With Trump’s new FDA com­mish nom­i­nee loom­ing, in­dus­try ex­ecs are keep­ing their fin­gers crossed for Scott Got­tlieb

Scott Got­tlieb

Last week Pres­i­dent Trump said he was just days away from an­nounc­ing an ab­solute­ly “fan­tas­tic” new FDA com­mis­sion­er. One who would cut up to 80% of the reg­u­la­tions around drug de­vel­op­ment, blaz­ing a short­er path to an ap­proval. One who would get ex­per­i­men­tal meds to dy­ing pa­tients, over­haul­ing time­lines and slash­ing de­vel­op­ment bud­gets.

By and large, the mes­sage has spurred more than a lit­tle trep­i­da­tion in the in­dus­try that the pres­i­dent, who has earned a rep­u­ta­tion for shoot­ing first and ask­ing ques­tions lat­er, was go­ing to rad­i­cal­ly change the ap­proval process at the FDA. And that might in­di­cate that lib­er­tar­i­an “seast­ead­er” Jim O’Neill — who has fa­mous­ly said that drugs should be ap­proved on safe­ty alone — has the in­side track with the new pres­i­dent.

But he’s not the in­dus­try’s first choice. That dis­tinc­tion ap­pears to be re­served al­most en­tire­ly for Scott Got­tlieb, the for­mer deputy com­mis­sion­er at the FDA un­der George W. Bush with a rep­u­ta­tion as a staunch con­ser­v­a­tive with no known ap­petite for toss­ing grenades in­to the reg­u­la­to­ry un­der­brush.

Mizuho Se­cu­ri­ties USA ran a quick sur­vey of 53 drug com­pa­nies ask­ing them which of the four can­di­dates men­tioned so far in the me­dia — Got­tlieb, O’Neill, Bal­a­ji Srini­vasan and Joseph Gul­fo — would get their vote.

Sev­en­ty-two per­cent picked Got­tlieb. The rest were al­so rans. O’Neill got a hand­ful of votes at 8%, with Gul­fo at 9% and Srini­vasan at a mere 2%. (Gul­fo has pro­mot­ed his own can­di­da­cy with­out any in­de­pen­dent con­fir­ma­tion from the Trump ad­min­is­tra­tion that he’s ever been a se­ri­ous can­di­date or been with­in 100 feet of the new pres­i­dent.)

In his week­ly roundup on Fri­day, Baird’s Bri­an Sko­r­ney came to the same con­clu­sion with­out any polling. He wrote: “We be­lieve in­dus­try and in­vestor sup­port is over­whelm­ing­ly in fa­vor of Scott Got­tlieb.”

“He leans right, he’s got ex­pe­ri­ence in the agency, he’s got the M.D. cre­den­tial, and he’s out­spo­ken,” Michael Ga­ba, fed­er­al pol­i­cy leader of law firm Hol­land & Knight’s na­tion­al Health­care & Life Sci­ences Team, told Reuters re­cent­ly.

I asked Ga­ba to elab­o­rate on that. And he sent me this:

When it comes to the FDA, bio­phar­ma, and reg­u­lat­ed in­dus­try as a whole, puts a pre­mi­um on pre­dictabil­i­ty, speed to mar­ket, and the abil­i­ty to make dif­fer­en­ti­at­ing claims in the mar­ket­place about their prod­ucts’ ef­fec­tive­ness to treat and mit­i­gate dis­ease.  Safe­ty is the giv­en once a prod­uct makes it through the FDA, and by it­self doesn’t dis­tin­guish prod­ucts in the mar­ket­place.  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.

The theme among the oth­er can­di­dates ap­pears to be to speed prod­ucts to mar­ket by lim­it­ing the agency’s role to eval­u­at­ing safe­ty on­ly.  Not on­ly would this dy­nam­ic be chal­leng­ing to in­dus­try, providers and pa­tients, it would re­quire a statu­to­ry change to the FD­CA – and we all know that the leg­isla­tive process it­self can be very messy and un­pre­dictable.

Got­tlieb’s nom­i­na­tion would con­tin­ue a long­stand­ing tra­di­tion of nam­ing a trained physi­cian for the top job at the FDA. As a res­i­dent fel­low at the Amer­i­can En­ter­prise In­sti­tute, he’s kept the heat on Oba­macare through a se­ries of op-eds in high pro­file pub­li­ca­tions like the Wall Street Jour­nal. 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 an ad­vi­sor to Glax­o­SmithK­line, which has shown no ap­petite for rad­i­cal change in the way de­vel­op­ers prove a drug works.

It’s the kind of re­sume that would in­di­cate a sym­pa­thy for con­tin­u­ing to evolve reg­u­la­tions to speed de­vel­op­ment, but with­out any ap­petite for play­ing rev­o­lu­tion­ary.

Of course, that’s one rea­son why he may not get the job. Trump has glee­ful­ly named new cab­i­net mem­bers based on their hos­til­i­ty to the agen­cies they’ll be run­ning and the reg­u­la­tions they’ve been in­volved in cre­at­ing. And his ex­ec­u­tive or­der de­mand­ing the elim­i­na­tion of two rules for every new one un­der­scores his com­mit­ment to elim­i­nat­ing hun­dreds of pages of regs for every one that sur­vives.

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For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
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A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

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UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

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Deer­field vaults to the top of cell and gene ther­a­py CD­MO game with $1.1B fa­cil­i­ty at Philadel­phi­a's newest bio­phar­ma hub

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Tri­fec­ta of sick­le cell dis­ease ther­a­pies ex­tend life ex­pectan­cy, but are not cost-ef­fec­tive — ICER

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Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.