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

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.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

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Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Lu­pus drug de­vel­op­ment mar­ket heat­ing up, while FDA links with ad­vo­ca­cy group to fur­ther ac­cel­er­ate re­search

The long-underserved systemic lupus erythematosus (SLE) market is suddenly buzzing with treatment possibilities. Less than two years after AstraZeneca’s approval for Saphnelo — the first new SLE drug in a decade and joining just one other approved in GSK’s Benlysta – the pipeline of potential drugs numbers in the dozens.

Although most are very early stage — Spherix Global Insights estimates five in Phase II/III — the pharma R&D enthusiasm is catching on among doctors, patients and advocacy groups. On Wednesday, the Lupus Research Alliance and the FDA formed a novel private-public partnership called Lupus Accelerating Breakthroughs Consortium (Lupus ABC) to help advance lupus clinical trial success.

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Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

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Nicklas Westerholm, Egetis Therapeutics CEO

Ac­qui­si­tion talks on­go­ing for Swedish rare dis­ease biotech Egetis, shares up al­most 40%

Shares of the Sweden-based rare disease biotech Egetis Therapeutics skyrocketed on Thursday afternoon as the company said it’s engaged in “ongoing discussion” with external parties regarding a “potential acquisition.”

Egetis confirmed rumors with a statement on Thursday while noting that there is no certainty that a takeover offer will be made.

Nonetheless, the possibility of an acquisition has shot up Egetis’ share price. By the afternoon on Thursday, its stock price was {$EGTX.ST} up over 38%. An Egetis spokesperson told Endpoints News in an email that it has no further comments.

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FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

Ivana Magovčević-Liebisch, Vigil Neuroscience CEO

FDA lifts par­tial clin­i­cal hold on Vig­il Neu­ro­science's TREM2 an­ti­body, re­mov­ing dos­ing cap

When Vigil Neuroscience filed its IPO papers in late 2021, the biotech revealed that the FDA had just cleared its Phase I trial — but with a partial clinical hold that limited dosing to under a certain level.

More than a year later, the FDA has lifted the hold.

Vigil is now free to dose VGL101, an antibody targeting TREM2, at levels higher than 20 mg/kg in its ongoing and future clinical trials in patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), an inherited condition that affects the brain and spinal cord.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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