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.

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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New stan­dard of care? FDA hands Pfiz­er, Mer­ck KGaA an OK for Baven­cio in blad­der can­cer

The breakthrough therapy designation Pfizer and Merck KGaA notched for Bavencio in bladder cancer has quickly paved way for a full approval.

The PD-L1 drug is now sanctioned as a first-line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma, applicable in cases where cancer hasn’t progressed after platinum-containing chemotherapy.

Petros Grivas, the principal investigator of the supporting Phase III JAVELIN Bladder 100, called the approval “one of the most significant advances in the treatment paradigm in this setting in 30 years.”

On a roll, Mer­ck blazes through a new seg­ment of the bio­mark­er trail

Merck has notched an approval for using Keytruda to treat a biomarker-based subset of first-line colorectal cancer patients with unresectable or metastatic tumors, as the pharma giant continues to find new niches for its blockbuster PD-1 star.

The OK is significant in a number of ways. Not only does it build on an accelerated approval for all tumors characterized as microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR); it also marks the first single treatment for colorectal cancer that doesn’t contain chemotherapy.

Vas Narasimhan, Novartis CEO (Patrick Straub/​EPA-EFE/​Shutterstock)

No­var­tis pays $678M for kick­back scheme as Vas Narasimhan tries to dis­tance phar­ma gi­ant from shady be­hav­ior

Novartis has reached another large settlement to resolve misconduct allegations, agreeing to pay more than $678 million to settle claims that it had spent hundreds of millions of dollars on lavish dinners, so-called speaking fees and expensive alcohol “that were nothing more than bribes” to get doctors to prescribe Novartis medications.

The top-shelf alcohol and lavish meals included a $3,250 per person night at Nobu in Dallas, a $672-per person dinner at Washington DC’s Smith & Wollensky and a $314 per person meal at Sushi Roku in Pasadena, according to the Justice Department complaint. There were at least 7 trips to Hooters and fishing trips in Alaska and off the Florida coast. Each of these events were supposed to be “speaker programs” where doctors educated other doctors on a drug, but the DOJ alleged many were “bogus” wine-and-dine events where the drug was barely mentioned, if at all.  (“Nobody presented slides on the fishing trips,” the complaint says.)

No­var­tis los­es biosim­i­lar ap­peal as court up­holds a 31-year mo­nop­oly by Am­gen's En­brel

A new court ruling has strengthened Amgen’s grip on the IP estate around Enbrel, keeping biosimilars of the autoimmune and inflammatory drug at bay until 2029.

Novartis, the patent challenger, isn’t throwing in the towel yet. In a statement noting the failed appeal, its generics division Sandoz noted its reviewing options, “including potential appeal to US Supreme Court.”

It’s been almost four years since the FDA approved Erelzi, Sandoz’s copycat version of Enbrel. While sales of the Pfizer-partnered drug in the US — the market Amgen is in charge of — have dipped slightly during that time, it remains a solid megablockbuster with 2019 revenue slightly above $5 billion.