FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA’s work­load buck­les un­der the strain, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Pe­ter Marks ap­peared be­fore a vir­tu­al SVB Leerink au­di­ence yes­ter­day and said that his staff at FDA’s CBER is on the verge of work­ing around the clock. Man­u­fac­tur­ing in­spec­tions, pol­i­cy work and spon­sor com­mu­ni­ca­tions have all been pushed down the to-do list so that they can be re­spon­sive to Covid-re­lat­ed in­ter­ac­tions. And the agency’s ob­jec­tive right now? “To save as many lives as we can,” Marks said, liken­ing the mor­tal­i­ty on the cur­rent out­break as equiv­a­lent to “a nu­clear bomb on a small city.”

Pe­ter Marks FDA

The FDA is work­ing as hard as they can to win back the con­fi­dence of the gen­er­al pub­lic, Marks added. “There are forces cur­rent­ly that are cre­at­ing … in­cred­i­ble doubt in vac­cines in this coun­try … it makes me shud­der.”

Lat­er that day at a cam­paign ral­ly in Flori­da, Pres­i­dent Don­ald Trump cast as­per­sions on the mo­ti­va­tions of FDA staff to in­sti­tute stronger guide­lines for the com­ing vac­cine EUAs. Us­ing the pro­noun they, Trump cit­ed un­named of­fi­cials, telling his sup­port­ers: “They are try­ing to do a lit­tle bit of a po­lit­i­cal hit. ‘Let’s de­lay the vac­cine just a lit­tle bit.’ Did you no­tice that?”

The agency has been square­ly in his sights since an Aug. 22 tweet where he said: “The deep state, or who­ev­er, over at the FDA is mak­ing very dif­fi­cult for drug com­pa­nies to get peo­ple in or­der to test the vac­cines and ther­a­peu­tics. Ob­vi­ous­ly, they are hop­ing to de­lay the an­swer un­til af­ter No­vem­ber 3rd. Must fo­cus on speed, and sav­ing lives!”

Mean­while, FDA chief Stephen Hahn ap­pears to be get­ting no sup­port from his di­rect su­pe­ri­or, Alex Azar. The HHS sec­re­tary told Trump on Wednes­day that Hahn act­ed alone in push­ing the new guide­lines, ac­cord­ing to a Politi­co re­port that cites a se­nior ad­min­is­tra­tion of­fi­cial and an­oth­er per­son briefed on the call. Now, se­nior Trump of­fi­cials want to see a “fuller jus­ti­fi­ca­tion” from Hahn and the FDA.

While the new stan­dards may still be ap­proved by Trump, their cur­rent sta­tus is un­clear.

Marks’ CBER has 1,200 staffers tasked with reg­u­lat­ing bi­o­log­ics, cell ther­a­pies, and vac­cines. At the SVB Leerink fo­rum he con­firmed that, af­ter a pos­i­tive read­out from one or more of the piv­otal tri­als, the tri­als would be un­blind­ed, al­low­ing spon­sors and the agency to mon­i­tor pa­tients for Covid-19. Elec­tron­ic phar­ma­covig­i­lance will be used to mon­i­tor ef­fi­ca­cy for short, medi­um and long-term im­mu­ni­ty.

Marks went on to ex­plain that a stream­lined EUA re­view will al­low for a flex­i­ble CMC eval­u­a­tion. The usu­al bits like con­sis­ten­cy be­tween lots and some el­e­ments of QA/QC val­i­da­tion can be sub­mit­ted in a fuller BLA, af­ter the EUA.

For a look at all End­points News coro­n­avirus sto­ries, check out our spe­cial news chan­nel.

Un­lock­ing ESG strate­gies for growth with Gilead Sci­ences

RBC Capital Markets explores what is material in ESG for biopharma companies with the ESG leads at Gilead Sciences. Gilead has long focused on sustainability but recognized a more robust framework was needed. Based on a materiality assessment, Gilead’s ESG strategy today focuses first on drug access and pricing, while also addressing D&I and climate change. Find out why Gilead’s board is “acutely aware” of the contribution that ESG makes to firm’s overall success.

What con­tro­ver­sy? Eli Lil­ly plots Alzheimer's BLA fil­ing lat­er this year as FDA taps more an­ti-amy­loid drugs as break­throughs

The FDA is keeping the good news coming for Alzheimer’s drug developers. And Eli Lilly is taking them up on it.

Amid continued controversy around whether Biogen’s new flagship drug, Aduhelm, should have been approved at all — and swelling, heated debates surrounding its $56,000 price tag — the agency had no issue handing them and their Japanese partner Eisai a breakthrough therapy designation for a second anti-amyloid beta antibody, lecanemab, late Wednesday.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 110,100+ biopharma pros reading Endpoints daily — and it's free.

New FDA doc­u­ments show in­ter­nal dis­sent on Aduhelm ap­proval

In a lengthy review document and a pair of memos from top officials, the FDA released on Tuesday night its most detailed argument yet for approving Biogen’s intensely controversial Alzheimer’s drug aducanumab.

