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

FDA's an­timi­cro­bial ad­comm unan­i­mous­ly backs Takeda's drug for post-trans­plant cy­tomegalovirus

The FDA’s an­timi­cro­bial drugs ad­vi­so­ry com­mit­tee on Thurs­day vot­ed unan­i­mous­ly in fa­vor of FDA ap­prov­ing Take­da’s an­tivi­ral for post-trans­plant cy­tomegalovirus.

The ad­comm vot­ed 17-0 on the ques­tion of whether the over­all ben­e­fit-risk pro­file is fa­vor­able for the use of Take­da’s marib­avir for the treat­ment of trans­plant re­cip­i­ents with CMV in­fec­tion and dis­ease re­frac­to­ry to treat­ment and with geno­typ­ic re­sis­tance to four oth­er an­tivi­rals — gan­ci­clovir, val­gan­ci­clovir, fos­car­net or cid­o­fovir.

On whether the drug, which took about two decades to de­vel­op, should be ap­proved for the same pop­u­la­tion but those with­out geno­typ­ic re­sis­tance to the four an­tivi­rals, the com­mit­tee vot­ed 17-0.

FDA’s brief­ing doc­u­ments re­leased ahead of the meet­ing showed how 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.

While sev­er­al mem­bers of the ad­comm said dur­ing the dis­cus­sion that they don’t want to let per­fect be the en­e­my of the good in terms of the da­ta around marib­avir, sev­er­al oth­ers ques­tioned the end­point used in that tri­al, and why the tri­al didn’t in­clude a more di­verse pop­u­la­tion.

Ad­comm chair Lind­sey Baden of the Dana-Far­ber Can­cer In­sti­tute, who vot­ed in fa­vor of the ap­proval, al­so called the da­ta “messy,” but said the to­tal­i­ty of da­ta is per­sua­sive. He al­so not­ed the ad­van­tages of an oral drug in this in­di­ca­tion.

Com­mit­tee mem­ber Nan­cy Bridges, chief of the trans­plan­ta­tion branch at the NIH’s Na­tion­al In­sti­tute of Al­ler­gy and In­fec­tious Dis­eases, who vot­ed in fa­vor of ap­proval al­so said that over­all, “the da­ta are very per­sua­sive.”

But she not­ed that the par­tic­i­pants in the Phase III tri­al “were over­whelm­ing­ly white,” even though this con­di­tion has a high­er preva­lence among African Amer­i­cans. “I’d like to see some da­ta from the com­pa­ny on ef­fi­ca­cy in African Amer­i­cans, which we don’t have,” Bridges said.

George Siber­ry, a med­ical of­fi­cer at the US Agency for In­ter­na­tion­al De­vel­op­ment, al­so said it was dis­ap­point­ing that no ado­les­cents were in­clud­ed in the piv­otal tri­al.

Pe­ter Weina, di­rec­tor of the De­fense Health Agency, said that even if the drug is ap­proved for a nar­row pop­u­la­tion, it’ll be used for all dif­fer­ent in­di­ca­tions off-la­bel.

“I’m torn that the dif­fer­ence be­tween geno­typ­ic re­sis­tance and no geno­typ­ic re­sis­tance is tru­ly a moot point here,” Weina said.

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

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

Vi­roPhar­ma then missed a 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 as part of a $4.2 bil­lion buy­out in 2013.

Shire upped the dose in var­i­ous phar­ma­co­ki­net­ic and ef­fi­ca­cy stud­ies, and in 2016, a Phase II study showed that the drug 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.

Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; 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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Post-hoc analy­sis: EMA's CHMP re­jects Ipsen's po­ten­tial drug for rare ge­net­ic dis­ease

The European Medicines Agency’s Committee for Medicinal Products for Human Use on Friday rejected Ipsen Pharma’s potential treatment for a rare genetic disease known as fibrodysplasia ossificans progressiva (FOP), which causes extra bone to form outside the skeleton.

The EMA said on its website that it could not draw any firm conclusions on the benefits of the French biopharma’s Sohonos (palovarotene), which selectively targets the retinoic-acid receptor gamma (RARγ), “as the applicant’s conclusion was based on a post-hoc analysis which was neither scientifically nor clinically justified and pre-specified study objectives were not met.”

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FDA ap­proves an­oth­er in­di­ca­tion for Keytru­da, this time in the ad­ju­vant NSCLC set­ting

Merck’s blockbuster cancer treatment Keytruda has been handed another indication by the FDA.

The US regulator announced on Thursday that it has approved Keytruda to serve as an adjuvant treatment for non-small cell lung cancer (NSCLC), which is its fifth indication in NSCLC and 34th indication overall.

According to a Merck release, the approval is based on data from a Phase III trial, dubbed Keynote-091, which measured disease-free survival in patients who received chemotherapy following surgery. The data from Merck displayed that Keytruda cut down on the risk of disease recurrence or death by 27% versus placebo.

FDA re­ports ini­tial 'no sig­nal' for stroke risk with Pfiz­er boost­ers, launch­es con­comi­tant flu shot study

The FDA hasn’t detected any potential safety signals, including for stroke, in people aged 65 years and older who have received Pfizer’s bivalent Covid booster, one senior official told members of the agency’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Thursday.

The update comes as the FDA and CDC investigate a “preliminary signal” that may indicate an increased risk of ischemic stroke in older Americans who received Pfizer’s updated shot.

FDA cuts off use for As­traZeneca’s Covid-19 ther­a­py Evusheld

The FDA has stopped use of another drug as a result of the new coronavirus variants. On Thursday, the agency announced that AstraZeneca’s antibody combo Evusheld, which was an important prevention option for many immunocompromised people and others, is no longer authorized.

The FDA said it made its decision based on the fact that Evusheld works on fewer than 10% of circulating variants.

Evusheld was initially given emergency authorization at the end of 2021. However, as Omicron emerged, so did studies that showed Evusheld might not work against the dominant Omicron strain. In October, the FDA warned healthcare providers that Evusheld was useless against the Omicron subvariant BA.4.6. It followed that up with another announcement earlier this month that it did not think Evusheld would work against the latest Omicron subvariant XBB.1.5.