EMA, FDA and WHO prep for new coro­n­avirus treat­ments, IVDs

Fol­low­ing the World Health Or­ga­ni­za­tion’s (WHO) de­ter­mi­na­tion last week that the spread of the nov­el coro­n­avirus (2019-nCoV) from Wuhan, Chi­na is a pub­lic health emer­gency of in­ter­na­tion­al con­cern, the Eu­ro­pean Med­i­cines Agency (EMA), FDA and WHO have tak­en var­i­ous pre­cau­tions and pre­pared for new med­i­cines, vac­cines and in vit­ro di­ag­nos­tics (IVDs).

FDA on Tues­day is­sued an Emer­gency Use Au­tho­riza­tion (EUA) to au­tho­rize the emer­gency use of Cen­ters for Dis­ease Con­trol and Pre­ven­tion’s (CDC) 2019-Nov­el Coro­n­avirus (2019-nCoV) Re­al-Time Re­verse Tran­scrip­tase (RT)-PCR Di­ag­nos­tic Pan­el for the “pre­sump­tive qual­i­ta­tive de­tec­tion of nu­cle­ic acid from the 2019-nCoV in up­per and low­er res­pi­ra­to­ry spec­i­mens (such as na­sopha­ryn­geal or oropha­ryn­geal swabs, spu­tum, low­er res­pi­ra­to­ry tract as­pi­rates, bron­choalve­o­lar lavage, and na­sopha­ryn­geal wash/as­pi­rate or nasal as­pi­rate) col­lect­ed from in­di­vid­u­als who meet CDC cri­te­ria for 2019-nCoV test­ing.”

To date, this test has been lim­it­ed to use at CDC lab­o­ra­to­ries, but FDA said to­day’s au­tho­riza­tion al­lows the use of the test at any CDC-qual­i­fied lab na­tion­wide.

EMA al­so said Tues­day that it is ready to sup­port drug de­vel­op­ers with all avail­able reg­u­la­to­ry tools (i.e., sci­en­tif­ic ad­vice, the PRIME scheme, the ac­cel­er­at­ed as­sess­ment and con­di­tion­al mar­ket­ing au­tho­riza­tion) to ad­vance and ex­pe­dite the de­vel­op­ment of ways to fight and pre­vent the spread of this virus.

“De­vel­op­ers work­ing on med­i­c­i­nal prod­ucts or vac­cines that could be used for treat­ment or pre­ven­tion of nov­el coro­n­avirus 2019-nCoV in­fec­tions are en­cour­aged to con­tact the Agency and dis­cuss their strat­e­gy for ev­i­dence gen­er­a­tion as soon as pos­si­ble. They can con­tact EMA by send­ing an email to 2019-nCoV@ema.eu­ropa.eu for ini­tial dis­cus­sions with EU reg­u­la­tors,” EMA said.

WHO on Tues­day said that there are now more than 20,000 cas­es of 2019-nCoV and 425 deaths. The death toll rose to 490 by Tues­day evening, ac­cord­ing to the Chi­nese gov­ern­ment.

Al­most a dozen clin­i­cal tri­als are be­gin­ning to take shape in Chi­na, in­clud­ing one for a re­com­bi­nase aid­ed am­pli­fi­ca­tion (RAA) as­say at a Bei­jing hos­pi­tal, as well as one to test the ef­fi­ca­cy and safe­ty of the an­tivi­ral treat­ment ar­bidol in pa­tients with 2019-nCoV.

The US De­part­ment of Health and Hu­man Ser­vices (HHS), af­ter de­clar­ing a pub­lic health emer­gency last Fri­day, said Tues­day that it’s work­ing with Re­gen­eron Phar­ma­ceu­ti­cals to de­vel­op mul­ti­ple mon­o­clon­al an­ti­bod­ies that could be used to treat 2019-nCoV. Oth­er large bio­phar­ma com­pa­nies are al­so work­ing on treat­ments and vac­cines, in­clud­ing Gilead Sci­ences, Glax­o­SmithK­line and John­son & John­son.

