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.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Vicente Anido (University of West Virginia via YouTube)

Aerie fires CEO af­ter lead pro­gram flop, com­ments about pri­ma­ry end­points be­ing 'not re­quired'

Aerie Pharmaceuticals CEO Vicente Anido has left the company less than a week after trying to chart a Phase III study in the wake of a serious Phase IIb flop.

Anido’s last day at Aerie was Friday, the biotech announced in a news release Tuesday morning, and Benjamin McGraw is taking his place in an interim role. The now former CEO was terminated without cause, according to an SEC filing.

The board has started looking for a full-time chief to take his place.

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When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

Take­da snaps up the Japan­ese rights to an old Shire cast-off; Boehringer In­gel­heim ac­quires Abexxa Bi­o­log­ics

A week before the FDA is set to decide on Mirum Pharmaceuticals’ lead liver disease drug — an old Shire cast-off called maralixibat — Takeda is swooping in to secure the rights in Japan.

Maralixibat’s roots trace back to Lumena, which was snapped up by Shire for $260 million-plus back in 2014. While the candidate had failed mid-stage studies at Shire, Mirum believes better trial design and patient selection will deliver the wins it needs. The drug is currently in development for Alagille syndrome (a condition called ALGS in which bile builds up in the liver), progressive familial intrahepatic cholestasis (PFIC, which causes progressive liver disease) and biliary atresia (a blockage in the ducts that carry bile from the liver to the gallbladder).