Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top ca­reer staff at the FDA has vowed not to let pol­i­tics over­rule sci­ence when look­ing at vac­cine da­ta this fall. But Alex Azar, who hap­pens to be their boss’s boss, ap­par­ent­ly won’t even give them a chance to stand in the way.

In a new mem­o­ran­dum is­sued Tues­day last week, the HHS chief stripped the FDA and oth­er health agen­cies un­der his purview of their rule mak­ing abil­i­ty, as­sert­ing all such pow­er “is re­served to the Sec­re­tary.” Sheila Ka­plan of the New York Times first ob­tained and re­port­ed the de­tails of the Sep­tem­ber 15 bul­letin.

Azar’s sig­na­ture is now re­quired on all new rule­mak­ing, which are typ­i­cal­ly signed by agency lawyers and bu­reau­crats.

“This was sim­ply push­ing a re­set but­ton,” claimed Azar’s top aide Bri­an Har­ri­son to the Times. “This is good gov­er­nance and should have no op­er­a­tional im­pact.”

It’s not im­me­di­ate­ly clear what the im­pact on this or­der will have on the agency’s day-to-day op­er­a­tions — es­pe­cial­ly on rou­tine mat­ters where it has long en­joyed po­lit­i­cal in­de­pen­dence. An agency of­fi­cial not au­tho­rized to speak with the me­dia told the Times they’re still “de­ci­pher­ing what Mr. Azar’s memo would mean for their work.”

The move comes just days af­ter a group of high-pro­file FDA ca­reer ser­vants — in­clud­ing Pe­ter Marks, Jeff Shuren, and Janet Wood­cock — wrote in a USA To­day op-ed that “we will work with agency lead­er­ship to main­tain FDA’s stead­fast com­mit­ment to en­sur­ing our de­ci­sions will con­tin­ue to be guid­ed by the best sci­ence.”

By tak­ing au­thor­i­ty that was once the clear do­main of pro­fes­sion­al staff, the new memo is in line with the cen­tral­iza­tion of pow­er across the fed­er­al gov­ern­ment in the hands of Trump loy­al­ists. And crit­ics note that it blazes a path where the FDA could be side-stepped for an emer­gency use au­tho­riza­tion on an ear­ly coro­n­avirus vac­cine. Er­ic Topol, di­rec­tor at the Scripps Re­search In­sti­tute, spelled out on Twit­ter a po­ten­tial sce­nario with Pfiz­er’s can­di­date:

Low­ell Schiller

The pow­er grab by Azar has ev­i­dent­ly dri­ven at least one es­tab­lished ca­reer ser­vant to the ex­its. On Thurs­day, Politi­co re­port­ed that the FDA’s prin­ci­pal as­so­ciate com­mis­sion­er for pol­i­cy, Low­ell Schiller, left the agency last week. Schiller, a Scott Got­tlieb hire, signed near­ly every FDA no­tice on the fed­er­al reg­is­ter and played a cen­tral role in writ­ing the FDA’s Covid-19 guid­ance. (Up­date: Politi­co now re­ports the de­par­ture was un­re­lat­ed to the memo.)

Af­ter the Times pub­lished its sto­ry, an HHS spokesper­son took to Twit­ter to again re­fute any sug­ges­tion that the FDA’s work would be slowed down but did not ad­dress con­cerns that the new memo could be used to over­rule de­ci­sions by FDA staff. “The memo ma­ligned in this sto­ry is a good gov­er­nance, house­keep­ing ac­tion, which will not slow down the im­por­tant work of the FDA or any HHS di­vi­sion,” the tweet said. Lat­er on Sun­day, the agency put out a FAQ-style re­sponse to news sto­ries on the memo but did not pro­vide the memo’s ac­tu­al text.

The HHS memo is one of the most far-reach­ing ac­tions in a decades-old quest by hard-right Amer­i­can con­ser­v­a­tives to crip­ple the reg­u­la­to­ry pow­ers of pro­fes­sion­als in ex­ec­u­tive-branch agen­cies. De­spite be­ing a con­gres­sion­al­ly del­e­gat­ed au­thor­i­ty, Pres­i­dent Trump and his po­lit­i­cal al­lies rou­tine­ly frame the is­sue of as one of fed­er­al em­ploy­ees act­ing like “un­ac­count­able bu­reau­crats.” Last year, while Trump was sign­ing ex­ec­u­tive or­ders aimed at pair­ing back rule­mak­ing across sev­er­al agen­cies, his pre­pared re­marks stat­ed that “all too of­ten guid­ance doc­u­ments are a back door for reg­u­la­tors to ef­fec­tive­ly change the laws and vast­ly ex­pand their scope and reach.”

Scott Got­tlieb

Scott Got­tlieb, a con­ser­v­a­tive who won wide praise across the po­lit­i­cal spec­trum for his tenure as Trump’s first FDA com­mis­sion­er, fought the en­croach­ment on the agency’s rule­mak­ing abil­i­ty through­out his en­tire tenure. He used the pow­ers as a cud­gel against to­bac­co and va­p­ing in­dus­tries, and ac­cord­ing to the Times re­port, even signed one of the last or­ders on it, hop­ing that his sig­na­ture, rather than a low­er-rank­ing staffer’s, would give his mea­sure stay­ing pow­er on the books.

Azar, who has now as­sert­ed him­self as the ul­ti­mate rule mak­er at the FDA, is Trump’s sec­ond HHS sec­re­tary, tak­ing of­fice in 2018. He was named pres­i­dent of the US di­vi­sion of Eli Lil­ly in 2012 and served on BIO’s board. He de­part­ed Lil­ly in 2017 when CEO David Ricks re­vamped the org chart.

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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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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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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‘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.

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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UP­DAT­ED: CMS to re­strict cov­er­age of Bio­gen's con­tro­ver­sial Alzheimer's drug to on­ly clin­i­cal tri­als

The Centers for Medicare and Medicaid Services on Tuesday said it will only pay for Biogen’s Aduhelm and other FDA-approved anti-amyloid monoclonal antibodies for Alzheimer’s disease under CMS-approved randomized controlled trials.

The draft national coverage decision, which insurers nationwide are likely to follow, makes clear that CMS will be looking for randomized controlled trials that “demonstrate a clinically meaningful benefit in cognition and function.” That will be a tough task for Biogen, which previously showed conflicting benefits from past Aduhelm trials that were initially cut short due to futility and then resurrected for the accelerated approval.

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.

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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