And the US Bio­med­ical Ad­vanced Re­search and De­vel­op­ment Au­thor­i­ty (BAR­DA) is pro­vid­ing a por­tal for com­pa­nies and in­ter­est­ed stake­hold­ers to sub­mit mar­ket re­search pack­ages. FDA last week al­so out­lined the prod­uct de­vel­op­ment process for com­bat­ing this virus.

“Fed­er­al agen­cies are par­tic­u­lar­ly in­ter­est­ed in iden­ti­fy­ing prod­ucts and tech­nolo­gies that have pro­gressed in­to or be­yond non-clin­i­cal stud­ies, have es­tab­lished large-scale com­mer­cial Good Man­u­fac­tur­ing Prac­tices (cGMP) man­u­fac­tur­ing ca­pa­bil­i­ty, or uti­lize a plat­form al­ready ap­proved by the U.S. Food and Drug Ad­min­is­tra­tion (FDA),” HHS said.

Mean­while, FDA, which has can­celed all staff trav­el to Chi­na, on Tues­day said it is aware that some blood es­tab­lish­ments are con­sid­er­ing donor ed­u­ca­tion and/or donor de­fer­ral mea­sures in re­sponse to 2019-nCoV. How­ev­er, FDA said that res­pi­ra­to­ry virus­es, in gen­er­al, are not known to be trans­mit­ted by blood trans­fu­sion and there have not been any re­port­ed cas­es of trans­fu­sion-trans­mit­ted coro­n­avirus.

“Rou­tine blood donor screen­ing mea­sures that are al­ready in place should pre­vent in­di­vid­u­als with clin­i­cal res­pi­ra­to­ry in­fec­tions from do­nat­ing blood,” FDA says.

WHO al­so pub­lished its Emer­gency Use List­ing (EUL) pro­ce­dure to stream­line the process by which new or un­li­censed prod­ucts can be used dur­ing pub­lic health emer­gen­cies. The EUL re­places the Emer­gency Use As­sess­ment and List­ing (EU­AL) pro­ce­dure, which was used dur­ing the West Africa Ebo­la out­break of 2014-2016.

Ed­i­tor’s note: Ar­ti­cle up­date on 2/4/20 with more in­for­ma­tion about the EUA and the death toll in Chi­na.

RAPS: First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion.

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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New stan­dard of care? FDA hands Pfiz­er, Mer­ck KGaA an OK for Baven­cio in blad­der can­cer

The breakthrough therapy designation Pfizer and Merck KGaA notched for Bavencio in bladder cancer has quickly paved way for a full approval.

The PD-L1 drug is now sanctioned as a first-line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma, applicable in cases where cancer hasn’t progressed after platinum-containing chemotherapy.

Petros Grivas, the principal investigator of the supporting Phase III JAVELIN Bladder 100, called the approval “one of the most significant advances in the treatment paradigm in this setting in 30 years.”

Covid-19 roundup: Vac­cines will need to beat place­bo by 50% to qual­i­fy for FDA OK; UK tri­al drops Kale­tra

The FDA will set the bar for approving a Covid-19 vaccine at 50% efficacy, the Wall Street Journal reported, meaning any successful candidate will have to reduce the risk of coronavirus disease by at least half compared to placebo.

That requirement is part of guidance that the agency is set to release later today, laying out detailed criteria for vaccine developers — some of whom are eyeing an OK by the end of the year, in line with expectations at Operation Warp Speed.

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Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Pos­i­tive Covid-19 vac­cine da­ta? New mouse study? OWS in­clu­sion? Yep, but some­how, the usu­al tid­bits from In­ovio back­fire

You don’t go more than 40 years in biotech without ever getting a product to market unless you can learn the art of writing a promotional press release. And Inovio captures the prize in baiting the hook.

Tuesday morning Inovio, which has been struggling to get its Covid-19 vaccine lined up for mass manufacturing, put out a release that touched on virtually every hot button in pandemic PR.

There was, first and foremost, an interim snapshot of efficacy from their Phase I program for INO-4800.