Once re­ject­ed, FDA now rolls out a short red car­pet for Am­i­cus’ mi­gala­s­tat

John Crow­ley

Am­i­cus CEO $FOLD John Crow­ley must have made a very fa­vor­able im­pres­sion on the FDA as he lob­bied se­nior of­fi­cials to over­turn their re­jec­tion of his ex­per­i­men­tal Fab­ry drug mi­gala­s­tat.

Not on­ly did reg­u­la­tors just ac­cept the fil­ing for a new re­view, the agency is giv­ing it pri­or­i­ty sta­tus, cut­ting the re­think to 6 months and flag­ging a clear in­di­ca­tion that it’s an odds-on fa­vorite for an of­fi­cial mar­ket­ing OK .

Cut­ting 4 months off the re­view process, the FDA will have its new de­ci­sion out by Au­gust 13.

An­a­lysts are al­ready count­ing the near-term rev­enue that Am­i­cus stands to earn. Joseph Schwartz at Leerink not­ed:

With the PDU­FA goal date of Aug 13, 2018, US rev­enue could con­tribute to FY2018 rev­enue guid­ance of $75M-$85M an­nounced last month. We con­tin­ue to see strong growth dri­vers for Am­i­cus this yr. as the com­pa­ny con­tin­ues to ad­vance its mi­gala­s­tat (Galafold; Fab­ry dis­ease) and ATB200/AT2221 (Pompe dis­ease) pro­grams through the clin­ic.

Crow­ley had em­ployed some du­bi­ous po­si­tions as he fought against the re­jec­tion, which came with a de­mand from the FDA for a new Phase III study of gas­troin­testi­nal symp­toms. Ini­tial­ly, the CEO had told in­vestors it would take 2 years to come up with the da­ta. But he told Janet Wood­cock and oth­ers that the ac­tu­al time­line would be 5 to 7 years, which made the de­vel­op­ment pro­gram im­prac­ti­cal.

Scott Got­tlieb

This was one of three drug pro­grams that the FDA re­versed it­self on in the months af­ter Scott Got­tlieb took over as com­mis­sion­er at the FDA, with Eli Lil­ly and Ther­a­peu­tic­sMD — chaired by ex-HHS sec­re­tary Tom­my Thomp­son — get­ting a do-over. The agency has nev­er ex­plained the re­jec­tions, nev­er ex­plained why it de­cid­ed to re­con­sid­er their de­ci­sion, or why it would of­fer Am­i­cus a quick de­ci­sion now.

But they nev­er do, re­quired by the rules to stay qui­et. Got­tlieb had ini­tial­ly said he would press for the redact­ed pub­li­ca­tion of com­plete re­sponse let­ters like these, but he re­cent­ly shrugged that off.

“Con­grat­u­la­tions, again!” Crow­ley wrote in a note to Got­tlieb at­tached to his case for a re­view. “As dis­cussed, the hopes and well be­ing of so many liv­ing with rare, dev­as­tat­ing dis­eases rests now with your great lead­er­ship and wis­dom. Thanks for your at­ten­tion to the at­tached.”

Got­tlieb’s staff told me that the com­mis­sion­er nev­er saw that pack­et, redi­rect­ing it to oth­er se­nior of­fi­cials at the FDA. Of­fi­cials, though, de­clined to ex­plain what Crow­ley meant by the word “again,” or if Got­tlieb had dis­cussed the is­sue with the CEO.

That note, along with Crow­ley’s as­ser­tions on the time­line to re­spond, were ob­tained un­der FOIA by the Pro­ject on Gov­ern­ment Over­sight.

Got­tlieb has open­ly ad­vo­cat­ed for a more ef­fi­cient re­view process, par­tic­u­lar­ly where rare dis­ease are con­cerned. That al­so squares with Pres­i­dent Don­ald Trump’s vow to speed up the reg­u­la­to­ry process. And af­ter start­ing out with a high rat­ing from the in­dus­try, biotech ex­ecs are now cheer­ing him on as one of the best things that’s hap­pened at the FDA in years.

Alexander Lefterov/Endpoints News

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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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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In a set­back, FDA or­ders Gilead to hit the brakes on their late-stage, $5B can­cer play

Gilead’s $5 billion drug magrolimab has run into a serious setback.

The FDA ordered Gilead to halt enrollment on their studies of the drug in combination with azacitidine after investigators reports revealed an “apparent imbalance” in the suspected unexpected serious adverse reactions between study arms. And the halt is raising questions about Gilead’s plans for a quick pitch to regulators.

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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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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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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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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.