Opin­ion: Biden needs to step up and nom­i­nate an FDA com­mis­sion­er

Enough with the wait­ing. Pres­i­dent Joe Biden needs to em­brace the FDA dur­ing this pan­dem­ic and kick­start the Sen­ate con­fir­ma­tion process for the next per­ma­nent com­mis­sion­er.

And this needs to hap­pen quick­ly. There are plen­ty of qual­i­fied can­di­dates for the role out­side of act­ing com­mis­sion­er Janet Wood­cock and Josh Sharf­stein. Biden needs to move fast not be­cause the bio­phar­ma world strug­gles with even the per­cep­tion of FDA un­cer­tain­ty (it does), but be­cause thou­sands of FDA em­ploy­ees have been deal­ing with four years of po­lit­i­cal smears while putting in over­time to re­view thou­sands of Covid-19 drug and vac­cine tri­als, and to au­tho­rize three safe vac­cines in record time.

Their hard work should be re­paid with some good news and longer-term di­rec­tion, and it’s time the pub­lic saw state­ments and com­ments from the FDA com­mis­sion­er, not the act­ing com­mis­sion­er.

The po­lit­i­cal pres­sure and name call­ing (Trump once re­ferred to the FDA as a “big, old, slow tur­tle,”) have come and gone. Now, the FDA must wait (com­ing up on three months, plus at least an­oth­er cou­ple for the con­fir­ma­tion process) be­fore they get an of­fi­cial leader and po­lit­i­cal staff they can look to for en­cour­age­ment rather than em­bar­rass­ment from for­mer FDA com­mis­sion­er Stephen Hahn’s ex­ag­ger­a­tion of the ben­e­fits of con­va­les­cent plas­ma and sub­se­quent apol­o­gy tour, and the fir­ing of a for­mer Wash­ing­ton Times ed­i­tor turned com­mu­ni­ca­tions leader just 11 days in­to the job.

As many at the agency have ex­plained in pub­lic and pri­vate, it doesn’t re­al­ly mat­ter who’s at the very top (un­less it’s Scott Got­tlieb, every­one at FDA adores Dr. Got­tlieb) be­cause the com­mis­sion­er’s job is most­ly about serv­ing as the pub­lic face of the agency, and less about dri­ving the dai­ly agen­da, most of which is set months and years in ad­vance.

But it does mat­ter that there’s some­one in that top slot who’s a cham­pi­on for pub­lic safe­ty, com­pe­tent and who un­der­stands how the agency works, and what that role can and can­not do. No one wants an FDA com­mis­sion­er who needs to learn the ropes over the first 3-6 months, on­ly to leave af­ter a year.

So what’s the hold up for Biden?

For a pres­i­dent who has made clear his in­ten­tion to re­move his pre­de­ces­sor’s po­lit­i­cal traps around pub­lic health, and to res­ur­rect the bat­tered CDC, Biden re­mained mum on FDA and his fail­ure to name FDA’s per­ma­nent com­mis­sion­er seems to be very much about pol­i­tics.

At least four Sen­ate De­moc­rats – Mag­gie Has­san of New Hamp­shire, Ed Markey of Mass­a­chu­setts, Joe Manchin of West Vir­ginia and Cather­ine Cortez Mas­to of Neva­da – have pre-empt­ed Biden’s nom­i­na­tion and voiced their op­po­si­tion to the lead­ing con­tender for the job, Wood­cock.

The opi­oid epi­dem­ic re­mains at the fore­front of some of the sen­a­tors’ minds, and right­ly so, con­sid­er­ing the dev­as­ta­tion across the coun­try. Oth­ers have raised con­cerns on in­dus­try in­flu­ence, but Got­tlieb did a sol­id job of show­ing how pri­or in­dus­try ex­pe­ri­ence does not nec­es­sar­i­ly re­flect on life as a com­mis­sion­er.

What those sen­a­tors fail to ac­count for in their op­po­si­tion to Wood­cock is that the FDA is well past the days when a com­mis­sion­er is mak­ing uni­lat­er­al de­ci­sions for the en­tire agency, or when in­dus­try in­flu­ence might tip the scale. The bio­phar­ma in­dus­try owns the scale. Al­most 70% of the FDA’s bud­get for drugs is from in­dus­try user fees. Work­ing with in­dus­try isn’t just in the job de­scrip­tion, it’s a large part of the rea­son FDA ex­ists now. But it’s the oth­er, much more im­por­tant part of FDA’s role, the part where the FDA pro­tects the Amer­i­can pub­lic and Amer­i­cans can trust the voice of the com­mis­sion­er. That should push Biden to move for­ward with a per­ma­nent com­mis­sion­er.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; 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.

For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

A patient in Alaska receiving an antibody infusion to prevent Covid hospitalizations in September. All but one of these treatments has been rendered useless by Omicron (Rick Bowmer/AP Images)

How a tiny Swiss lab and two old blood sam­ples cre­at­ed one of the on­ly ef­fec­tive drugs against Omi­cron (and why we have so lit­tle of it)

Exactly a decade before a novel coronavirus broke out in Wuhan, Davide Corti — a newly-minted immunologist with frameless glasses and a quick laugh — walked into a cramped lab on the top floor of an office building two hours outside Zurich. He had only enough money for two technicians and the ceiling was so low in parts that short stature was a job requirement, but Corti believed it’d be enough to test an idea he thought could change medicine.

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