis ex­ecs at Ru­bius. Those de­par­tures came on top of a ma­jor re­vamp for the cell and gene ther­a­py unit in 2016, which raised ques­tions at the time about No­var­tis’ abil­i­ty to keep up with Kite — though the phar­ma gi­ant wound up with the pi­o­neer­ing first OK. And No­var­tis’ is­sues with man­u­fac­tur­ing cell ther­a­pies ex­tend back to the sna­fus it ex­pe­ri­enced with late de­liv­ery to a small but sig­nif­i­cant group of pa­tients en­rolled in clin­i­cal tri­als.

At one stage in the de­vel­op­ment process, No­var­tis had a 6% fail­ure rate on the man­u­fac­tur­ing process in a key clin­i­cal tri­al, which they lat­er said was large­ly re­solved and brought down to 3%.

This morn­ing No­var­tis is fol­low­ing up with plans to in­vest €78.8 mil­lion in­to their cell and gene ther­a­py man­u­fac­tur­ing op­er­a­tions in Switzer­land in their lat­est stab at fix­ing the prob­lems.

But af­ter record­ing on­ly $16 mil­lion in sales for Q2, the big ques­tion loom­ing for No­var­tis is why pa­tients would want to take a risk with their ther­a­py if an­oth­er provider comes in­to the mar­ket which can do bet­ter on a com­plex task: ex­tract­ing T cells from pa­tients, mod­i­fy­ing them and then in­fus­ing pa­tients with a ther­a­peu­tic punch?

You can be sure that the top crew at Gilead and Kite — work­ing with a be­spoke man­u­fac­tur­ing site — are pay­ing close at­ten­tion.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Emma Walmsley, GlaxoSmithKline CEO (Credit: Fang Zhe/Xinhua/Alamy Live News)

The fire un­der Glax­o­SmithK­line's Em­ma Walm­s­ley grows as an­oth­er well-known ac­tivist in­vestor grabs its pitch­fork — re­port

Bluebell Capital Partners, a proxy brawler fresh off a campaign to oust global food giant Danone’s CEO and most of its board of directors, has bought a stake in UK drugmaker GlaxoSmithKline with its eyes trained directly on Emma Walmsley, the Financial Times reported Wednesday.

The London-based hedge fund joins another notorious activist firm in Paul Singer’s Elliott Management, which earlier this year called for a shakeup in leadership at GSK to handle what the company described as a wealth of riches across the drug giant’s portfolio hindered by limited vision from top staff.

FDA au­tho­rizes Pfiz­er's vac­cine boost­er for se­niors, those at high risk for se­vere Covid-19

The Biden administration’s goal of kicking off its booster shot drive for the entire US population this week is not quite going as planned.

First, Pfizer applied for approval of a supplemental application for the booster shots, but since last Friday’s adcomm reviewing them, the plan has devolved into an EUA, which the FDA issued late Thursday evening.

The population that is now eligible for the booster, six months after receiving the first pair of Pfizer-BioNTech vaccines, also narrowed from what Pfizer applied for (everyone who’s eligible for the initial Pfizer shots) to just those who are 65 or older, or at high-risk of a Covid infection, including health care workers and others with occupational hazards.

Stéphane Bancel, AP Images

Fi­nal analy­sis of US-fund­ed Mod­er­na Covid vac­cine tri­al shows 98% ef­fi­ca­cy against se­vere dis­ease

A final look at the results of the placebo-controlled Moderna trial in the New England Journal of Medicine, published Thursday afternoon, shows how the vaccine continues to prevent Covid-19 and severe cases after more than five months following the second shot.

Of the more than 30,000 enrolled in the trial that ultimately led to the vaccine’s EUA, only two people in the vaccine group got a severe form of the disease, compared to 106 in the placebo group — leading to an efficacy of 98%.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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Jean Bennett (Brent N. Clarke/Invision/AP Images)

Lux­tur­na in­ven­tor Jean Ben­nett starts a new gene ther­a­py com­pa­ny to tack­le rare dis­eases left be­hind by phar­ma, VCs

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

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