Peter Marks (Jim Lo Scalzo/Pool via AP Images)

Tur­moil at CBER: Pe­ter Marks grabs con­trol of FDA's Of­fice of Vac­cines ahead of 2 key ca­reer leader de­par­tures

FDA’s top vac­cine of­fi­cial Pe­ter Marks is pulling the plug on a months-long tran­si­tion for two top ca­reer vac­cine of­fi­cials who abrupt­ly called it quits in late Au­gust.

Mar­i­on Gru­ber, di­rec­tor of the FDA’s Of­fice of Vac­cines Re­search & Re­view and 32-year vet­er­an of the agency, her deputy di­rec­tor Phil Krause, an­nounced their de­par­tures and then raised con­cerns with Covid-19 boost­er shots ahead of and dur­ing a re­cent Covid-19 boost­er vac­cine ad­vi­so­ry com­mit­tee.

“It is im­por­tant to al­low both Mar­i­on and Phil to have time to tran­si­tion be­fore their de­par­ture af­ter so many years with our Cen­ter,” Marks said in a memo to CBER staff on Mon­day morn­ing. “There­fore, I want to let you know that be­gin­ning to­day, Sep­tem­ber 27, I will be as­sum­ing re­spon­si­bil­i­ties as the Act­ing Of­fice Di­rec­tor, and Mar­i­on will tran­si­tion over­sight and man­age­ment of the ac­tiv­i­ties of the of­fice to me.”

The memo fol­lows a hec­tic and at-times con­fus­ing few weeks where both Gru­ber, who said she was leav­ing in Oc­to­ber, and Krause, who said he was leav­ing in No­vem­ber, an­nounced their de­par­tures from FDA, then raised con­cerns with the boost­er shots in the Lancet, and ques­tioned Pfiz­er’s da­ta at the FDA’s vac­cine ad­vi­so­ry com­mit­tee re­view­ing the boost­ers.

At the be­gin­ning of the Q&A ses­sion at the boost­er ad­comm with Pfiz­er and the FDA, Gru­ber gave Krause the op­por­tu­ni­ty to ques­tion Pfiz­er on the sta­tis­ti­cal mod­els used in a re­al-world study in Is­rael.

Krause claimed there was a dis­crep­an­cy be­tween the ef­fi­ca­cy Pfiz­er re­port­ed us­ing a spe­cif­ic mod­el, which said ef­fi­ca­cy af­ter 8 months fell to 58% from 61%, but num­bers cal­cu­lat­ed through a dif­fer­ent math­e­mat­i­cal process us­ing cas­es per per­son-years re­sult­ed in 93% ef­fi­ca­cy. These dis­parate fig­ures, Krause said, came from the same num­ber of cas­es in a study by Cal­i­for­nia health care com­pa­ny Kaiser Per­ma­nente.

Two for­mer FDA chief sci­en­tists — Jesse Good­man and Lu­ciana Bo­rio — raised con­cerns with the New York Times over Marks’ de­ci­sion to name him­self, with Good­man call­ing it, “ex­treme­ly un­usu­al and con­cern­ing.”

But oth­er for­mer se­nior of­fi­cials have tak­en on ad­di­tion­al roles as the FDA looks for a per­ma­nent re­place­ment. Janet Wood­cock was at one time head of both CDER and OND on an act­ing ba­sis, be­fore be­com­ing act­ing com­mis­sion­er, as the agency looked for a per­ma­nent OND leader.

Bio­Cen­tu­ry al­so re­port­ed Mon­day that two ad­di­tion­al FDA vac­cine re­view­ers quit their jobs as a re­sult of Marks’ an­nounce­ment. FDA me­dia re­la­tions di­rec­tor Stephanie Cac­co­mo did not re­spond to an End­points News re­quest to ver­i­fy those oth­er two de­par­tures.

Paul Of­fit, a vac­cine ex­pert and mem­ber of the FDA ad­comm that met to dis­cuss the boost­ers, told End­points he thought Gru­ber and Krause are leav­ing be­cause they didn’t like the way the boost­er process played out.

“They see their job as pro­tect­ing the pub­lic from phar­ma­ceu­ti­cal com­pa­nies’ prod­ucts that may not have not been ad­e­quate­ly test­ed and I think that’s what wor­ries them about this,” Of­fit said.

Saad Omer, di­rec­tor of Yale’s in­sti­tute for glob­al health, added, “A lot of spe­cial­ized ex­per­tise has been un­der­uti­lized and that’s a prob­lem.”

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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An­oth­er Cipla site lands a Form 483 over clean­ing is­sues and QC con­trols

A Cipla drug manufacturing site in India has once again landed in the crosshairs of FDA inspectors.

The facility in question is Cipla’s drug manufacturing facility in the village of Verna, in the state of Goa in India’s southwest. In a sign that foreign inspections might ramp up again, the FDA’s visit from Aug. 16 to Aug. 22 uncovered six observations.

The 11-page report noted that environmental monitoring at the site did not properly ensure that microbial contaminants were not making any impact in the aseptic filling areas. It also found that procedures meant to stop microbial contamination were not adequately conducted in aseptic areas of the facility.

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FDA ad­comm takes down Se­cu­ra Bio's leukemia drug af­ter fi­nal tri­al re­sults show po­ten­tial OS detri­ment

The FDA’s Oncologic Drugs Advisory Committee on Friday voted 8-4 against the benefit-risk profile of Secura Bio’s PI3K inhibitor Copiktra (duvelisib), which won approval in September 2018 as a third-line treatment for relapsed or refractory CLL or SLL, but updated pivotal trial results raised safety questions.

In addition to the serious and fatal toxicities of duvelisib, FDA speakers at the ODAC meeting pointed to an evolved treatment landscape for CLL and SLL, with targeted BTK or BCL2 inhibitors (front-line or second-line), and data pointing to a “potential detriment” in overall survival for duvelisib. But some ODAC members noted that the detriment was likely small and that there is some efficacy even as the data are difficult to interpret.

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FDA's out­side ex­perts vote in fa­vor of Fer­ring's fe­cal trans­plant for C. dif­fi­cile, set­ting the stage for Seres

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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Richard Pazdur, FDA's OCE director (Flatiron Health via YouTube)

FDA's OCE makes the case for ac­cel­er­at­ed ap­proval rid­er in user fee reau­tho­riza­tion

Four experts from the FDA’s Oncology Center of Excellence took to the New England Journal of Medicine yesterday to make the case for not only improving the agency’s ability to expeditiously pull dangling accelerated approvals when, on the rare occasion, confirmatory trials fail, but also better building “quality and efficiency into the AA on-ramp.”

The timely perspective arrives as Congress has exactly one week left to draft, release and sign off on the reauthorized user fee deals before layoff notices will be sent to drug reviewers. That package, which is likely to hitch a ride with the continuing resolution, may or may not include several policy riders (opposed by Republicans), including one that would allow the FDA to require confirmatory trials to be underway before an AA is granted, and would improve the process by which FDA can withdraw AAs.

As­traZeneca, Mer­ck cull one Lyn­parza in­di­ca­tion in heav­i­ly pre­treat­ed ovar­i­an can­cer pa­tients

Just one day after blockbuster Lynparza got access to another indication in China, its Big Pharma owners have decided to withdraw it in certain patients after reviewing Phase III data.

The two companies that work together on Lynparza decided to recall one of the indications several weeks ago in a specific type of ovarian cancer, Lynparza’s first indication when it was first FDA-approved in 2014. Initial data showed that rates of overall survival in patients with at least three rounds of chemo before getting on the PARP inhibitor were lower than in patients with less previous chemo treatment.

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