Christophe Weber, Takeda CEO (Miho Takahashi/The Yomiuri Shimbun via AP Images)

FDA of­fers sup­port for Takeda's po­ten­tial cy­tomegalovirus drug ahead of ad­comm

Af­ter al­most two decades of dis­cus­sions with FDA re­gard­ing the de­vel­op­ment of Take­da’s po­ten­tial drug for post-trans­plant cy­tomegalovirus (CMV), which it bought in its ac­qui­si­tion of Shire in 2019, the agency seems to be of­fer­ing pos­i­tive marks for the drug’s safe­ty and ef­fi­ca­cy pro­file ahead of Thurs­day’s ad­comm.

Ac­cord­ing to FDA’s brief­ing doc­u­ments re­leased ahead of the meet­ing, marib­avir proved safe across mul­ti­ple stud­ies and sta­tis­ti­cal­ly su­pe­ri­or to in­ves­ti­ga­tor-as­signed treat­ment (IAT) in a Phase III tri­al, which Take­da an­nounced ear­li­er this year.

FDA’s con­cerns about dis­con­tin­u­a­tions in the IAT arm of the tri­al al­so did not over­shad­ow that sta­tis­ti­cal suc­cess.

While FDA not­ed the Phase III re­sults “ap­peared to be dri­ven by treat­ment dis­con­tin­u­a­tion in the IAT group (dis­con­tin­u­a­tion was con­sid­ered treat­ment fail­ure in the pri­ma­ry analy­sis),” sev­er­al sen­si­tiv­i­ty analy­ses of the pri­ma­ry end­point “showed that marib­avir re­mained sta­tis­ti­cal­ly su­pe­ri­or to IAT for the pri­ma­ry end­point, and thus the treat­ment ef­fect re­mains ro­bust.”

In ad­di­tion­al im­por­tant sub­group analy­ses, FDA said marib­avir re­mained su­pe­ri­or to IAT for the pri­ma­ry end­point with the ex­cep­tion of the “re­frac­to­ry” sub­group and for the sub­group of pa­tients with CMV dis­ease.

But FDA not­ed that one of its “ma­jor con­cerns” in the tri­al “was the po­ten­tial for bias due to the open-la­bel de­sign of the tri­al. Al­though bias is sug­gest­ed by the demon­stra­tion of more dis­con­tin­u­a­tions in the IAT arm than in the marib­avir arm, bias can­not be de­fin­i­tive­ly demon­strat­ed or ruled out, par­tic­u­lar­ly be­cause of the known safe­ty pro­file of the drugs in the IAT arm.”

And over­all, the agency said marib­avir “pro­vides some ad­van­tages over cur­rent­ly avail­able ther­a­pies, i.e., it ap­pears to have a bet­ter safe­ty pro­file, with taste dis­tur­bance be­ing the most com­mon ad­verse re­ac­tion as­so­ci­at­ed with its use. In these tri­als, most sub­jects did not dis­con­tin­ue marib­avir due to taste dis­tur­bance or oth­er ad­verse re­ac­tions. Ad­di­tion­al­ly, marib­avir is avail­able as an oral tablet (as is val­gan­ci­clovir), pro­vid­ing ease of ad­min­is­tra­tion.”

Ad­comm mem­bers will vote on two ques­tions on whether the over­all ben­e­fit-risk as­sess­ment is fa­vor­able for marib­avir to treat trans­plant re­cip­i­ents with CMV in­fec­tion and dis­ease, with one ques­tion ask­ing if that risk-ben­e­fit pro­file is still fa­vor­able with or with­out those with geno­typ­ic re­sis­tance to oth­er an­tivi­rals, in­clud­ing gan­ci­clovir, val­gan­ci­clovir, fos­car­net or cid­o­fovir.

If ap­proved, Take­da’s marib­avir could be the first drug specif­i­cal­ly ap­proved for treat­ment of post-trans­plant CMV in­fec­tion, which FDA says is one of the most com­mon in­fec­tious com­pli­ca­tions in post-trans­plant re­cip­i­ents. FDA al­so not­ed that the avail­able CMV an­tivi­ral drugs, in­clud­ing gan­ci­clovir, val­gan­ci­clovir, fos­car­net and cid­o­fovir, all have sig­nif­i­cant tox­i­c­i­ties.

An ap­proval would al­so cap a long and rocky his­to­ry for the drug, which has changed hands sev­er­al times over the last two decades through pos­i­tive and neg­a­tive tri­als.

Glax­o­SmithK­line first syn­the­sized the drug 20 years ago and did some ear­ly clin­i­cal work, be­fore li­cens­ing it to the rare dis­ease and in­fec­tious dis­ease-fo­cused biotech Vi­roPhar­ma.

Vi­roPhar­ma then took it through Phase II, then missed the pri­ma­ry end­point in a Phase III study in 2009. Marib­avir failed to pre­vent CMV in­fec­tions bet­ter than place­bo in pa­tients re­ceiv­ing bone mar­row trans­plants. Vi­roPhar­ma sug­gest­ed a high­er dose might lead to a bet­ter re­sponse, but end­ed up pass­ing the can­di­date to Shire dur­ing a $4.2 bil­lion buy­out in 2013.

Shire ran with Vi­roPhar­ma’s idea, in­creas­ing the dose in var­i­ous phar­ma­co­ki­net­ic and ef­fi­ca­cy stud­ies, and in 2016, a Phase II study showed that it helped clear in­fec­tions. The FDA com­mend­ed the re­sults with a break­through des­ig­na­tion in 2018. And the fol­low­ing year, Take­da com­plet­ed its ac­qui­si­tion of Shire for $62 bil­lion.

