High­light­ing a ma­jor shift at the FDA, reg­u­la­tors put Am­i­cus' once-spurned drug on fast track

In gen­er­al, the FDA’s fast track des­ig­na­tions don’t make news. The ma­jor­i­ty of new drugs which gain ap­proval these days get some sort of spe­cial sta­tus — to the point that it isn’t so spe­cial any­more.

But when you’re Am­i­cus Ther­a­peu­tics $FOLD, and you’re bounc­ing back from a sting­ing set­back for the lead drug mi­gala­s­tat, it’s a spe­cial mo­ment to an­nounce that the agency has put your drug for Fab­ry dis­ease on the fast track. Reg­u­la­tors are es­sen­tial­ly promis­ing to make things hap­pen fast as Am­i­cus lines up a new fil­ing be­fore the end of this year, though it’s not a guar­an­tee of any kind.

It’s al­so note­wor­thy for the in­dus­try, as the change of sta­tus high­lights a new regime at the FDA which has vowed to step up drug ap­provals — with­out dam­ag­ing the gold stan­dard on drug ap­provals.

FDA com­mis­sion­er Scott Got­tlieb is draw­ing a new line in the sand.

John Crow­ley, Am­i­cus

“As we ex­e­cute our in­ter­na­tion­al launch and con­tin­ue pur­su­ing glob­al reg­u­la­to­ry ap­provals for mi­gala­s­tat, it is our vi­sion to bring this im­por­tant treat­ment to even more peo­ple in more ge­o­gra­phies who may ben­e­fit,” said CEO John Crow­ley in a state­ment. “We look for­ward to sub­mit­ting our NDA and col­lab­o­rat­ing with the U.S. FDA through­out the reg­u­la­to­ry process.”

The FDA was in any­thing but a col­lab­o­ra­tive mood when it slammed Am­i­cus with a ma­jor de­lay last fall, de­mand­ing that it con­duct a Phase III study. But then came a new Trump ad­min­is­tra­tion and a new FDA com­mis­sion­er. And Crow­ley — who had talked di­rect­ly with Trump about the sit­u­a­tion, gain­ing his sup­port — was giv­en the green light to re­file over the sum­mer.

To­day, he al­so has the FDA’s com­mit­ment to speed things along.

Sig­nif­i­cant­ly, Am­i­cus isn’t the on­ly drug de­vel­op­er to ben­e­fit from the change of the pow­er group in Wash­ing­ton DC. Phar­ma gi­ant Eli Lil­ly al­so ex­e­cut­ed a swift turn­around af­ter bit­ter­ly crit­i­ciz­ing the FDA’s re­jec­tion of baric­i­tinib on safe­ty rea­sons. Now, in­stead of be­ing forced back in­to the clin­ic, Lil­ly has the FDA’s ap­proval to re­file as well. And both these drugs loom large for their re­spec­tive com­pa­nies.

The FDA doesn’t do any­thing like com­ment pub­licly about its stance on any drug, un­til and un­less they ac­tu­al­ly ap­prove it. But Am­i­cus and Lil­ly are prime ex­hibits in the case that the Trump/Got­tlieb ap­proach to drug de­vel­op­ment won’t be quite as ready to throw the red flag.

The im­pli­ca­tions for bio­phar­ma are huge.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Sanofi/Re­gen­eron mus­cle ahead of a ri­val No­var­tis/Roche team, win first ap­proval in key rhi­nos­i­nusi­tis field

Re­gen­eron and their part­ners at Sanofi have beat the No­var­tis/Roche team to the punch on an­oth­er key in­di­ca­tion for their block­buster an­ti-in­flam­ma­to­ry drug Dupix­ent. The drug team scored an ac­cel­er­at­ed FDA ap­proval for chron­ic rhi­nos­i­nusi­tis with nasal polyps, mak­ing this the first such NDA for the field.

An­a­lysts have been watch­ing this race for awhile now, as Sanofi/Re­gen­eron won a snap pri­or­i­ty re­view for what is now their third dis­ease in­di­ca­tion for this treat­ment. And they’re not near­ly done, build­ing up hopes for a ma­jor fran­chise.

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

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Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.

No­var­tis holds back the copy­cat brigade's at­tack on its top drug fran­chise — for now

A fed­er­al judge has put a gener­ic chal­lenge to No­var­tis’ block­buster mul­ti­ple scle­ro­sis drug Gilenya on hold while a patent fight plays out in court.

Judge Leonard P. Stark is­sued a tem­po­rary in­junc­tion ear­li­er this week, forc­ing My­lan, Dr. Red­dy’s Lab­o­ra­to­ries and Au­robindo Phar­ma to shelve their launch plans to al­low the patent fight to pro­ceed. He ruled that al­low­ing the gener­ics in­to the mar­ket now would per­ma­nent­ly slash the price for No­var­tis, even if it pre­vails. 

Top an­a­lyst finds a sil­ver lin­ing in Ab­b­Vie’s $63B Al­ler­gan buy­out — but there’s a catch

Af­ter get­ting beat up on all sides from mar­ket ob­servers who don’t much care for the lat­est mega-deal to ar­rive in bio­phar­ma, at least one promi­nent an­a­lyst now is start­ing to like what he sees in the num­bers for Ab­b­Vie/Al­ler­gan.

But it’s go­ing to take some en­cour­age­ment if Ab­b­Vie ex­ecs want it to last.

Ab­b­Vie’s mar­ket cap de­clined $20 bil­lion on Tues­day as the stock took a 17% hit dur­ing the day. And SVB Leerink’s Ge­of­frey Porges can see a dis­tinct out­line of an up­side af­ter re­view­ing the fun­da­men­tals of the deal.

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While Ako­rn works to re­vive its for­tunes, the FDA hits it with an­oth­er warn­ing let­ter

Ako­rn just can’t dig it­self out of its hole.

The spe­cial­ty gener­ic drug­mak­er has re­ceived yet an­oth­er warn­ing let­ter from the FDA this year. With­out dis­clos­ing any specifics, the Lake For­est, Illi­nois-based drug­mak­er on Wednes­day said the US reg­u­la­tor had is­sued the let­ter, cit­ing an in­spec­tion of its Som­er­set, New Jer­sey man­u­fac­tur­ing fa­cil­i­ty in Ju­ly and Au­gust of 2018. The com­pa­ny’s shares $AKRX dipped about 1.7% to $4.65 be­fore the bell.