UP­DAT­ED: FDA says ben­e­fits of Covid-19 vac­cine for younger chil­dren out­weigh risks

The FDA said in its analy­sis of Pfiz­er’s da­ta that the ben­e­fits of the 2-dose pri­ma­ry se­ries in chil­dren 5-11 years of age out­weigh the risks, even in sit­u­a­tions where there’s a low num­ber of Covid-19 cas­es cir­cu­lat­ing.

The agency mod­eled six dif­fer­ent sce­nar­ios, look­ing at dif­fer­ent po­ten­tial Covid-19 case­loads — from peak Delta surge to the low­est record­ed in­ci­dence of Covid-19 from last June — find­ing that the ben­e­fits of the vac­cine out­weighed the risks in every sce­nario, and “the over­all ben­e­fits of the vac­cine may still out­weigh the risks un­der this low­est in­ci­dence sce­nario.”

While ac­knowl­edg­ing there may be ex­cess hos­pi­tal­iza­tions due to vac­cine-re­lat­ed my­ocardi­tis/peri­cardi­tis com­pared to pre­vent­ed hos­pi­tal­iza­tions due to Covid-19 in that low­est in­ci­dence sce­nario, “If the my­ocardi­tis/peri­cardi­tis risk in this age group is low­er than the con­ser­v­a­tive as­sump­tion used in the mod­el, the ben­e­fit-risk bal­ance would be even more fa­vor­able,” the agency said.

Pfiz­er will present its case to the FDA’s vac­cine ad­comm to­mor­row, seek­ing au­tho­riza­tion for a low­er-dose ver­sion of its Covid-19 vac­cine for kids ages 5 through 12, which the Biden ad­min­is­tra­tion said will like­ly be­gin rolling out next week.

Two pri­ma­ry dos­es of the 10 µg vac­cine (the dose for those ages 12 and up is 30 μg) giv­en 3 weeks apart in this group of chil­dren “have shown a fa­vor­able safe­ty and tol­er­a­bil­i­ty pro­file, ro­bust im­mune re­spons­es against all vari­ants of con­cern in­clud­ing Delta, and vac­cine ef­fi­ca­cy of 90.7% against lab­o­ra­to­ry-con­firmed symp­to­matic COVID-19,” the com­pa­ny said in brief­ing doc­u­ments.

But on the top­ic of rare heart-re­lat­ed side ef­fects, which have cropped up among the mR­NA vac­cines when used in mil­lions of peo­ple, Pfiz­er made clear that the num­ber of par­tic­i­pants “in the cur­rent clin­i­cal de­vel­op­ment pro­gram is too small to de­tect any po­ten­tial risks of my­ocardi­tis as­so­ci­at­ed with vac­ci­na­tion.” Hence the need for FDA to run its mod­els.

Five long-term safe­ty stud­ies in chil­dren in this age range will be con­duct­ed to bet­ter un­der­stand the risks, the com­pa­ny said, in­clud­ing a 5-year fol­low-up study to eval­u­ate long term se­que­lae of post-vac­ci­na­tion my­ocardi­tis/peri­cardi­tis.

The bar for an EUA for this younger pop­u­la­tion is like­ly to be high­er than for adults. Ac­cord­ing to re­cent CDC da­ta, among chil­dren ages 5 to less than 12, there have been about 1.8 mil­lion con­firmed and re­port­ed Covid-19 cas­es, about 8600 hos­pi­tal­iza­tions, and 143 Covid-re­lat­ed deaths in the US. That’s just a small frac­tion of the 45 mil­lion to­tal US Covid-19 cas­es, and more than 700,000 deaths.

Still, the ex­pec­ta­tion is that the Pfiz­er vac­cine for kids will be ready in the near term, “mean­ing weeks, not months,” Biden said at a CNN town hall on Thurs­day. He al­so not­ed that there are over 800,000 sites right now where chil­dren can get their vac­cines, once they’re au­tho­rized, and his ad­min­is­tra­tion is work­ing with child­care cen­ters and pe­di­a­tri­cians to make the vac­cines more ac­ces­si­ble.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; 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.

The slate of products we’re offering here at Endpoints is continuing to grow, and it’s not just limited to editorial. If you haven’t, do visit your reader profile to see if there are any other weekly newsletters you’re interested in — as each comes with its own exclusive content. And don’t miss the publisher’s note from Arsalan Arif on Endpoints Studio, our latest avenue for advertising on Endpoints.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

Re­searchers move clos­er to de­ci­pher­ing blood clots from As­traZeneca, J&J's Covid-19 vac­cines

Researchers may be nearing an answer for the mysterious and life-threatening blood clots that appeared on very rare occasions in people who received the J&J or AstraZeneca Covid-19 vaccine.

The new work builds on an early hypothesis researchers in Norway put forward last spring, when the cases first cropped up. They proposed the events were similar to blood clots that can occur in a small subset of patients who receive heparin, one of the most commonly used blood thinners.

Janet Woodcock (AP Images)

Janet Wood­cock plots her fu­ture at FDA, with se­nior ad­vi­sor role to fall back on if Califf wins con­fir­ma­tion

Acting FDA commissioner Janet Woodcock has been the face of just about every drug approval decision at the agency since the turn of the century. Since the pandemic began, she’s moved between the top of the drugs center to the head of therapeutics at Operation Warp Speed, leading the drive for work on Covid-targeted mAbs and antivirals.

Looking forward — and pending a quick Senate confirmation to cement Rob Califf’s return to the top of FDA early next year — Woodcock’s role at the agency will again be in flux.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

Richard Pazdur (via AACR)

Ac­cel­er­at­ed ap­proval re­forms need mean­ing­ful con­fir­ma­to­ry tri­al im­prove­ments, pro­fes­sors write in Sci­ence

Outside of Covid-19, 2021 has been the year of the accelerated approval.

Beginning last spring, FDA openly challenged six “dangling” accelerated approvals (hadn’t confirmed their clinical benefit yet), three of which were later pulled by the companies.

Then in June, FDA pulled out the accelerated approval pathway, seemingly out of nowhere, to sign off on Biogen’s controversial Alzheimer’s drug Aduhelm. It hadn’t even been mentioned at the drug’s adcomm.

In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.