How do you re­place a rock star like Scott Got­tlieb at the FDA? Maybe you can't

Any­one look­ing for a con­ve­nient weath­er vane to de­ter­mine the re­ac­tion to Scott Got­tlieb’s abrupt de­par­ture from the FDA need go no fur­ther than the $XBI. The S&P Biotech ETF took a 4% hit. And it was down an­oth­er 1.8% ahead of the bell on Thurs­day.


The mar­ket and the in­dus­try loved Got­tlieb, as we un­der­scored on sev­er­al oc­ca­sions with in­dus­try sur­veys high­light­ing the in­tense en­thu­si­asm for some­one who ad­vo­cat­ed for col­lab­o­rat­ing with the drug in­dus­try. Bio­phar­ma had a part­ner in Got­tlieb, and they all knew it.

Va­p­ing and the opi­oid epi­dem­ic — while big pub­lic pol­i­cy is­sues — meant ab­solute­ly noth­ing to the in­dus­try. An agency com­mit­ted to push­ing ap­provals while em­brac­ing in­no­va­tion at a time some land­mark ther­a­pies are be­ing ap­proved at a more rapid pace: That was pure gold. And it wasn’t just lip ser­vice. In this brave new world J&J could slip around the gold stan­dard on de­pres­sion drug da­ta and score an OK this week for es­ke­t­a­mine, a drug they plan to make a block­buster. A few years ago, that like­ly wouldn’t have hap­pened.

The one thing that could set­tle in­vestors and ex­ecs back down would be a new head of the FDA that could walk the line be­tween main­tain­ing the some­what weak­ened gold stan­dard on piv­otal da­ta and a free-for-all that would clear­ly aban­don a com­mit­ment to ap­prov­ing safe and ef­fec­tive drugs that met the risk/ben­e­fit analy­sis. 

Got­tlieb emerged ear­ly as a fa­vorite for the FDA job. He had ex­pe­ri­ence at the agency, he fit in­to the Trump ad­min­is­tra­tion’s orb with vows to speed things up, and an amaz­ing num­ber of in­dus­try in­sid­ers knew him and liked him.

He quick­ly be­came a rock star in bio­phar­ma, tweet­ing his way through every day about pol­i­cy.

So how do you re­place him and main­tain the same lev­el of en­thu­si­asm in bio­phar­ma? The usu­al sus­pects don’t cut it. 

We’re not even sure right now who will be the in­ter­im chief. Stephen Os­troff has tak­en the helm on a strict­ly in­ter­im ba­sis be­fore, but he re­tired last fall. Amy Aber­nethy was named prin­ci­pal deputy com­mis­sion­er just 2 months ago, com­ing to the FDA from a se­nior sci­en­tif­ic po­si­tion at Flat­iron af­ter a stint at Duke. She’s been tapped as the like­ly in­ter­im chief, and will def­i­nite­ly make the ru­mor mill as pos­si­bly the next head of the FDA. But she’s still large­ly an un­known in the in­dus­try, so don’t ex­pect much en­thu­si­asm right off the bat.

On the oth­er hand, if she gets the in­ter­im job, she’ll have a chance to shine. And Pres­i­dent Trump doesn’t seem pressed to get big jobs filled fast in Wash­ing­ton DC, so the in­ter­im role could last awhile. 

Na­tion­al Can­cer In­sti­tutes di­rec­tor Ned Sharp­less has emerged as one of the most fre­quent­ly cit­ed can­di­dates for the FDA job, with an ear­ly push from the Wall Street Jour­nal, which al­so quick­ly named Brett Giroir, as­sis­tant sec­re­tary at HHS, as an­oth­er pos­si­ble suc­ces­sor.

Jim O’Neill ev­i­dent­ly had a shot at the job a cou­ple of years ago, but the in­dus­try was shocked by the fact that Pe­ter Thiel’s can­di­date was even in the run­ning. He’s been as­so­ci­at­ed with some loony en­deav­ors, like seast­eading and the hunt for im­mor­tal­i­ty, and the in­dus­try sees him as a dis­as­ter wait­ing to hap­pen. In this en­vi­ron­ment, that makes O’Neill a cred­i­ble threat again, though it seems far fetched.

But he’s still go­ing to make every­one’s list. Fun­ny how that works.

Richard Paz­dur

How about Richard Paz­dur, the on­col­o­gy czar at the FDA whose com­mit­ment to rapid drug re­views has played a ma­te­r­i­al role in rev­o­lu­tion­iz­ing on­col­o­gy R&D over the last 5 years? It’s been sug­gest­ed. But when it comes to agency in­sid­ers, though, no one out­shines Janet Wood­cock at CDER. She not on­ly knows where the bod­ies are buried at the FDA, she of­ten helped put them there. Maybe she could cap a decades-long run as FDA chief? 

Janet Wood­cock

Stranger things have hap­pened.

Two years ago I called Got­tlieb the Re­pub­li­can shad­ow com­mis­sion­er, out front of­fer­ing po­si­tions on re­form­ing the FDA. We don’t seem to have one of those now, by virtue of the fact that no one ex­pect­ed Got­tlieb to stand down so soon. And with a na­tion­al elec­tion loom­ing next year with a wild­ly con­tro­ver­sial pres­i­dent, any­one who does get the job may ul­ti­mate­ly prove an in­ter­im chief in any case.

Bio­phar­ma hates reg­u­la­to­ry un­cer­tain­ty of any kind. This year we’ve had a gov­ern­ment shut­down fol­lowed by an abrupt res­ig­na­tion by Got­tlieb. Un­cer­tain­ty at the FDA ap­pears to now be the rule. 

In a note to in­vestors, Jef­feries’ Michael Yee not­ed that who­ev­er steps in will find an agency that has es­tab­lished a “pro-in­no­va­tion” ap­proach that won’t sud­den­ly end now, re­gard­less of who gets the job.

That sounds rea­son­able, but it won’t stop the fret­ting that’s go­ing on now. Ap­proach­es are one thing, ex­e­cu­tion is an­oth­er.

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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[via AP Images]

Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.