Mar­ket­ingRx roundup: Pfiz­er de­buts Pre­vnar 20 TV ads; Lil­ly gets first FDA 2022 pro­mo slap down let­ter

Pfizer debuted its first TV ad for its Prevnar 20 next-generation pneumococcal pneumonia vaccine. In the 60-second spot, several people (actor portrayals) with their ages listed as 65 or older are shown walking into a clinic as they turn to say they’re getting vaccinated with Prevnar 20 because they’re at risk.

The update to Pfizer’s blockbuster Prevnar 13 vaccine was approved in June, and as its name suggests is a vaccine for 20 serotypes — the original 13 plus seven more that cause pneumococcal disease. Pfizer used to spend heavily on TV ads to promote Prevnar 13 in 2018 and 2019 but cut back its TV budgets in the past two fall and winter seasonal spending cycles. Prevnar had been Pfizer’s top-selling drug, notching sales of just under $6 billion in 2020, and was the world’s top-selling vaccine before the Covid-19 vaccines came to market last year.

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Albert Bourla (Photo by Steven Ferdman/Getty Images)

UP­DAT­ED: Pfiz­er fields a CRL for a $295M rare dis­ease play, giv­ing ri­val a big head start

Pfizer won’t be adding a new rare disease drug to the franchise club — for now, anyway.

The pharma giant put out word that their FDA application for the growth hormone therapy somatrogon got the regulatory heave-ho, though they didn’t even hint at a reason for the CRL. Following standard operating procedure, Pfizer said in a terse missive that they would be working with regulators on a followup.

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Graphic: Alexander Lefterov for Endpoints News

Small biotechs with big drug am­bi­tions threat­en to up­end the tra­di­tion­al drug launch play­book

Of the countless decisions Vlad Coric had to make as Biohaven’s CEO over the past seven years, there was one that felt particularly nerve-wracking: Instead of selling to a Big Pharma, the company decided it would commercialize its migraine drug itself.

“I remember some investors yelling and pounding on the table like, you can’t do this. What are you thinking? You’re going to get crushed by AbbVie,” he recalled.

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A new can­cer im­munother­a­py brings cau­tious hope for a field long await­ing the next big break­through

Bob Seibert sat silent across from his daughter at their favorite Spanish restaurant near his home in Charleston County, SC, their paella growing cold as he read through all the places in his body doctors found tumors.

He had texted his wife, a pediatric intensive care nurse, when he got the alert that his online chart was ready. Although he saw immediately it was bad, many of the terms — peritoneal, right iliac — were inscrutable. But she was five hours downstate, at a loud group dinner the night before another daughter’s cheer competition.

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Not cheap­er by the dozen: Bris­tol My­ers be­comes the 12th phar­ma com­pa­ny to re­strict 340B sales

Bristol Myers Squibb recently joined 11 of its peer pharma companies in limiting how many contract pharmacies can access certain drugs discounted by a federal program known as 340B.

Bristol Myers is just the latest in a series of high-profile pharma companies moving in their own direction as the Biden administration’s Health Resources and Services Administration struggles to rein in the drug discount program for the neediest Americans.

Joaquin Duato, J&J CEO (Photo by Charles Sykes/Invision/AP)

New J&J CEO Joaquin Du­a­to promis­es an ag­gres­sive M&A hunt in quest to grow phar­ma sales

Joaquin Duato stepped away from the sideline and directly into the spotlight on Tuesday, delivering his first quarterly review for J&J as its newly-tapped CEO after an 11-year run in senior posts. And he had some mixed financial news to deliver today while laying claim to a string of blockbuster drugs in the making and outlining an appetite for small and medium-sized M&A deals.

Duato also didn’t exactly shun large buyouts when asked about the future of the company’s medtech business — where they look to be in either the top or number 2 position in every segment they’re in — even though the bar for getting those deals done is so much higher.

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Amgen's Twitter campaign #DearAsthma inspired thousands of people to express struggles and frustrations with the disease

Am­gen’s #Dear­Asth­ma spon­sored tweet lands big on game day, spark­ing thou­sands to re­spond

Amgen wanted to know how people with asthma really felt about daily life with the disease. So it bought a promoted tweet on Twitter noting the not-so-simple realities of life with asthma and ended the post with a #DearAsthma hashtag, a megaphone emoji and a re-tweet button.

That was just over one week ago and the responses haven’t stopped. More than 7,000 posts so far on Twitter replied to #DearAsthma to detail struggles of daily life, expressing humor, frustration and sometimes anger. More than a few f-bombs have been typed or gif-ed in reply to communicate just how much many people “hate” the disease.

Pfiz­er, Bris­tol My­ers dom­i­nate top 10 pre­dic­tions for the best-sell­ing drugs of 2022

The annual exercise where analysts try and predict which drugs will become blockbusters and make the most money tends to highlight the biggest trends in biopharma R&D. 2022 is no exception.

The team at Evaluate Vantage published its predictions for the top 10 selling drugs for the year — expecting tens of billions of dollars in sales and highlighting an industry-wide focus on certain diseases and indications.

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UP­DAT­ED: FDA slams Eli Lil­ly's 'mis­lead­ing' In­sta­gram ad for its type 2 di­a­betes in­jec­tion

In a first for 2022, the FDA’s Office of Prescription Drug Promotion has issued an untitled letter, which was recently sent to Eli Lilly over what the agency calls a “misleading” and “particularly concerning” Instagram ad the company posted for its type 2 diabetes drug Trulicity.

The questionable Instagram post, which has since been deleted by Lilly, failed to adequately communicate the indication and limitations of use associated with Trulicity, FDA says.