Pres­i­dent Trump names Scott Got­tlieb as the next FDA com­mis­sion­er. What now?

Scott Got­tlieb

Pres­i­dent Trump has named Scott Got­tlieb as the next FDA com­mis­sion­er, re­solv­ing an is­sue that has huge im­pli­ca­tions for the bio­phar­ma in­dus­try and drug de­vel­op­ment.

Got­tlieb’s nom­i­na­tion will be wide­ly ap­plaud­ed by the bio­phar­ma in­dus­try, which will see the move as a com­mit­ment for con­tin­ued re­form with­out the kind of whole­sale dereg­u­la­tion that would scut­tle the agency’s gold stan­dard for drug re­views.

“Thank God it’s Got­tlieb,” mes­saged Baird’s Bri­an Sko­r­ney in his week­ly wrap-up.

As I re­port­ed two weeks ago, Got­tlieb has mapped out an ag­gres­sive re­form agen­da in an­tic­i­pa­tion of this ap­point­ment.

Ac­cord­ing to his state­ments as well as com­ments he’s made to peo­ple fa­mil­iar with his think­ing on the FDA, Got­tlieb in­tends to shoot for the rapid ap­proval of com­plex gener­ics, ush­er­ing in a wave of less ex­pen­sive ri­vals to some of the biggest block­busters on the mar­ket. He’s al­so like­ly to spur the FDA to fol­low the course laid out by agency can­cer czar Richard Paz­dur in speed­ing new ap­provals, pos­si­bly set­ting up a spe­cial unit aimed at or­phan drugs to has­ten OKs with small­er, bet­ter de­signed clin­i­cal tri­als.

Got­tlieb is like­ly to roll back rules re­lat­ed to pre-mar­ket de­vel­op­ment, most no­tice­ably the car­dio­vas­cu­lar out­comes stud­ies for some new meds. A new em­pha­sis on bio­mark­ers as a sur­ro­gate end­point along with an open em­brace of sim­u­la­tion and mod­el­ing could be next.

Oth­er po­ten­tial re­forms in­clude the pos­si­ble quick adop­tion of new de­vices that could be used to  im­prove the kind of medtech Ap­ple, Ver­i­ly and oth­ers have been work­ing on.

Got­tlieb has al­so backed the pub­li­ca­tion of the FDA’s com­plete re­sponse let­ters, de­tail­ing the rea­sons why the agency re­jects a drug. Over the years reg­u­la­tors have rou­tine­ly com­plained that many com­pa­nies have been less than hon­est in re­count­ing the FDA’s po­si­tion on a drug. The FDA is cur­rent­ly re­strict­ed by law in its pub­lic dis­cus­sion about a new drug ap­proval.

The move could spell re­lief for Am­i­cus Ther­a­peu­tics $FOLD and CEO John Crow­ley, who per­son­al­ly lob­bied the pres­i­dent on the FDA’s de­ci­sion to de­lay any fi­nal de­ci­sion on their drug for Fab­ry dis­ease so they could see the re­sults of a safe­ty study in 2019.

Left out in the move: Jim O’Neill. The close as­so­ciate of Pe­ter Thiel, O’Neill fa­mous­ly sug­gest­ed that drugs should be ap­proved based on safe­ty alone, let­ting con­sumers sort out what works. That left many fear­ing that Trump in­tend­ed to toss out the reg­u­la­to­ry frame­work for new drug ap­provals, rais­ing fears that his idea of com­pe­ti­tion would al­low de fac­to place­bos to com­pete for mar­ket share.

Trump has re­peat­ed­ly crit­i­cized bio­phar­ma for out­ra­geous­ly high drug prices, vow­ing to sim­pli­fy the drug de­vel­op­ment process as he al­so sought to dra­mat­i­cal­ly low­er the price of drugs. Got­tlieb will be ex­pect­ed to de­liv­er.

“Among the FDA com­mis­sion­er can­di­dates we’ve read about, Dr. Got­tlieb is well known to in­dus­try and has the req­ui­site ex­pe­ri­ence and tal­ent to re­form and stream­line the FDA ap­proval process,” Hol­land & Knight’s Michael Ga­ba told me re­cent­ly.

One of the rea­sons that Got­tlieb is well known in the in­dus­try is that the physi­cian and for­mer FDA of­fi­cial un­der George W. Bush has plen­ty of di­rect ties to it. As a ven­ture part­ner at New En­ter­prise As­so­ci­ates he’s a board mem­ber at Me­dA­vante, which mar­kets soft­ware for an­a­lyz­ing clin­i­cal tri­al da­ta. And he’s been an ad­vi­sor to Glax­o­SmithK­line, which has shown no ap­petite for rad­i­cal change in the way de­vel­op­ers prove a drug works.

Those ties, along with Trump’s free­wheel­ing com­ments on dereg­u­la­tion, will now be­come tar­gets for De­moc­rats in Wash­ing­ton DC. But un­less there’s a nasty sur­prise hid­den from view, none of it presents a se­ri­ous chal­lenge to his ac­cep­tance as head of the FDA.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.