Robert Califf, FDA commissioner (Tom Williams/CQ Roll Call via AP Images)

Hop­ing to ex­pand mon­key­pox vac­cine sup­ply, US paves the way for new route of ad­min­is­tra­tion

Af­ter mak­ing it clear that the US’ cur­rent mon­key­pox vac­cine sup­ply is in­suf­fi­cient, the FDA on Tues­day au­tho­rized a new route of ad­min­is­tra­tion that should in­crease the num­ber of avail­able dos­es by five-fold.

Daniel Ku­ritzkes

Reg­u­la­tors cleared Bavar­i­an Nordic’s Jyn­neos vac­cine for in­tra­der­mal in­jec­tion in adults old­er than 18. Un­like sub­cu­ta­neous in­jec­tion — the cur­rent method by which vac­cine is de­liv­ered un­der the skin — an in­tra­der­mal jab goes di­rect­ly in­to the skin. It’s be­lieved that this method re­quires less vac­cine, since the der­mis is rich in den­drit­ic cells which spe­cial­ize in tak­ing up for­eign anti­gens and pre­sent­ing them to the im­mune sys­tem, ac­cord­ing to Daniel Ku­ritzkes, chief of in­fec­tious dis­eases at Brigham and Women’s Hos­pi­tal in Boston.

“In re­cent weeks the mon­key­pox virus has con­tin­ued to spread at a rate that has made it clear our cur­rent vac­cine sup­ply will not meet the cur­rent de­mand,” FDA com­mis­sion­er Robert Califf said in a news re­lease.

The news comes as the New York Times re­ports that about 20 mil­lion dos­es of the vac­cine have ex­pired in the US na­tion­al stock­pile.

The down­side to in­tra­der­mal in­jec­tion? It’s more dif­fi­cult to per­form cor­rect­ly, William Schaffn­er, a pro­fes­sor in Van­der­bilt Uni­ver­si­ty’s in­fec­tious dis­eases di­vi­sion, told End­points News on Tues­day.

William Schaffn­er

As Schaffn­er put it, in­tra­der­mal in­jec­tion is “a bit of an art form.” It was most com­mon­ly used for tu­ber­cu­lo­sis skin tests; how­ev­er, he not­ed that it’s like­ly the av­er­age nurse now may have nev­er giv­en an in­tra­der­mal in­oc­u­la­tion, or on­ly had brief ex­po­sure. It’s much eas­i­er to stick a nee­dle through the skin for a sub­cu­ta­neous in­jec­tion.

“That’s easy. I can show you how to do that in five min­utes,” Schaffn­er said.

But get­ting it in­to the skin cor­rect­ly takes tech­nique. If it isn’t done prop­er­ly, vac­cine can ei­ther leak out or make its way through the skin, where it may be less ef­fec­tive giv­en the low­er dose, he said.

“The rea­son this isn’t em­ployed more is that it’s not easy,” he added.

The White House Na­tion­al Mon­key­pox Out­break Re­sponse team will over­see the roll­out of this new strat­e­gy and co­or­di­nate with the CDC, FDA, HHS and lo­cal of­fi­cials, ac­cord­ing to a fact sheet put out on Tues­day. Providers cur­rent­ly have all the sup­plies they need, ac­cord­ing to the White House, and the Biden ad­min­is­tra­tion added that it’s launch­ing a “ro­bust ef­fort to train health care work­ers and providers.”

The 400,000 vials of Jyn­neos vac­cine in the na­tion­al stock­pile should ex­pand to 2 mil­lion dos­es us­ing in­tra­der­mal in­jec­tion.

In ad­di­tion, the FDA au­tho­rized the Jyn­neos vac­cine for in­di­vid­u­als un­der 18 years old who are at high-risk for mon­key­pox in­fec­tion, though ado­les­cents must re­ceive sub­cu­ta­neous in­jec­tion.

To date, HHS has made 1.1 mil­lion dos­es of the Jyn­neos vac­cine avail­able for or­der and has shipped more than 620,000 dos­es. Plus, the Ad­min­is­tra­tion for Strate­gic Pre­pared­ness and Re­sponse an­nounced that it will pro­cure an ad­di­tion­al 5.5 mil­lion vials, which trans­lates to more than 25 mil­lion dos­es via in­tra­der­mal in­jec­tion.

Last week, of­fi­cials said an ad­di­tion­al 150,000 vials will ar­rive two months ahead of sched­ule in Sep­tem­ber.

While Jyn­neos re­mains the on­ly vac­cine in the US specif­i­cal­ly ap­proved for mon­key­pox, HHS has al­so cleared states and ju­ris­dic­tions to or­der the small­pox vac­cine ACAM2000. How­ev­er, the agency pre­vi­ous­ly not­ed that ACAM2000 isn’t rec­om­mend­ed for every­one “due to sig­nif­i­cant side ef­fects.”

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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FDA's out­side ex­perts vote in fa­vor of Fer­ring's fe­cal trans­plant for C. dif­fi­cile, set­ting the stage for Seres

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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Richard Pazdur, FDA's OCE director (Flatiron Health via YouTube)

FDA's OCE makes the case for ac­cel­er­at­ed ap­proval rid­er in user fee reau­tho­riza­tion

Four experts from the FDA’s Oncology Center of Excellence took to the New England Journal of Medicine yesterday to make the case for not only improving the agency’s ability to expeditiously pull dangling accelerated approvals when, on the rare occasion, confirmatory trials fail, but also better building “quality and efficiency into the AA on-ramp.”

The timely perspective arrives as Congress has exactly one week left to draft, release and sign off on the reauthorized user fee deals before layoff notices will be sent to drug reviewers. That package, which is likely to hitch a ride with the continuing resolution, may or may not include several policy riders (opposed by Republicans), including one that would allow the FDA to require confirmatory trials to be underway before an AA is granted, and would improve the process by which FDA can withdraw AAs.

An­oth­er Cipla site lands a Form 483 over clean­ing is­sues and QC con­trols

A Cipla drug manufacturing site in India has once again landed in the crosshairs of FDA inspectors.

The facility in question is Cipla’s drug manufacturing facility in the village of Verna, in the state of Goa in India’s southwest. In a sign that foreign inspections might ramp up again, the FDA’s visit from Aug. 16 to Aug. 22 uncovered six observations.

The 11-page report noted that environmental monitoring at the site did not properly ensure that microbial contaminants were not making any impact in the aseptic filling areas. It also found that procedures meant to stop microbial contamination were not adequately conducted in aseptic areas of the facility.

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FDA ad­comm takes down Se­cu­ra Bio's leukemia drug af­ter fi­nal tri­al re­sults show po­ten­tial OS detri­ment

The FDA’s Oncologic Drugs Advisory Committee on Friday voted 8-4 against the benefit-risk profile of Secura Bio’s PI3K inhibitor Copiktra (duvelisib), which won approval in September 2018 as a third-line treatment for relapsed or refractory CLL or SLL, but updated pivotal trial results raised safety questions.

In addition to the serious and fatal toxicities of duvelisib, FDA speakers at the ODAC meeting pointed to an evolved treatment landscape for CLL and SLL, with targeted BTK or BCL2 inhibitors (front-line or second-line), and data pointing to a “potential detriment” in overall survival for duvelisib. But some ODAC members noted that the detriment was likely small and that there is some efficacy even as the data are difficult to interpret.

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Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.