Scott Got­tlieb aced his con­fir­ma­tion hear­ing, vow­ing to main­tain R&D gold stan­dard

Scott Got­tlieb – April 5, 2017 Zach Gib­son/Get­ty Im­ages


Sen­ate De­moc­rats didn’t waste any time in rais­ing their ob­jec­tions to Scott Got­tlieb as the next FDA com­mis­sion­er.

Sen­a­tor Pat­ty Mur­ray (D-Wash­ing­ton) start­ed her re­marks at Got­tlieb’s con­fir­ma­tion hear­ing this morn­ing voic­ing her con­cerns about “whether you can with­stand po­lit­i­cal pres­sure” in main­tain­ing an un­bi­ased, sci­ence-based ap­proach in main­tain­ing the gold stan­dard on safe­ty and ef­fi­ca­cy re­views of new drugs.

Mur­ray com­plained that sen­a­tors had on­ly days to re­view Got­tlieb’s “un­prece­dent­ed fi­nan­cial en­tan­gle­ments” in the phar­ma in­dus­try — with dozens of drugs from the com­pa­nies he’s worked with head­ed to the FDA — as well as more than 800 list­ed pub­li­ca­tions which “raise con­cerns about po­ten­tial con­flicts of in­ter­est.”

Scott Got­tlieb this morn­ing on Capi­tol Hill at his nom­i­na­tion hear­ing. (CRED­IT: C-SPAN/End­points News)

Got­tlieb, though, came pre­pared to re­but those sug­ges­tions, mak­ing promis­es to main­tain an in­de­pen­dent FDA com­mit­ted to find­ing new ef­fi­cien­cies at the agency while mak­ing sure that all the new prod­ucts the agency ap­proves are safe and ef­fec­tive, meet­ing the agency’s long-term gold stan­dard on R&D.

“I’ve seen the im­por­tance of the FDA’s work as both a doc­tor and a pa­tient,” Got­tlieb, a can­cer sur­vivor, said in his open­ing state­ment. He added that his in­vest­ments and en­deav­ors were aimed at im­prov­ing health­care in the US. Some have worked, oth­ers didn’t and many oth­ers are still in de­vel­op­ment.

Re­gard­less of those ties, he vowed to be an “im­par­tial and in­de­pen­dent ad­vo­cate for the pub­lic health” main­tain­ing a rig­or­ous ap­proach to re­view­ing drugs. “We’re at an in­flec­tion point in bio­med­ical sci­ence,” he added, and the chal­lenge at the FDA will be get­ting the most bang for its reg­u­la­to­ry bucks.

“We should re­ject the false di­choto­my that it all boils down to a choice be­tween speed and safe­ty.” Bet­ter ef­fi­cien­cy and safe­ty are both pos­si­ble, he said, while main­tain­ing the gold stan­dard. And he added that “it’s in­cum­bent on us to have a world class work force,” hir­ing the best for reg­u­la­to­ry work, which Sen­a­tor Lamar Alexan­der not­ed was Got­tlieb’s pick as his most im­por­tant chal­lenge.

Got­tlieb al­so helped soothe any in­dus­try con­cerns that he might want to come in as an abra­sive re­former, work­ing at odds with staff.

“I think every­thing is a bot­tom-up ap­proach in the agency,” he told the law­mak­ers. “The ideas re­al­ly need to come from the ca­reer staff in the cen­ters.” That’s go­ing to be a chal­lenge as the agency re­cruits re­place­ments for a line­up of se­nior agency of­fi­cials who are due for re­tire­ment.

Got­tlieb promised to bring best prac­tices to those ar­eas in the FDA that have been lag­ging. There’s been an “un­even adop­tion” of the break­through drug pro­gram, he added, which now re­quires broad adop­tion.  The prospec­tive com­mis­sion­er al­so said that while he was not crit­i­cal of Phase III, there is a pos­si­bil­i­ty with new tech­nol­o­gy to com­press Phase II and Phase III in­to an adap­tive de­sign.

And, as ex­pect­ed, he rel­ished the prospect of tack­ling a slew of com­plex gener­ics await­ing an ap­proval at the FDA. Many drugs are sold at high prices, he said, but should be sub­ject to com­pe­ti­tion.

A few days ago Got­tlieb care­ful­ly de­lin­eat­ed ties to some two dozen bio­phar­ma com­pa­nies he’s been work­ing with over the past eight years of the Oba­ma ad­min­is­tra­tion. He’s worked with Glax­o­SmithK­line, Ver­tex and Bris­tol-My­ers, so you can check the box on in­dus­try con­flicts of in­ter­est that De­moc­rats will con­demn.

Among Got­tlieb’s oth­er lines of work, the for­mer deputy FDA com­mis­sion­er penned a se­ries of ar­ti­cles on bio­phar­ma for Forbes, and the Dems have been giv­ing them a close look. Two pieces float­ed to the top of the read­ing list. One cen­tered on his pro­pos­al to move re­spon­si­bil­i­ty for iden­ti­fy­ing the opi­oid sup­pli­ers who were break­ing the law from the DEA to HHS. The oth­er dealt with his crit­i­cism of the REMS pro­grams, which set up post-mar­ket­ing safe­ty pro­grams to lim­it risks seen in cer­tain drugs.

Add it all up, says Sher­rod Brown (D-Ohio), and you find some­one who will “roll over for his Big Phar­ma friends,” as The Hill re­ports.

Re­pub­li­cans, though, were large­ly con­tent to lim­it their com­ments to praise for the nom­i­nee and his three young daugh­ters, who were seat­ed be­hind the can­di­date. And it’s the Re­pub­li­cans who have the votes to make this hap­pen.

The in­dus­try, mean­while, has made it clear that af­ter hear­ing some of the ideas backed by oth­er com­mis­sion­er can­di­dates like Jim O’Neill – who’s sug­gest­ed ap­prov­ing drugs as soon as they’re de­ter­mined safe — Got­tlieb is the best can­di­date by far. In a snap poll of qual­i­fied End­points News sub­scribers, 87% of the 580 who reg­is­tered an opin­ion backed Got­tlieb’s nom­i­na­tion.

So you can ex­pect most peo­ple in bio­phar­ma to keep their fin­gers crossed for Got­tlieb.

Bot­tom line: Got­tlieb came well pre­pared for every ques­tion, care­ful not to step be­yond the bound­aries of the com­mis­sion­er’s job when in­vit­ed to com­ment on Oba­macare. His an­swers were pol­ished and prac­ticed, leav­ing lit­tle room for un­ex­pect­ed at­tacks. And he de­flect­ed crit­i­cism eas­i­ly, promis­ing to avoid any po­ten­tial con­flicts of in­ter­est.

Bar­ring a last minute sur­prise, Got­tlieb’s con­fir­ma­tion as the next FDA com­mis­sion­er is all but guar­an­teed.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

[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.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.