Opin­ion: The FDA's mR­NA vac­cine full ap­proval re­views are mov­ing plen­ty fast. Start the man­dates now

As the US con­tin­ues to en­joy a sur­plus of pro­tec­tive Covid vac­cines, some ex­perts are hop­ing that a tran­si­tion from EUA to full ap­proval sta­tus for the two mR­NA vac­cines will boost the pub­lic’s trust in them and in turn, amp up the coun­try’s vac­ci­na­tion lev­els just in time to block the resur­gence of Covid that we’re see­ing else­where dri­ven by the Delta vari­ant.

While such a quick up-shift may have that im­me­di­ate ef­fect, let’s not lose sight of the fact that the FDA is like­ly to re­view the Pfiz­er/BioN­Tech and Mod­er­na vac­cines in record time. And their mas­sive trove of re­al-world safe­ty and man­u­fac­tur­ing da­ta don’t make that easy. Even still, the FDA is plan­ning its Covid vac­cine ap­proval re­views to last about half the time (3-4 months) that it cur­rent­ly takes the agency to sign off on a pri­or­i­ty drug/vac­cine ap­pli­ca­tion (6 months), and just a frac­tion of the time for a non-ex­pe­dit­ed new drug ap­pli­ca­tion (12 months).

There’s no ques­tion the agency should pick up the speed for both Pfiz­er and Mod­er­na’s ap­pli­ca­tions. Em­ploy­ers may be more will­ing to push for man­dates once the vac­cines are ful­ly ap­proved. Let’s not for­get the rea­son we’re in this en­vi­able po­si­tion, with al­most 160 mil­lion peo­ple ful­ly vac­ci­nat­ed in the US in just a lit­tle more than a year and a half since the pan­dem­ic be­gan: The FDA was able to move quick­ly on the stel­lar ef­fi­ca­cy da­ta at hand from both vac­cines, and en­sure the man­u­fac­tur­ing process was ro­bust enough and could be repli­cat­ed in a way to make all of those hun­dreds of mil­lions of dos­es safe­ly and ef­fi­cient­ly.

A rub­ber stamp push from EUA to full ap­proval doesn’t help the mil­lions of Amer­i­cans who still haven’t been vac­ci­nat­ed, may be hes­i­tant, or may be en­tire­ly op­posed to the idea just be­cause of the EUA sta­tus. And as the EUA to full ap­proval tran­si­tion is com­ing, em­ploy­ers across the coun­try need to stand strong and re­quire vac­ci­na­tions so we can re­main pro­tect­ed. As lawyers have in­di­cat­ed, there’s noth­ing il­le­gal about re­quir­ing the ad­min­is­tra­tion of vac­cines un­der EUAs.

Er­ic Topol, pro­fes­sor of mol­e­c­u­lar med­i­cine at Scripps Re­search, re­cent­ly au­thored an op-ed in the New York Times, call­ing on the FDA to ful­ly ap­prove the mR­NA vac­cines now, not­ing that they “have over­whelm­ing­ly been proved safe and ef­fec­tive by clin­i­cal tri­als, in­de­pen­dent re­search and the ex­pe­ri­ence of mil­lions of peo­ple around the world who re­ceived them.”

But FDA’s re­views of Pfiz­er’s and Mod­er­na’s vac­cines have so far last­ed less than 3 months. And there is noth­ing in the EUA or re­al-world da­ta for ei­ther the Mod­er­na or the Pfiz­er/BioN­Tech vac­cine that should bring pause or some­how sug­gest ei­ther vac­cine’s clin­i­cal da­ta pack­age isn’t equal to oth­er, man­dat­ed vac­cines that have al­ready won full ap­proval.

And while some seem to sug­gest the FDA is drag­ging its feet, the size (thou­sands of pages long) and com­plex­i­ty of a full ap­proval pack­age should not be tak­en light­ly. If the FDA needs to take longer to en­sure the vac­cines are man­u­fac­tured prop­er­ly enough to win a full ap­proval, so be it.

Pe­ter Marks

As FDA’s top vac­cine of­fi­cial Pe­ter Marks made clear from the very be­gin­ning, the vac­cines were cleared at a high­er bar than the oth­er treat­ments win­ning EUAs, and the Amer­i­can pub­lic should be just as con­fi­dent in the vac­cines un­der EUAs as un­der full ap­provals.

For those who re­main un­vac­ci­nat­ed, Marks and Janet Wood­cock re­it­er­at­ed the ur­gent need to move now, even with the very rare my­ocardi­tis risk in most­ly younger men, as part of a stake­hold­er call last week. The CDC al­so made clear on Tues­day evening that more than half of all cas­es in the US are now the re­sult of the Delta vari­ant, which orig­i­nat­ed in In­dia.

