Albert Bourla, Pfizer CEO (Patrick Semansky/AP Images)

FDA takes a rare step and asks Pfiz­er to sub­mit a Covid-19 vac­cine EUA for the youngest chil­dren

What does the FDA know that we don’t? Hope­ful­ly a lot.

With­out of­fer­ing much de­tail, the FDA yes­ter­day af­ter­noon asked Pfiz­er to send over a rolling sub­mis­sion to amend its Covid-19 vac­cine EUA to in­clude chil­dren 6 months to un­der 5 years of age.

The tricky part in mak­ing such a re­quest is that last month, Pfiz­er an­nounced that its vac­cine (a 3 µg dose for the youngest pop­u­la­tion) demon­strat­ed non-in­fe­ri­or­i­ty for the 6- to 24-month-old pop­u­la­tion, when com­pared to the 16- to 25-year-old pop­u­la­tion in which high ef­fi­ca­cy was demon­strat­ed, but not for the 2- to un­der 5-year-old pop­u­la­tion.

The com­pa­ny said it would con­tin­ue to study the vac­cine in these youngest chil­dren with a third dose, and doesn’t seem to be chang­ing its tune even with this lat­est FDA re­quest.

“Ul­ti­mate­ly, we be­lieve that three dos­es of the vac­cine will be need­ed for chil­dren 6 months through 4 years of age to achieve high lev­els of pro­tec­tion against cur­rent and po­ten­tial fu­ture vari­ants. If two dos­es are au­tho­rized, par­ents will have the op­por­tu­ni­ty to be­gin a COVID-19 vac­ci­na­tion se­ries for their chil­dren while await­ing po­ten­tial au­tho­riza­tion of a third dose,” Pfiz­er CEO Al­bert Bourla said in a state­ment.

No safe­ty con­cerns were iden­ti­fied in that pri­or analy­sis of the 3 µg dose da­ta among chil­dren 6 months to un­der 5 years of age, Pfiz­er said.

While the FDA of­ten re­quests that com­pa­nies pro­vide ad­di­tion­al safe­ty or ef­fi­ca­cy da­ta (usu­al­ly be­fore a new drug or vac­cine is ap­proved or au­tho­rized), the agency rarely re­quests a spe­cif­ic sub­mis­sion, but act­ing FDA com­mis­sion­er Janet Wood­cock said this is a pri­or­i­ty right now for the agency.

“I don’t think au­tho­riz­ing two dos­es in chil­dren ages 2 to 4 years of age where ef­fec­tive­ness in this age group hasn’t been con­firmed is go­ing to con­vince the ma­jor­i­ty of par­ents to vac­ci­nate their chil­dren,” Nor­man Bay­lor, pres­i­dent and CEO of Bi­o­log­ics Con­sult­ing and a for­mer head of the FDA’s vac­cine of­fice, told STAT News. “If the vac­cine in this age co­hort is a three-dose vac­cine, FDA should re­view the da­ta from the three-dose se­ries be­fore au­tho­riz­ing the vac­cine.”

On Feb. 15, the FDA will con­vene its VRB­PAC, an ad­comm of out­side ad­vi­sors to dis­cuss this ap­pli­ca­tion. The com­mit­tee kicked off the Covid-19 vac­cine boost­er sea­son last fall by pledg­ing to not re­quire boost­ers for all, and then boost­ers were rec­om­mend­ed for all, and have proven to be help­ful in re­duc­ing cas­es and hos­pi­tal­iza­tions in this lat­est Omi­cron surge.

An­oth­er big ques­tion, as has been the case for the en­tire pan­dem­ic, is up­take in this youngest pop­u­la­tion. The FDA notes that as of Dec. 19, the Cen­ters for Dis­ease Con­trol and Pre­ven­tion said just 8.7 mil­lion Covid-19 vac­ci­na­tions have been giv­en to kids 5 through 11 years of age, which is on­ly about one-third of the 24 mil­lion US kids this age range. There are about 23 mil­lion chil­dren less than 5 years of age in the US, the FDA es­ti­mat­ed.

Hes­i­tan­cy has been a big is­sue na­tion­wide, with the US trail­ing be­hind the vac­ci­na­tion rates of oth­er wealthy na­tions around the globe. But Wood­cock pre­vi­ous­ly told End­points News that this fight might be about more than just vac­cines.

