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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.

Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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David Light, Valisure CEO

Val­isure in the hot seat: New Form 483 over a 2021 in­spec­tion as CEO fires back

The notorious drug testing company Valisure, which has made a name for itself by forcing FDA’s hand with some of its safety-related uncoverings, received a letter this week after the FDA uncovered violations at its Connecticut-based testing lab in 2021.

The letter, which was sent on Dec. 5, stated that the FDA is “concerned” that Valisure was not aware of  drug supply chain security requirements.

WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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FDA re­view­ers head back to White Oak in 2023, with lead­er­ship look­ing to ap­pease a new Con­gress

Republicans have taken a stand against the pandemic era habit of lax work-from-home schedules. Now that they’ve wrestled control of the House majority, the FDA’s leadership is playing ball, sending many of the agency’s more than 18,000 employees back to their desks early next year.

Whether this exodus back to White Oak in Silver Spring, MD (many staff will still be allowed to work from home for multiple days per week) will mean more defections to industry and elsewhere remains to be seen.

Bags of shred­ded docs: In­di­an drug­mak­er Lupin hand­ed a Form 483 by FDA in­spec­tors

The generics manufacturer Lupin has been given another Form 483 from the FDA this year.

US regulators inspected Lupin’s pharmaceutical manufacturing site in the town of Mandideep, India from Nov. 14 through Nov. 23, with the 14-page report marking 16 observations.

The inspection report stated that the site did not have the appropriate controls over its computer systems to ensure that changes in “master production” or records are only done by authorized personnel, along with written procedures not being established to conduct annual reviews of records associated with drug batches.

Bro­ken promis­es? FDA needs more pow­er to re­move drugs from mar­ket­place, JA­MA analy­sis finds

The FDA is struggling to remove drugs from the marketplace that don’t show effectiveness in late stage trials, new JAMA analyses found, thanks to the persistent tension between speed and confidence in early clinical data.

Congress, regulated industry and patients have urged the FDA to shorten the amount of time that the market has to wait for drugs to become available that may help severe and prevalent diseases – and the FDA has listened, offering up a quick accelerated approval pathway that’s frequently used by new cancer drugs.