FDA com­mis­sion­er Scott Got­tlieb sets some goals: Find ways to low­er drug prices, use new tech to speed de­vel­op­ment

Scott Got­tlieb start­ed his first full week as FDA com­mis­sion­er by lay­ing out some goals and guide­lines for the agency now un­der his di­rec­tion, hop­ing to al­le­vi­ate some con­cerns he’s been hear­ing about the fu­ture. In an all-hands speech to staff on Mon­day, Got­tlieb re­it­er­at­ed some now fa­mil­iar themes on what he hopes to ac­com­plish, and how every­one at the FDA is now ex­pect­ed to lend a hand.

Aside from some health ini­tia­tives on smok­ing and com­bat­ing the opi­oid ad­dic­tion cri­sis in the coun­try, Got­tlieb pledged to find new ways to low­er drug prices, push­ing through com­plex gener­ics and biosim­i­lars while lay­ing the ground­work to pre­vent any more sweet­heart deals to de­lay the ar­rival of low-cost knock­offs, im­ply­ing that he’s prepar­ing to take ac­tion on that front.

He not­ed:

We al­so need to take steps to make sure the gener­ic drug process isn’t be­ing in­ap­pro­pri­ate­ly gamed to de­lay com­pe­ti­tion and dis­ad­van­tage con­sumers. I hope to have much more to say on this top­ic in the com­ing weeks.

In the area of new drug de­vel­op­ment:

Con­gress gave us a clear man­date to be for­ward-lean­ing when it comes to how we’ll eval­u­ate safe­ty and ef­fi­ca­cy in view of emerg­ing sci­en­tif­ic in­sight and bet­ter an­a­lyt­i­cal tools. Im­ple­ment­ing the 21st Cen­tu­ry Cures Act is a key pri­or­i­ty. We need to make sure we’re tak­ing steps to fos­ter in­no­va­tion and reg­u­lat­ing ar­eas of promis­ing new tech­nol­o­gy in ways that don’t raise the cost of de­vel­op­ment or re­duce in­no­va­tion….

We need to be pa­tient-cen­tric and sci­ence-based in every­thing we do. And, we must make sure that in all our ef­forts, we main­tain the gold stan­dard for reg­u­la­to­ry sci­ence and in­de­pen­dent, sci­ence-led de­ci­sion-mak­ing, all led by a strong ca­reer work­force.

Got­tlieb al­so high­light­ed a change­up in the Of­fice of Reg­u­la­to­ry Af­fairs. As Bloomberg re­port­ed ear­li­er Mon­day, Got­tlieb is fo­cus­ing work­ers on spe­cif­ic ar­eas like drugs and de­vices in or­der to achieve greater ex­per­tise as they in­spect fa­cil­i­ties and probe con­sumer com­plaints, aban­don­ing the old re­gion­al fo­cus with shared re­spon­si­bil­i­ties.

“As ORA’s man­date be­comes more com­plex and more glob­al, we look for­ward to achiev­ing op­er­a­tional ef­fi­cien­cies that can im­prove our abil­i­ty to ful­fill our pub­lic health mis­sion and pro­tect con­sumers,” not­ed Got­tlieb.

None of this is a dra­mat­ic de­par­ture for the FDA. But Got­tlieb is al­so send­ing the mes­sage that busi­ness as usu­al won’t be the theme for his tenure as head of the FDA. His im­me­di­ate pre­de­ces­sor, Robert Califf, of­fered a quick thumbs up for get­ting start­ed ear­ly with a re­or­ga­ni­za­tion.

A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

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 59,900+ biopharma pros reading Endpoints daily — and it's free.

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 59,900+ 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.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

Mer­ck helps bankroll new part­ner Themis' game plan to fin­ish the chikun­gun­ya race and be­gin on­colyt­ic virus quest

As Themis gears up for a Phase III trial of its chikungunya vaccine, the Vienna-based biotech has closed out €40 million ($44 million) to foot the clinical and manufacturing bills.

Its heavyweight partners at Merck — which signed a pact around a mysterious “blockbuster indication” last month — jumped into the Series D, led by new investors Farallon Capital and Hadean Ventures. Adjuvant Capital also joined, as did current investors Global Health Investment Fund, aws Gruenderfonds, Omnes Capital, Ventech and Wellington Partners Life Sciences.