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

UP­DAT­ED: FDA's Pe­ter Marks to Con­gress: Youngest kids vac­cine won't need to hit 50% ef­fi­ca­cy mark

The FDA’s top vac­cine leader told a con­gres­sion­al com­mit­tee on Fri­day af­ter­noon that al­though the adult vac­cines had to meet a 50% thresh­old for ef­fi­ca­cy against Covid-19 in­fec­tions, that same stan­dard will not need to be met for the vac­cines for the youngest group of chil­dren, for which a vac­cine is not yet avail­able.

The agency is cur­rent­ly re­view­ing da­ta from Mod­er­na’s two-shot vac­cine for this youngest group as it awaits fur­ther da­ta from Pfiz­er on its po­ten­tial three-dose shot. Mod­er­na has said its vac­cine is 51% ef­fec­tive in chil­dren 6 months to 2 years of age and 37% ef­fec­tive in 2- to 5-year-olds. The agency al­so pre­vi­ous­ly sched­uled and then can­celed an ad­comm to re­view da­ta on two dos­es of Pfiz­er’s vac­cine for chil­dren un­der the age of 5.

Ac­cord­ing to a read­out of the meet­ing from the House se­lect sub­com­mit­tee on the coro­n­avirus cri­sis, Marks ex­plained that the FDA would not with­hold au­tho­riza­tion — de­spite pre­vi­ous guid­ance — for a pe­di­atric vac­cine sole­ly be­cause it did not reach a 50% ef­fi­ca­cy thresh­old at block­ing symp­to­matic in­fec­tions. All of the oth­er adult and chil­dren’s vac­cines cur­rent­ly au­tho­rized in the US have lost sig­nif­i­cant amounts of ef­fi­ca­cy due to the Omi­cron vari­ant, but they still re­main ef­fec­tive at re­duc­ing the risk of se­vere dis­ease, hos­pi­tal­iza­tion and death.

“If these vac­cines seem to be mir­ror­ing ef­fi­ca­cy in adults and just seem to be less ef­fec­tive against Omi­cron like they are for adults, we will prob­a­bly still au­tho­rize,” Marks said.

Jim Clyburn

Mean­while, Marks di­rect­ly ad­dressed con­cerns that the agency might slow-play the Mod­er­na ap­pli­ca­tion and wait to re­view both ap­pli­ca­tions to­geth­er, say­ing that the VRB­PAC meet­ings set for next month will be moved up if nec­es­sary.

“Ob­vi­ous­ly if we get through re­views faster, then we will send them to com­mit­tees soon­er,” Marks said, ac­cord­ing to Rep. Jim Clyburn’s ac­count of the meet­ing. Marks al­so told Clyburn and his staff that VRB­PAC mem­bers have re­served ear­li­er dates, en­abling FDA to po­ten­tial­ly “move dates up even by a week for any of these re­views.”

But he al­so ex­plained that re­view­ing these vac­cines for the youngest chil­dren in­volves more “com­plex­i­ty” than the re­view of adult vac­cines, and the agency’s re­view process may re­quire more time.

He al­so said that the emer­gence of new vari­ants made the process of re­view­ing vac­cines more com­plex, ex­plain­ing it “was a lit­tle sim­pler for the orig­i­nal sub­mis­sions in late 2020 when we didn’t have the vari­ants and tons of im­muno­genic­i­ty da­ta to go through.

“At the end of the day, we want peo­ple to have con­fi­dence in get­ting vac­ci­nat­ed,” he said, ac­cord­ing to Clyburn. Marks al­so re­it­er­at­ed the im­por­tance of vac­ci­na­tions to pro­tect chil­dren against the coro­n­avirus, stat­ing: “We need to get more kids vac­ci­nat­ed, not just in the younger than 5 age range, but al­so old­er than 5.”

Since mak­ing these re­marks to Clyburn, Marks told a me­dia brief­ing ear­li­er this week that he will main­tain the stan­dard for 50% ef­fi­ca­cy for this shot for the youngest kids group, but that “it’s pos­si­ble in sub­pop­u­la­tions they might be a lit­tle less ef­fec­tive than 50%.”

Ed­i­tor’s note: Ar­ti­cle up­dat­ed with Marks’ lat­est com­ment and to note Mod­er­na’s stat­ed ef­fi­ca­cy for its vac­cine for these two groups of the un­der 6 pop­u­la­tion.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

FDA preps for DMD drug gener­ics as Sarep­ta has yet to fin­ish its con­fir­ma­to­ry tri­al

The FDA typically releases guidance to help generic drug manufacturers develop new copycats of small molecule drugs, oftentimes in preparation for a brand name product’s patents or exclusivity to expire.

This week, FDA released such bioequivalence guidance for any generic drugmakers looking to take on Sarepta’s Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen), even though the drug’s sponsor has yet to convert the accelerated approval to a full approval, showing clinical benefit.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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Isao Teshirogi, Shionogi president and CEO (Kyodo via AP Images)

Sh­ionogi's Covid an­tivi­ral lands first ap­proval in Japan's new emer­gency ap­proval path­way

Japanese regulators on Tuesday signed off on Shionogi’s homegrown antiviral for Covid-19, known as Xocova (ensitrelvir), making it the first approval under Japan’s emergency regulatory approval system.

The emergency approval, following a back-and-forth with regulators since last February, is based on a safety profile with more than 2,000 patients who have accessed the pill, and clinical symptomatic efficacy for five typical Omicron-related symptoms (primary endpoint) and antiviral efficacy (key secondary endpoint) in patients with mild to moderate SARS-CoV-2 infection, regardless of risk factors or vaccination status, and during the Omicron-dominant phase of the pandemic.

Stanley Erck, Novavax CEO (Andrew Harnik/AP Images)

No­vavax pulls out of Covid-19 vac­cine al­liance with Gavi

Novavax is pulling out of its Covid-19 vaccine deal with Gavi, the Vaccine Alliance, a global partnership tasked with ensuring vaccine access in lower-income countries, following an alleged contract violation.

The Maryland-based company claimed on Friday that Gavi failed to purchase at least 350 million doses of its protein-based vaccine Nuvaxovid by the end of the year, per an advanced purchase agreement. Gavi, the World Health Organization and the Coalition for Epidemic Preparedness Innovations (CEPI) are co-leaders of COVAX, an effort to ensure that all participating countries, regardless of income levels, have access to vaccines.

Days af­ter re­port­ing PhI­II fail­ure, GSK pulls BC­MA drug from US mar­ket — but it's not giv­ing up en­tire­ly yet

GSK is pulling its BCMA-targeting drug from the US market, ending a short, two-year run for a high-profile product that, among other things, was hailed for marking the pharma giant’s return to oncology.

The company is initiating the process for withdrawal at the request of the FDA, which in turn was based on the negative readout of a confirmatory Phase III trial earlier this month. In that trial, GSK’s Blenrep failed to extend progression-free survival over standard of care for patients with multiple myeloma who have received at least two prior lines of therapy.

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