FDA de­ci­sion on Ver­tex's CF triple will come just ahead of planned CEO shake­up

Ver­tex has clinched a pri­or­i­ty re­view for the all-im­por­tant cys­tic fi­bro­sis triple that will blaze the trail for treat­ing a large group of pa­tients un­helped by its cur­rent drugs.

Resh­ma Ke­wal­ra­mani

FDA reg­u­la­tors have set a PDU­FA date of March 19, 2020, just a year af­ter the Boston biotech post­ed pos­i­tive Phase III re­sults show­ing that peo­ple with two F508del mu­ta­tions ex­pe­ri­enced sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments in lung func­tion af­ter a 4-week reg­i­men of VX-445, teza­caftor and iva­caftor. Af­ter re­view­ing 24-week da­ta among pa­tients with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion — and thor­ough­ly com­par­ing the VX-445 triple with an­oth­er com­bo fea­tur­ing VX-659 on scores like safe­ty, drug-drug in­ter­ac­tions, and pho­to­sen­si­tiv­i­ty — Ver­tex ul­ti­mate­ly went with VX-445.

Dubbed elex­a­caftor, the new drug is a “cor­rec­tor” de­signed to fix the shape and course of de­fec­tive CFTR pro­tein, bring­ing more of them to the cell sur­face. No­tably, teza­caftor has a sim­i­lar func­tion but does not ad­dress min­i­mal func­tion mu­ta­tions. Mean­while, iva­caftor is a “po­ten­tia­tor” that in­creas­es the func­tion of the pro­tein once it reach­es the cell sur­face.

Iva­caftor is mar­ket­ed on its own as Ka­ly­de­co, while Symdeko con­sists of both iva­caftor and teza­caftor.

Jeff Lei­den

An ap­proval in March would mark a fi­nal vic­to­ry for CEO Jeff Lei­den, who has an­nounced a sur­prise tran­si­tion to the ex­ec­u­tive chair­man role while pass­ing the ba­ton to cur­rent CMO Resh­ma Ke­wal­ra­mani in April 2020. Sub­se­quent dis­cus­sions on pric­ing would al­so draw at­ten­tion to her ne­go­ti­at­ing style, giv­en Ver­tex’s high pro­file con­fronta­tions with Eu­ro­pean sin­gle-pay­er sys­tems.

“If ap­proved, the VX-445 (elex­a­caftor), teza­caftor and iva­caftor triple com­bi­na­tion reg­i­men would be a sig­nif­i­cant ad­vance in CF treat­ment as the first CFTR mod­u­la­tor for those with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion, and bring ad­di­tion­al ben­e­fit to pa­tients with two F508del mu­ta­tions,” Ke­wal­ra­mani said in a state­ment.

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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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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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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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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Roy Baynes, Merck

FDA bats back Mer­ck’s ‘pipeline in a prod­uct,’ de­mands more ef­fi­ca­cy da­ta

Despite some heavy blowback from analysts, Merck execs maintained an upbeat attitude about the market potential of its chronic cough drug gefapixant. But the confidence may be fading somewhat today as Merck puts out news that the FDA is handing back its application with a CRL.

Dubbed by Merck’s development chief Roy Baynes as a “pipeline in a product” with a variety of potential uses, Merck had fielded positive late-stage data demonstrating the drug’s ability to combat chronic cough. The drug dramatically reduced chronic cough in Phase III, but so did placebo, leaving Merck’s research team with a marginal success on the p-value side of the equation.

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Florida Gov. Ron DeSantis (AP Photo/Wilfredo Lee, File)

Opin­ion: Flori­da is so mAb crazy, Ron De­San­tis wants to use mAbs that don't work

Florida Gov. Ron DeSantis is trying so hard to politicize the FDA and demonize the federal government that he entered into an alternate universe on Monday evening in describing a recent FDA action to restrict the use of two monoclonal antibody, or mAb, treatments for Covid-19 that don’t work against Omicron.

Without further ado, let’s break down his statement from last night, line by line, adjective by adjective.

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.

Steve Worland, eFFECTOR CEO

Sur­prise piv­ot rocks eF­FEC­TOR's I/O plans — al­though ex­ecs promise big­ger slice of the NSCLC mar­ket in the long run

When eFFECTOR Therapeutics went public last summer on the coattails of a reverse merger with Locust Walk’s SPAC, the potential of its lead drug, tomivosertib, as a combo agent with Merck’s flagship PD-1 Keytruda was hailed as the main draw.

But the biotech is now axing those plans and essentially starting over.

In a surprise move, San Diego-based eFFECTOR said it’s halting the development of tomivosertib in non-small cell lung cancer patients who have already progressed on Keytruda monotherapy after running into enrollment challenges in a Phase IIb trial.

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US re­stricts use of two mon­o­clon­al an­ti­bod­ies that don't work against Omi­cron

Two monoclonal antibody combos from Eli Lilly and Regeneron are no longer authorized in the US, and shipments to states have ceased because HHS said they are “highly unlikely” to work against the sweeping new variant Omicron.

The move by the FDA comes as states like Florida have become insistent that the mAbs need to be independently evaluated, although the federal Department of Health and Human Services, which has shipped hundreds of thousands of these two mAbs to states in recent weeks, did not ship any this week.