The documents amount to an agency attempt to quench the firestorm their decision kindled, as outside advisors members resigned and experts warned that an unproven drug now could stretch Medicare’s budget to a breaking point. Ultimately, the documents show how CDER director Patrizia Cavazzoni and Office of New Drugs director Peter Stein both concurred with FDA neuroscience head Billy Dunn on the accelerated approval while the staff at FDA’s Office of Biostatistics did not think an approval was warranted.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 110,100+ biopharma pros reading Endpoints daily — and it's free.

Hervé Hoppenot, Incyte CEO (Jeff Rumans)

ODAC echoes FDA con­cern over In­cyte PD-1, as Paz­dur sig­nals broad­er shift for ac­cel­er­at­ed ap­proval

After the FDA lambasted their PD-1 ahead of an adcomm earlier this week, Incyte ran into new trouble Thursday as ODAC panelists voted against an accelerated OK by a wide margin.

Members of the Oncologic Drugs Advisory Committee recommended with a 13-4 vote to defer a regulatory decision on Incyte’s retifanlimab until after more data can be collected from a placebo-controlled trial. The PD-1 therapy is due for a PDUFA date in late July after receiving priority review earlier this year.

Richard Pazdur (vis AACR)

FDA en­cour­ages in­clud­ing in­cur­able can­cer pa­tients in tri­als, re­gard­less of pri­or ther­a­pies

The FDA on Thursday called to include those with incurable cancers (when there is no potential for cure or for prolonged/near normal survival) in appropriate clinical trials, regardless of whether they have received existing alternative treatments.

Historically, many cancer clinical trials have required that participating patients previously received multiple therapies, according to Richard Pazdur, director of the FDA’s Oncology Center of Excellence.

Karen Flynn, Catalent

Q&A: When the pan­dem­ic struck, Catal­en­t's CCO had just joined the team

Karen Flynn came aboard Catalent’s team just in time.

The company was going through a surge of changes, and she had been brought over from her role as CCO of West Pharmaceutical Services to serve in the same capacity for the New Jersey-based CDMO. Then a few months later, the pandemic was in full-force.

Since then, Catalent’s been in hyper-expansion mode. In early May, it acquired Promethera’s Hepatic Cell Therapy Support SA subsidiary and its 32,40-square-foot facility in Gosselies, Belgium. Prior to that, the company acquired Belgian CDMO Delphi Genetics, wrapped up the expansion of an already-existing site in Madison, WI and added an ultra-low temperature freezer partner in Sterling. As Emergent has botched millions of doses of AstraZeneca’s vaccine, the company has swooped in to move that production to its Maryland plant as well.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 110,100+ biopharma pros reading Endpoints daily — and it's free.

On heels of Aduhelm ap­proval, Bris­tol My­ers jumps back in­to Alzheimer's race

Bristol Myers Squibb last put major resources behind an Alzheimer’s drug nearly a decade ago, when their own attempt at targeting amyloid flamed out in mid-stage studies. They invented another molecule, a Tau-targeted antibody, but jettisoned it to Biogen in 2017 as they dropped out of neuroscience altogether.

But on Thursday, the New York pharma announced they were getting back in the game. Bristol Myers exercised an $80 million option to bring a tau-targeted antibody from Prothena into a Phase I study. The opt-in, which Bristol Myers triggered ahead of analyst expectations, opens the door for another $1.7 billion in milestones down the road.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 110,100+ biopharma pros reading Endpoints daily — and it's free.

Elizabeth Warren (Michael Brochstein/Sipa USA)(Sipa via AP Images)

Sen­a­tors call for hear­ing to ex­am­ine how Medicare will han­dle Bio­gen's new Alzheimer's drug

Two top Senate Finance committee senators on Thursday called for a hearing to examine the questions and challenges for Medicare arising from the FDA’s recent approval of Biogen’s Aduhelm, the controversial new drug approved to treat Alzheimer’s disease.

In a letter to Senate Finance chair Ron Wyden (D-OR) and ranking member Mike Crapo (R-ID), subcommittee chair Elizabeth Warren (D-MA) and Bill Cassidy (R-LA) hinted at making policy changes to enable Medicare to more directly connect prescription drug pricing to clinical effectiveness. They raised questions about the “dramatic implications for our health care system” from the approval, which they said “stretch well beyond the scope of FDA’s jurisdiction.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 110,100+ biopharma pros reading Endpoints daily — and it's free.

James Peyer, Cambrian

Can a cell ther­a­py treat mus­cu­lar dy­s­tro­phy? A Ger­man bil­lion­aire's an­ti-ag­ing start­up is try­ing to find out

Gene therapy companies have faced huge hurdles trying to deliver healthy genes into muscular dystrophy patients’ muscle cells, so here’s an idea: Why don’t we just replace the muscle cells themselves?

Over the last two years, Vita Therapeutics has been exploring that possibility, building on early stem cell work from Johns Hopkins professor Peter Andersen. And on Tuesday they announced a $32 million Series A to begin to move their first therapy into the clinic, where they hope it will help rebuild muscle in patients with a type of dystrophy that afflicts the arms and legs.