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.”

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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Sanofi, GSK tout 72% Omi­cron ef­fi­ca­cy in PhI­II tri­al of next-gen, bi­va­lent shot — with an eye to year-end roll­out

Sometimes, being late can give you an advantage.

That’s what Sanofi and GSK are trying to say as the Big Pharma partners report positive results from a late-stage trial of their next-gen bivalent Covid-19 vaccine, which was designed to protect against both the original strain of the SARS-CoV-2 virus and the Beta variant. Specifically, against Omicron, they note, the vaccine delivered 72% efficacy in all adults and 93.2% in those previously infected.

Alarmed by side ef­fect, FDA slaps clin­i­cal hold on Sarep­ta's next-gen Duchenne drug

Sarepta Therapeutics’ next-gen Duchenne muscular dystrophy drug has been hit with a clinical hold after investigators flagged a serious case of low magnesium levels in one patient’s blood.

Screening and dosing will be halted in what is known as Part B of the Phase II MOMENTUM study, which has enrolled about half of the planned patients. Sarepta said it will be submitting information on all cases of the condition, known as hypomagnesemia, per the FDA’s request and proposing some changes to the risk mitigation and safety monitoring plan.

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