Ned Sharp­less is not play­ing with the FDA’s gold stan­dard — any more than Scott Got­tlieb did

Ned Sharp­less got a chance to out­line just what kind of act­ing FDA chief he’ll be in the wake of Scott Got­tlieb’s de­par­ture from the agency. And aside from a more con­ser­v­a­tive se­lec­tion of socks, he’s clear­ly plan­ning to adopt the ex­act same fash­ions laid down by his pre­de­ces­sor.

Ned Sharp­less. (DARR BEIS­ER/NA­TION­AL CAN­CER IN­STI­TUTE)

Click on the im­age to see the full-sized ver­sion


In an all-hands-on-deck ad­dress to staffers, Sharp­less promised to fit the Got­tlieb mold per­fect­ly, vow­ing to main­tain the agency’s gold stan­dard on drug ap­provals while do­ing what he can to keep the copy­cats com­ing to help tamp down on drug costs.

So let me re­as­sure you, I am not plan­ning any rad­i­cal changes from what the FDA has been try­ing to ac­com­plish.

Bioreg­num Opin­ion Col­umn by John Car­roll

That’s a theme that will be mu­sic to the ears of the bio­phar­ma in­dus­try, which quick­ly swooned to Got­tlieb’s en­er­getic mes­sages on speed­ing ap­provals when­ev­er ap­pro­pri­ate while hold­ing the line on safe­ty and ef­fi­ca­cy stan­dards. The fu­ture of the FDA proved to be a huge is­sue at the start of Pres­i­dent Don­ald Trump’s term in of­fice, as Trump en­ter­tained some lib­er­tar­i­an no­tions that fright­ened the be­je­sus out of com­pa­nies that clear­ly want­ed a more re­cep­tive reg­u­la­to­ry ap­proach — with­out any reg­u­la­to­ry an­ar­chy that would de­stroy the val­ue of an FDA OK.

Sharp­less got right to it. His bot­tom line:

Nec­es­sar­i­ly, there will be course ad­just­ments as new facts emerge, but es­sen­tial­ly, I feel I am walk­ing in­to an or­ga­ni­za­tion on a good tra­jec­to­ry, and my main job is to fig­ure out how keep that go­ing.

Now how to do that:  let me sug­gest two guid­ing prin­ci­ples.

First, I be­lieve our ef­forts should re­ly on and be guid­ed by the sci­ence. As a re­searcher, I am used to let­ting da­ta dri­ve my de­ci­sion-mak­ing, and I know this has al­ways been the ap­proach at FDA.

Sec­ond, when wran­gling with the com­plex is­sues that face the agency, we will keep top of mind our mis­sion of pro­tect­ing and pro­mot­ing pub­lic health, and what that means to the Amer­i­can pub­lic.

So that’s what will steer my pri­or­i­ties as act­ing com­mis­sion­er: a com­mit­ment to sci­ence-based de­ci­sion-mak­ing and pri­or­i­tiz­ing our ef­forts for the ben­e­fit of the pub­lic health.

That mes­sage on con­ti­nu­ity ex­tends to the agency’s work hus­tling up less ex­pen­sive knock­offs.

I promise you, for ex­am­ple, that we’ll con­tin­ue our im­por­tant and suc­cess­ful work to in­crease com­pe­ti­tion and reign in pre­scrip­tion drug costs through ad­vances in our gener­ic drug and biosim­i­lars pro­grams

And we’ll con­tin­ue to do every­thing we can to make the de­vel­op­ment of new treat­ments and cures more ef­fi­cient across our med­ical prod­uct cen­ters, while en­sur­ing that we main­tain FDA’s gold stan­dard of safe­ty and ef­fi­ca­cy.

What could be more re­as­sur­ing to the in­dus­try, law­mak­ers and the pub­lic?

In­ter­est­ing­ly, Got­tlieb’s de­par­ture from the FDA was al­so marked by his in­sis­tence that the rank-and-file drug de­vel­op­ers of the world need­ed to do more to adopt the re­forms he had pushed, look­ing for faster, more ef­fi­cient ways to de­vel­op drugs. The chal­lenge for Sharp­less will be to see if he con­tin­ues that push as an in­dus­try train­er look­ing to get some out-of-shape de­vel­op­ers up to speed with the new or­der. And that means urg­ing reg­u­la­tors as well to get the lead out.

Scott Got­tlieb

Con­ti­nu­ity may sound like the ex­act right mes­sage here and now. But if that be­comes a con­ser­v­a­tive ap­proach to main­tain­ing stan­dards and tra­di­tions, plea­sure may soon be re­placed by an un­easy feel­ing that the FDA may once again be­come more of an ob­sta­cle and less of a part­ner. And that won’t be wel­come.

