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.

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

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?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

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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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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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

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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Ku­ra co-founder heads to Asian mul­ti-na­tion­al as biotech eyes the goal posts for lead drug

Six years after Kura Oncology snagged a farnesyl transferase inhibitor from J&J and leapt straight into clinical development, one of the biotech’s founders is leaving to start a new chapter in his career.

CMO and development chief Antonio Gualberto is exiting the company, and Kura — led by longtime biotech entrepreneur Troy Wilson — is on the hunt for a replacement. Wilson credited the CMO for some key biomarker work, including the discovery of the CXCL12 pathway as a target of their lead drug tipifarnib. Those biomarkers are being relied on to define the patient population most likely to benefit from the drug.

FDA waves Epizyme's $186K rare can­cer drug through to mar­ket — now get ready for the sec­ond act

After winning the hearts of the expert panel convened by the FDA despite a bleak in-house review and a checkered development history, Robert Bazemore has steered Epizyme to its first-ever OK for a rare cancer drug.

The approval in epithelioid sarcoma sets tazemetostat, now Tazverik, up nicely for a quick expansion to follicular lymphoma — a much bigger indication for which the biotech has just submitted an NDA.

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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2019 a 'trans­for­ma­tive year' for phar­ma M&A. Is that a good thing?

Big Pharma keeps getting bigger.

Fueled by the mega-mergers between Bristol-Myers Squibb and Celgene and between Allergan and AbbVie, the industry last year saw $350 billion worth of M&A, according to the new year-end report from the consultants at PwC.  That’s a more than 50% increase on 2018.

“I kind of look at 2019 as a transformational year,” report author Glen Hunzinger told Endpoints News. 

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