Study high­lights the FDA’s speed in ap­prov­ing can­cer drugs as a new com­mish looks to rev up re­views

Scott Got­tlieb

Dur­ing his con­fir­ma­tion hear­ing on Wednes­day Scott Got­tlieb em­pha­sized that he would start his new job as FDA com­mis­sion­er by fo­cus­ing on dif­fer­ent parts of the agency that have lagged in ac­cel­er­at­ing drug re­views. With the pres­i­dent beat­ing the ta­ble for a stream­lined ap­proval process, you can ex­pect some fresh pres­sure from the top to speed things up. And now Got­tlieb — who was al­ready on record on that score — has some ad­di­tion­al da­ta to point to for his ef­fi­cien­cy cam­paign, high­light­ing where the agency has been ef­fec­tive in speed­ing ap­provals and where it might push for faster ac­tion.

Writ­ing in the New Eng­land Jour­nal of Med­i­cine, re­searchers com­pared the ap­proval track record for the FDA against the EMA. And once again, the FDA comes out on top. But this is not a blan­ket en­dorse­ment.

Look­ing at reg­u­la­to­ry ac­tions tak­en be­tween 2011 and 2015, the re­searchers — Nicholas S. Down­ing, from Brigham and Women’s Hos­pi­tal, Au­drey D. Zhang, New York Uni­ver­si­ty School of Med­i­cine, and Joseph S. Ross, Yale School of Med­i­cine — found that the FDA had ap­proved 170 ther­a­pies, com­pared to 144 from the EMA.

Richard Padzur

There were sig­nif­i­cant­ly more or­phan drug ap­provals at the FDA than the EMA, 43.5% vs. 25% of the ap­proved agents, which will al­so at­tract scruti­ny as crit­i­cism of the or­phan in­cen­tive pro­gram mounts. Re­views were, on av­er­age, sig­nif­i­cant­ly short­er at the FDA: 306 days com­pared to 383 days. And if you just look at the same drugs ap­proved by both agen­cies, the FDA still wins hand­i­ly — 303 days to 369 days.

But here’s the in­ter­est­ing part.

Where the FDA ex­celled in beat­ing the EMA was in can­cer and hema­to­log­i­cal drugs. Drugs de­signed as or­phan ther­a­pies were al­so ad­van­taged. That dis­tinc­tion in on­col­o­gy high­lights the role that Richard Paz­dur has played in re­mak­ing the way that drugs are re­viewed and ap­proved in his wing of the FDA. And he’ll be the ex­am­ple that is held up to oth­er reg­u­la­tors to em­u­late.

Can­cer, though, lends it­self to faster re­views, as they’re head­ed to dy­ing, ad­vanced stage pa­tients ini­tial­ly. The risk/ben­e­fit pro­file is sig­nif­i­cant­ly dif­fer­ent in, say, heart dis­ease or di­a­betes.

You can ex­pect to hear plen­ty more af­ter Got­tlieb nails down the job as FDA chief.

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.

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.

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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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.