“Well the loss of trust seems to be a gen­er­al an­ti-au­thor­i­ty, an­ti-sci­ence thing,” she wrote. “Very strong streak of this in the US over the cen­turies. Sure, it would be fine to have an­oth­er tra­di­tion­al type voice mak­ing pub­lic health state­ments, but this may need to be fought out in the so­cial me­dia and oth­er cur­rent in­fo sources, oth­er­wise you are just preach­ing to the con­vert­ed. FDA would need more comms staff — our peo­ple are pret­ty ex­haust­ed and most­ly have the tra­di­tion­al skillset.”

FDA’s top vac­cine of­fi­cial Pe­ter Marks said in a re­cent keynote, re­flect­ing on some of the things that keep him up at night, “The lack of trust in a ba­sic pub­lic health mea­sure that saves lives is cat­a­stroph­ic in this coun­try.”

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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Zhi Hong, Brii Biosciences CEO

Brii Bio­sciences stops man­u­fac­tur­ing Covid-19 an­ti­body com­bo, plans to with­draw EUA re­quest

Brii Biosciences said it will stop manufacturing its Covid-19 antibody combination, sold in China, and is working to withdraw its emergency use authorization request in the US, which it started in October 2021.

The Beijing and North Carolina biotech commercially launched the treatment in China last July but is now axing the work and reverting resources to other “high-priority programs,” per a Friday update. The focus now is namely hepatitis B viral infection, postpartum depression and major depressive disorders.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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In­cyte hit by CRL on ex­tend­ed-re­lease JAK tablets, mud­dy­ing plans for Jakafi fran­chise ex­pan­sion

The FDA has rejected Incyte’s extended-release formulation of ruxolitinib tablets, in a surprise setback for the company’s plans to build on its blockbuster Jakafi franchise.

The ruxolitinib XR tablets are designed to be taken once a day, whereas Jakafi is indicated for twice daily dosage (although some patients can take it once daily).

According to Incyte, the FDA acknowledged in its complete response letter that the study submitted in the NDA “met its objective of bioequivalence based on area under the curve (AUC) parameters but identified additional requirements for approval.”

Eu­ro­pean Com­mis­sion de­lays pro­pos­al for ma­jor changes to phar­ma leg­is­la­tion

The European Commission has once again delayed the release of its proposal for an overhaul of the continent’s pharmaceutical legislation.

The release, previously anticipated on March 29, will occur “slightly later” than expected due to the “very busy College agendas of the last few weeks,” a Commission spokesperson told Endpoints News via email.

While the agency hasn’t provided an updated timeline, the spokesperson said the agenda is “always indicative and adoption dates of Commission proposals may change any time, especially when these proposals concern reforms of complex legislations of major importance.”

Sergio Traversa, Relmada Therapeutics CEO

Rel­ma­da makes 'crit­i­cal changes' to PhI­II tri­al to try and save de­pres­sion drug

Relmada Therapeutics is making changes to its Phase III study of its lead drug for major depressive disorder, in an attempt to avoid problems with a prior trial that showed little difference between the drug and a placebo.

That failure in October wiped 80% from Relmada’s stock price, and was followed by another negative readout a few months later. In both cases, the company said that there had been trial sites that were associated with what it called surprising placebo effects that skewed the results compared with the drug, REL-1017.

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Paul Song, NKGen Biotech CEO

NK cell ther­a­py-fo­cused biotech eyes SPAC deal

A small, Santa Ana-based biotech created in 2017 is looking to enter a SPAC deal as it lays out plans to begin trials in its lead cell therapy candidates and bring on new executives.

Graf Acquisition Corp. IV and NKGen Biotech announced Thursday, with few other details, that the two companies signed a non-binding letter of intent to “pursue a business combination.” Graf Acquisition II and III withdrew their IPOs last year.

Peter Hecht, Cyclerion Therapeutics CEO

Hard pressed for cash, Cy­cle­ri­on looks for help fund­ing rare dis­ease drug

Cyclerion Therapeutics may have the design of a Phase IIb study ready to go, but it’s scrambling for a way to fund it.

The company said in a press release that it’s “actively evaluating the best combination of capital, capabilities, and transactions available to it to advance the development of zagociguat,” its lead candidate for a rare, genetic mitochondrial disease known as MELAS.

In a separate SEC filing, Cyclerion once again flagged “substantial doubt about (its) ability to continue as a going concern.” As of the end of 2022, it had cash and cash equivalents of only $13.4 million.