FDA or­ders a rare re­treat on Am­i­cus’ mi­gala­s­tat, drop­ping PhI­II de­mand and sig­nal­ing a ma­jor shift for drug de­vel­op­ers

Megan Crow­ley is rec­og­nized by Pres­i­dent Don­ald Trump dur­ing his first ad­dress to a joint ses­sion of Con­gress on Feb­ru­ary 28, 2017. At 15 months old, Crow­ley was di­ag­nosed with Pompe Dis­ease and not ex­pect­ed to live more than a few short years. AP Im­ages


Am­i­cus Ther­a­peu­tics $FOLD an­nounced ear­ly Tues­day that the FDA has dropped its de­mand for a new Phase III study of its Fab­ry dis­ease drug mi­gala­s­tat af­ter it was stiff-armed by un­sat­is­fied reg­u­la­tors last fall — then un­der a dif­fer­ent ad­min­is­tra­tion with a dif­fer­ent FDA com­mis­sion­er.

In­stead of pur­su­ing a new and ex­pen­sive late-stage study, Am­i­cus CEO John Crow­ley tells me the com­pa­ny has the green light to file again with the da­ta at hand. And based on dis­cus­sions with the FDA, he adds, the com­pa­ny is shoot­ing to file in the 4th quar­ter, with an ac­cel­er­at­ed ap­proval that could ar­rive in 2018, po­ten­tial­ly slic­ing years off the process.

“These are the days it’s good to be a biotech CEO,” says Crow­ley, who has been on a roller coast­er ride at the FDA over the past year af­ter be­ing forced to walk back his own plans last No­vem­ber.

John Crow­ley

There is no guar­an­tee of an ap­proval here, but the agency’s sud­den volte-face clear­ly sig­nals a new tone that vast­ly im­proves the biotech’s shot at an OK — at least in so far as we hear it from Crow­ley. And that could have a ma­jor in­flu­ence on oth­er de­vel­op­ers tack­ling the same task.

In­vestors cheered on the sud­den change in for­tunes at Am­i­cus, bid­ding up shares by 43% in pre-mar­ket trad­ing Tues­day.

Crow­ley has nev­er made a se­cret of the fact that he felt the drug — ap­proved in Eu­rope last year — was ready to go in the US af­ter it com­plet­ed the piv­otal pro­gram. The FDA, though, re­ject­ed his ap­pli­ca­tion, say­ing reg­u­la­tors want­ed more safe­ty da­ta than the Eu­ro­peans need­ed.

That re­jec­tion be­came a cause cele­bre of sorts in DC, as Pres­i­dent Don­ald Trump turned to Crow­ley’s daugh­ter, Megan, who suf­fers from Pompe dis­ease, and called out the FDA for the “slow and bur­den­some” process in­volved in new drug ap­provals dur­ing an ad­dress to Con­gress.

“Our slow and bur­den­some ap­proval process at the Food and Drug Ad­min­is­tra­tion keeps too many ad­vances, like the one that saved Megan’s life, from reach­ing those in need,” Trump said at the time. “If we slash the re­straints, not just at the FDA, but across our gov­ern­ment, then we will be blessed with far more mir­a­cles like Megan.”

Crow­ley picked up on that change in think­ing at the White House in a blog post at the time in which he said: “The FDA’s reg­u­la­tion of the or­phan de­vel­op­ment process is be­com­ing less flex­i­ble, less ef­fi­cient and less pa­tient-cen­tered.”

I asked Crow­ley what had changed be­tween the set­back last fall and the new, soft green light that the FDA has switched to.

First and fore­most, Crow­ley hit the theme that the com­pa­ny had made a con­vinc­ing case with what was avail­able. That in­cludes new da­ta and analy­sis that was made pos­si­ble af­ter the re­jec­tion. The com­pa­ny start­ed from the be­gin­ning to mar­shal the ev­i­dence need­ed to meet the agency’s gold stan­dard for an OK. That, he says, was ab­solute­ly key, ul­ti­mate­ly win­ning the day.

In ad­di­tion, he says there’s al­so a new frame­work for drug re­views that’s been gath­er­ing steam at the agency.

“I do think, can­did­ly, that there is an emerg­ing frame­work in rare dis­eases,” he says, point­ing to new ap­provals for Bio­marin on Bat­ten dis­ease as well as a nov­el la­bel ex­ten­sion for Ver­tex based on their in vit­ro as­say.

Scott Got­tlieb ap­pears be­fore a US Sen­ate sub­com­mit­tee, June 20, 2017 AP Im­ages

You can bet, though, that there will be plen­ty of sen­ti­ment sug­gest­ing that a change of ad­min­is­tra­tions and a new FDA com­mis­sion­er in Scott Got­tlieb made a telling dif­fer­ence for an abrupt about-face that is ex­tra­or­di­nar­i­ly rare to see at the FDA.

What­ev­er the cause, though, in­stead of prepar­ing to gath­er new da­ta that wouldn’t be avail­able un­til 2019, Crow­ley is back to mak­ing launch plans in the US. That will prob­a­bly re­quire a com­mer­cial group of about 50, he says, com­pared to the 80 need­ed to ad­dress a more com­plex and chal­leng­ing pay­er en­vi­ron­ment in Eu­rope.

Biotech has large­ly cheered on new FDA com­mis­sion­er Scott Got­tlieb af­ter he won con­fir­ma­tion by the Sen­ate. He promised to turn to new tech­nolo­gies to help ac­cel­er­ate new ap­provals, but vowed to stick with the in­dus­try gold stan­dard that has gov­erned reg­u­la­tors thoughts on safe­ty and ef­fi­ca­cy date.

So did the FDA just soft­en its stan­dards or wake up to the fact that the drug should nev­er have been re­ject­ed in the first place?

