FDA post­pones ad­comm for Pfiz­er's Covid-19 vac­cine for the youngest age group

The FDA on Fri­day af­ter­noon said it’s no longer mov­ing for­ward with its plan to au­tho­rize two dos­es (while wait­ing for da­ta on a third dose) of Pfiz­er’s Covid-19 vac­cine for the youngest group of chil­dren, from 6 months through 4 years of age.

Pe­ter Marks FDA

“Based on the agency’s pre­lim­i­nary as­sess­ment, and to al­low more time to eval­u­ate ad­di­tion­al da­ta, we be­lieve ad­di­tion­al in­for­ma­tion re­gard­ing the on­go­ing eval­u­a­tion of a third dose should be con­sid­ered as part of our de­ci­sion-mak­ing for po­ten­tial au­tho­riza­tion,” FDA’s top vac­cine of­fi­cial Pe­ter Marks and act­ing FDA com­mis­sion­er Janet Wood­cock said in a state­ment while an­nounc­ing that next week’s VRB­PAC meet­ing would be de­layed.

Marks said the da­ta, some of which was “late-break­ing,” are com­ing in very rapid­ly be­cause of the large num­ber of cas­es of Omi­cron and “at this time it makes sense to wait for the third dose be­fore tak­ing ac­tion.”

Janet Wood­cock

“The da­ta we saw made us re­al­ize we need to see a third dose in or­der to pro­ceed with an au­tho­riza­tion,” he added.

The ex­ten­sion al­lows the FDA time to re­ceive up­dat­ed da­ta on the two and three-dose reg­i­men, Pfiz­er said, while con­duct­ing a thor­ough eval­u­a­tion of it.

The com­pa­ny had ini­tial­ly said in De­cem­ber that it would test a third dose of its Covid-19 vac­cine in the tri­al af­ter an­nounc­ing that non-in­fe­ri­or­i­ty was not met for chil­dren be­tween the ages of two and five when com­pared to old­er teenagers in the cur­rent tri­al. But for the youngest group, from 6 months to 2-years-old, non-in­fe­ri­or­i­ty was met.

But Marks said it was a “safe as­sump­tion” that the ef­fi­ca­cy de­ci­sion would be based on clin­i­cal da­ta from Pfiz­er.

Pfiz­er said in a state­ment that in these chil­dren, da­ta on the first two 3 µg dos­es in this age group are be­ing shared with the FDA on an on­go­ing ba­sis. Cas­es con­tin­ue to ac­cu­mu­late ac­cord­ing to the study pro­to­col and more da­ta are be­ing gen­er­at­ed be­cause rates of in­fec­tion and ill­ness re­main high in chil­dren of this age, es­pe­cial­ly due to the re­cent Omi­cron surge.

“The in­de­pen­dent Da­ta Mon­i­tor­ing Com­mit­tee (DMC) for the study sup­ports the con­tin­u­a­tion of the tri­al ac­cord­ing to the pro­to­col and be­lieve that the da­ta col­lect­ed to date in­di­cate the vac­cine is well tol­er­at­ed and sup­port a po­ten­tial three-dose reg­i­men,” Pfiz­er added.

Some pub­lic health ex­perts said they thought FDA was cor­rect in press­ing the pause but­ton.

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.

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.

J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

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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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.

Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

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