“The Delta vari­ant is ris­ing in this coun­try and its in­ci­dence may dou­ble every week or so, and ex­perts ex­pect it will be the dom­i­nant vari­ant with­in a month or so,” Wood­cock said. The time to get vac­ci­nat­ed is now, with or with­out a full ap­proval.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Graphic: Kathy Wong for Endpoints News

What kind of biotech start­up wins a $3B syn­di­cate, woos a gallery of mar­quee sci­en­tists and re­cruits GSK's Hal Bar­ron as CEO in a stun­ner? Let Rick Klaus­ner ex­plain

It started with a question about a lifetime’s dream on a walk with tech investor Yuri Milner.

At the beginning of the great pandemic, former NCI chief and inveterate biotech entrepreneur Rick Klausner and the Facebook billionaire would traipse Los Altos Hills in Silicon Valley Saturday mornings and talk about ideas.

Milner’s question on one of those mornings on foot: “What do you want to do?”

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FDA+ roundup: FDA's neu­ro­science deputy de­parts amid on­go­ing Aduhelm in­ves­ti­ga­tions; Califf on the ropes?

Amid increased scrutiny into the close ties between FDA and Biogen prior to the controversial accelerated approval of Aduhelm, the deputy director of the FDA’s office of neuroscience has called it quits after more than two decades at the agency.

Eric Bastings will now take over as VP of development strategy at Ionis Pharmaceuticals, the company said Wednesday, where he will provide senior clinical and regulatory leadership in support of Ionis’ pipeline.

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CBO: Medicare ne­go­ti­a­tions will ham­per drug de­vel­op­ment more than pre­vi­ous­ly thought

As President Biden’s Build Back Better Act — and, with it, potentially the Democrats’ last shot at major drug pricing reforms in the foreseeable future — remains on life support, the Congressional Budget Office isn’t helping their case.

The CBO last week released a new slide deck, outlining an update to its model on how Medicare negotiations might take a bite out of new drugs making it to market. The new model estimates a 10% long-term reduction in the number of new drugs, whereas a previous CBO report from August estimated that 8% fewer new drugs will enter the market over 30 years.

Sec­ondary patents prove to be key in biosim­i­lar block­ing strate­gies, re­searchers find

While the US biosimilars industry has generally been a disappointment since its inception, with FDA approving 33 biosimilars since 2015, just a fraction of those have immediately followed their approvals with launches. And more than a handful of biosimilars for two of the biggest blockbusters of all time — AbbVie’s Humira and Amgen’s Enbrel — remain approved by FDA but still have not launched because of legal settlements.

Hal Barron (GSK via YouTube)

GSK R&D chief Hal Bar­ron jumps ship to run a $3B biotech start­up, Tony Wood tapped to re­place him

In a stunning switch, GlaxoSmithKline put out word early Wednesday that R&D chief Hal Barron is exiting the company after 4 years — a relatively brief run for the man chosen by CEO Emma Walmsley in late 2017 to turn around the slow-footed pharma giant.

Barron is being replaced by Tony Wood, a close associate of Barron’s who’s taking one of the top jobs in Big Pharma R&D. He’ll be closer to home, though, for GSK. Barron has been running a UK and Philadelphia-based research organization from his perch in San Francisco.

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Chamath Palihapitiya and Pablo Legorreta

Bil­lion­aires Chamath Pal­i­hapi­tiya and Pablo Legor­re­ta hatch an $825M SPAC for cell ther­a­py biotech

Three years after Royalty Pharma chief Pablo Legorreta led a group of investors to buy up a pair of biotechs and create a new startup called ProKidney, the biotech is jumping straight into an $825 million public shell created by SPAC king and tech billionaire Chamath Palihapitiya.

ProKidney was founded 6 years ago but really got going at the beginning of 2019 with the $62 million acquisition of inRegen, which was working on an autologous — from the patient — cell therapy for kidney disease. After extracting kidney cells from patients, researchers expand the cells in the lab and then inject them back into patients, aiming to restore the kidneys of patients suffering from CKD.

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Joshua Brumm, Dyne Therapeutics CEO

FDA or­ders DMD tri­al halt, rais­ing ques­tions about a whole class of promis­ing drugs

Dyne Therapeutics’ stock took a nasty hit this morning after the biotech put out word that the FDA had slapped a clinical hold on their top program for Duchenne muscular dystrophy. And now speculation is bouncing around Biotwitter that there could be a class effect at work here that would implicate other drug developers in the freeze.

Dyne execs didn’t have a whole lot to say about why the FDA sidelined their IND for DYNE-251 in DMD while “requesting additional clinical and non-clinical information for” the drug.

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With few re­main­ing un­ap­proved drugs, re­searchers tell FDA its Un­ap­proved Drugs Ini­tia­tive is no longer ef­fec­tive

Back in May, the FDA decided to reverse a Trump-era decision to pull the agency’s controversial Unapproved Drugs Initiative, with the FDA citing “multiple legal and factual inaccuracies.”

But now a group of researchers from Harvard, funded by Arnold Ventures, raises new questions about why the UDI program might not be necessary in its current form.

Designed in 2006 as a way for the FDA to transition old drugs from unapproved to approved products, the agency sought to better ensure that unsafe products were removed from the market.

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