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.

Turns out, Rudy Tanzi did­n't see much of a sto­ry about a hid­den link be­tween En­brel and Alzheimer's ei­ther

The Wash­ing­ton Post man­aged to whip up the quick­est in­dus­try con­sen­sus I’ve ever seen that one of its re­porters was pur­vey­ing overblown non­sense with a sto­ry that Pfiz­er was sit­ting on da­ta sug­gest­ing that En­brel could be an ef­fec­tive treat­ment for Alzheimer’s. 

In cov­er­ing that bit of an­ti-Big Phar­ma fan­ta­sy — there are lots of rea­sons to go af­ter phar­ma, but this piece was lu­di­crous — I not­ed com­ments in the sto­ry from some promi­nent peo­ple in the field crit­i­ciz­ing Pfiz­er for not pub­lish­ing the da­ta. I sin­gled out Rudy Tanzi at Har­vard and then ap­plied some added crit­i­cism for the things he’s done to hype — in my opin­ion — high­ly ques­tion­able as­sump­tions. You can see it in the link. 

Neil Woodford, Woodford Investment Management via YouTube

Un­der siege, in­vest­ment man­ag­er Wood­ford faces an­oth­er in­vest­ment shock

Em­bat­tled UK fund man­ag­er Neil Wood­ford — who has con­tro­ver­sial­ly blocked in­vestors from pulling out from his flag­ship fund to stem the blood­let­ting, af­ter a slew of dis­ap­point­ed in­vestors fled fol­low­ing a se­ries of sour bets — is now pay­ing the price for his ac­tions via an in­vestor ex­o­dus on an­oth­er fund.

Har­g­reaves Lans­down, which has in the past sold and pro­mot­ed the Wood­ford funds via its re­tail in­vest­ment plat­form, has re­port­ed­ly with­drawn £45 mil­lion — its en­tire po­si­tion — from the in­vest­ment man­ag­er’s In­come Fo­cus Fund.

Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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J&J gains an en­thu­si­as­tic en­dorse­ment from Pres­i­dent Don­ald Trump for their big new drug Spra­va­to

Pres­i­dent Don­ald Trump has lit­tle love for Big Phar­ma, but there’s at least one new drug that just hit the mar­ket which he is en­am­ored with.

Trump, ev­i­dent­ly, has been read­ing up on J&J’s new an­ti-de­pres­sion drug, Spra­va­to. And the pres­i­dent — who of­ten likes to break out in­to a full-throat­ed at­tack on greedy drug­mak­ers — ap­par­ent­ly en­thused about the ther­a­py in a meet­ing with of­fi­cials of Vet­er­ans Af­fairs, which has long grap­pled with de­pres­sion among vet­er­ans.

Sil­i­con Val­ley's most an­tic­i­pat­ed slide deck just dropped. What does it mean for bio­phar­ma's dig­i­tal teams?

These aren’t the typ­i­cal slides you’d see at End­points — no mol­e­cules, clin­i­cal pro­grams, or p-val­ues. In­stead, we’ll talk dig­i­tal and in­ter­net trends, fac­tors that elite glob­al brands — re­gard­less of in­dus­try — must first mea­sure and un­der­stand be­fore de­ploy­ing prod­ucts in­to the world. That’s a con­cept that most of our Big Phar­ma au­di­ence is in tune with. Dig­i­tal aware­ness is key to suc­cess in the dis­cov­ery, de­vel­op­ment, and mar­ket­ing of new bio­phar­ma­ceu­ti­cals, and most of the ma­jors now have a chief dig­i­tal of­fi­cer: No­var­tis, Sanofi, and Pfiz­er, just to name a few.

Bain’s biotech team has cre­at­ed a $1B-plus fund — with an eye to more Big Phar­ma spin­outs

One of the biggest investors to burst onto the biotech scene in recent years has re-upped with more than a billion dollars flowing into its second fund. And this next wave of bets will likely include more of the Big Pharma spinouts that highlighted their first 3 years in action.

Adam Koppel and Jeff Schwartz got the new life sciences fund at Bain Capital into gear in the spring of 2016, as they were putting together a $720 million fund with $600 million flowing in from external investors and the rest drawn from the Bain side of the equation. This time the external investors chipped in $900 million, with Bain coming in for roughly $180 million more.

They’re not done with Fund I, with plans to add a couple more deals to the 15 they’ve already posted. And once again, they’re estimating another 15 to 20 investments over a 3- to 5-year time horizon for Fund II.

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News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.