Let’s start the dis­cus­sion.

Amarin CEO John Thero discussing the company's plans for Vascepa, August 2019 — via Bloomberg

Amarin wins a block­buster ap­proval from the FDA. Now every­one can shift fo­cus to the patent

For all those people who could never quite believe that Amarin $AMRN would get an expanded label with blockbuster implications, the stress and anxiety on display right up to the last minute on Twitter can now end. But new, pressing questions will immediately surface now that the OK has come through.

On Friday afternoon, the FDA stamped its landmark approval on the industrial strength fish oil for reducing cardio risks for a large and well defined population of patients. The approval doesn’t give Amarin everything it wants in expanding its use, losing out on the primary prevention group, but it goes a long way to doing what the company needed to make a major splash. The approval was cited for patients with “elevated triglyceride levels (a type of fat in the blood) of 150 milligrams per deciliter or higher. Patients must also have either established cardiovascular disease or diabetes and two or more additional risk factors for cardiovascular disease.”

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Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Sarep­ta was stunned by the re­jec­tion of Vyondys 53. Now it's stun­ning every­one with a sur­prise ac­cel­er­at­ed ap­proval

Sarepta has a friend in the FDA after all. Four months after the agency determined that it would be wrong to give Sarepta an accelerated approval for their Duchenne MD drug golodirsen, regulators have executed a stunning about face and offered the biotech a quick green light in any case.

It was the agency that first put out the news late Thursday, announcing that Duchenne MD patients with a mutation amenable to exon 53 skipping will now have their first targeted treatment: Vyondys 53, or golodirsen. Having secured the OK via a dispute resolution mechanism, the biotech said the new drug has been priced on par with their only other marketed drug, Exondys 51 — which for an average patient costs about $300,000 per year, but since pricing is based on weight, that sticker price can even cross $1 million.

Sarepta shares $SRPT surged 23% after-market to $124.

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Paul Biondi (File photo)

Paul Biondi's track record at Bris­tol-My­ers cov­ered bil­lions in deals of every shape and size. Here's the com­plete break­down

Paul Biondi was never afraid to bet big during his stint as business development chief at Bristol-Myers Squibb. And while the gambles didn’t all pay out, by any means, his roster of pacts illustrates the broad ambitions the pharma giant has had over the last 5 years — capped by the $74 billion Celgene buyout.

On Thursday, we learned that Biondi had exited the company. And Chris Dokomajilar at DealForma came up with the complete breakdown on every buyout, licensing pact and product purchase Bristol-Myers forged during his tenure in charge of the BD team at one of the busiest companies in biopharma.

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Arie Belldegrun (Photo: Jeff Rumans for Endpoints News)

Ju­ry finds Gilead li­able for $585M and big roy­al­ties in Kite CAR-T patent case

A Kite deal that’s already become a burden on Gilead’s back just got heavier as a California jury has ruled Gilead must pay Bristol-Myers Squibb and Sloan Kettering $585 million plus a 27.6% royalty for patent infringement committed by its subsidiary. The ruling is almost certain to be appealed.

Kite Pharma — founded by Arie Belldegrun, now focused on a next-gen CAR-T company — has been facing a lawsuit since the day its first CAR–T therapy won approval in October, 2017. Juno Therapeutics and Sloan Kettering filed a complaint saying Kite had copied its technology. Gilead acquired Kite in June of that year for $11.9 billion.  Juno was acquired the following year by Celgene for $9 billion, before Celgene was acquired by Bristol-Myers Squibb in 2019.

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FDA ex­pert pan­el unan­i­mous­ly rec­om­mends ap­proval for Hori­zon Ther­a­peu­tics eye drug

An FDA advisory committee noted with concern a small safety database but unanimously endorsed a Horizon Therapeutics drug for a rare eye autoimmune disease that can blind patients: teprotumumab for thyroid eye disease (TED).

“It was a pretty easy vote,” said Erica Brittain, an NIH biostatistician and one of the 12 panelists on FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee.

This image shows a lab technician measuring the zone of inhibition during an antibiotic sensitivity test, 1972. The zone of inhibition is measured and compared to a standard in order to determine if an antibiotic is effective in treating the bacterial infection. (Gilda Jones/CDC via Getty Images)

Bio­phar­ma has aban­doned an­tibi­ot­ic de­vel­op­ment. Here’s why we did, too.

Timing is Everything
When we launched Octagon Therapeutics in late 2017, I was convinced that the time was right for a new antibiotic discovery venture. The company was founded on impressive academic pedigree and the management team had known each other for years. Our first program was based on a compelling approach to targeting central metabolism in the most dangerous bacterial pathogens. We had already shown a high level of efficacy in animal infection models and knew our drug was safe in humans.

Shehnaaz Suli­man dives back in­to Alzheimer's at Alec­tor; Pyx­is re­cruits Spring­Works founder Lara Sul­li­van as CEO

Amid Shehnaaz Suliman’s lengthy resume it could be easy to miss her stint leading early-stage Alzheimer’s R&D at Genentech, where she oversaw a program for the ill-fated crenezumab and initiated one of the first prevention studies around the devastating neurodegenerative disease. But it is this experience that she — after thinking long and hard about her next career move over the past months — will be leaning heavily on as the first president and COO of Alector.

PhII fail­ure in rare neu­rode­gen­er­a­tive dis­ease? No mat­ter, Bio­gen will mo­tor on in Alzheimer's

Biogen’s fierce focus on disorders of the brain has hit another roadblock.

On Friday, the US drugmaker — which recently resurrected its amyloid-targeting Alzheimer’s drug, aducanumab — said its anti-tau drug, gosuranemab, failed a mid-stage study in patients with progressive supranuclear palsy (PSP), a rare brain disorder that results from deterioration of brain cells that control movement and thought.